GNE Myopathy, Hereditary Inclusion Body Myopathy (HIBM)
Conditions
Brief summary
The safety objectives of the study are to: evaluate additional long-term safety of SA-ER treatment of participants with GNE myopathy previously treated with SA-ER at dose of 6g/day (Part I); evaluate the safety of 12g /day SA (delivered by 1.5g of SA-ER tablets and 1.5g of SA-IR capsules 4 times per day) in the treatment of participants with GNE myopathy (Part II) over a 6 month treatment period; evaluate the safety of SA treatment at both 6g/day and 12 g/day (Part III \[SA-ER/SA-IR\] and Part IV \[SA-ER\]).
Interventions
DRUGSA-ER 500 mg
oral tablets
DRUGSA-IR 500 mg
oral capsules
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Enrollment in, and successful completion of the UX001-CL201 (NCT01517880) protocol OR (for 10 treatment naïve subjects): * Have a confirmed diagnosis of GNE Myopathy * Aged 18 -65 years of age, inclusive * Able to walk ≥ 200 meters and \< 80% of predicted normal during the 6-Minute Walk Test (6MWT; orthotics and assistive devices allowed) * Must be willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures * Must be willing and able to comply with all study procedures * Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study * Females of childbearing potential must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause for at least two years, or have had tubal ligation at least one year prior to Baseline, or who have had total hysterectomy
Exclusion criteria
* Use of any investigational product (other than SA-ER tablets) to treat GNE myopathy * Ingestion of N-acetyl-D-mannosamine (ManNAc) or similar SA-producing compounds * Pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study * Has had any hypersensitivity to SA or its excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects * Have any co-morbid conditions, including unstable major organ-system disease(s) that in the opinion of the investigator, places the subject at increased risk of complications, interferes with study participation or compliance, or confounds study objectives.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE (SAE) is an AE that at any dose results in any of the following: death, a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. TEAEs include all adverse events that start on or after the first dose of study medication, or adverse events that are present prior to the first dose of study medication, but their severity or relationship increases after the first dose of study medication up to and including 30 days after the final study medication dosing date. TEAEs were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). TEAE relationship to study medication was classified as not related, possibly related, or probably related. |
| Clinically Significant Changes From Baseline in Vital Signs, Physical and Neurological Examination Findings and Laboratory Evaluations | From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants | — |
| Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
| Interval History: Typical Number of Falls Per Year | Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 6, 12, 18, 24, 30, 36, study termination | Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments. |
Countries
Israel, United States
Participant flow
Recruitment details
Participants completing study UX001-CL201 (NCT01517880) were eligible to continue treatment under this protocol. Additional participants with glucosamine (UDP-N-acetyl)-2-epimerase (GNE) myopathy were enrolled to assess Sialic Acid Extended Release/Sialic Acid Immediate Release (SA-ER/SA-IR) in a treatment naïve population.
Pre-assignment details
A total of 46 crossover participants started the study in Part 1, with an additional 13 naive participants starting the study in Part 2.
Participants by arm
| Arm | Count |
|---|---|
| Crossover Participants Participants completing the 48-week study (UX001-CL201) were enrolled into Part I of the study:
* Part I: participants continued on 6 g/day SA-ER for approximately 6 months
* Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment 4 times per day \[QID\]) for 36 months
* Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR)
* Part IV: 6 g/day or 12 g/day SA (SA-ER only) | 46 |
| Naïve Participants Treatment naïve participants with GNE myopathy were enrolled into Part II of the study:
* Part II: 12 g/day SA (1.5 g of SA-ER and 1.5 g of SA-IR treatment QID) for 36 months
* Part III: 6 g/day or 12 g/day SA (both SA-ER and SA-IR)
* Part IV: 6 g/day or 12 g/day SA (SA-ER only) | 13 |
| Total | 59 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part II | Withdrawal by Subject | 2 | 2 |
| Part III | Noncompliance | 1 | 0 |
| Part III | Withdrawal by Subject | 1 | 0 |
| Part IV | Non-Compliance | 1 | 0 |
| Part IV | Withdrawal by Subject | 2 | 1 |
Baseline characteristics
| Characteristic | Naïve Participants | Total | Crossover Participants |
|---|---|---|---|
| Age, Continuous | 35.6 years STANDARD_DEVIATION 10.97 | 39.9 years STANDARD_DEVIATION 10.79 | 41.1 years STANDARD_DEVIATION 10.55 |
| Race/Ethnicity, Customized Asian | 0 Participants | 8 Participants | 8 Participants |
| Race/Ethnicity, Customized Hispanic/Latino | 0 Participants | 2 Participants | 2 Participants |
| Race/Ethnicity, Customized Not Hispanic/Latino | 13 Participants | 57 Participants | 44 Participants |
| Race/Ethnicity, Customized White | 13 Participants | 51 Participants | 38 Participants |
| Sex: Female, Male Female | 8 Participants | 37 Participants | 29 Participants |
| Sex: Female, Male Male | 5 Participants | 22 Participants | 17 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk |
|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 46 | 0 / 46 | 0 / 46 | 0 / 1 | 0 / 13 | 0 / 59 |
| other Total, other adverse events | 42 / 46 | 44 / 46 | 46 / 46 | 1 / 1 | 13 / 13 | 59 / 59 |
| serious Total, serious adverse events | 2 / 46 | 0 / 46 | 2 / 46 | 0 / 1 | 1 / 13 | 3 / 59 |
Outcome results
Primary
Clinically Significant Changes From Baseline in Vital Signs, Physical and Neurological Examination Findings and Laboratory Evaluations
Time frame: From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants
Population: Clinically significant changes from Baseline for vital signs, physical and neurological examination findings and laboratory evaluations were recorded as AEs and are reported as part of the Safety section of this record and Outcome Measure 1.
Primary
Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit?
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Time frame: Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination
Population: All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): No | 17 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I Month 6: Yes | 6 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Baseline: Yes | 28 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 1: Yes | 36 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 1: No | 10 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 3: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 3: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 6: Yes | 27 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 6: No | 19 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 9: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 9: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 12: Yes | 29 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 12: No | 17 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 15: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 15: No | 1 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 18: Yes | 18 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 18: No | 21 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 21: Yes | 18 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 24: Yes | 12 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 24: No | 2 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 27: Yes | 21 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 27: No | 21 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 33: Yes | 11 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 33: No | 5 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 36: Yes | 10 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 36: No | 13 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Termination: No | 1 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): Yes | 29 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part I Month 6: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 21: No | 12 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Baseline: No | 18 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Baseline: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Baseline: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 18: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 1: Yes | 12 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 33: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 1: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 21: Yes | 8 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 3: Yes | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 21: No | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 3: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 18: Yes | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 6: Yes | 7 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 36: Yes | 6 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 6: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 24: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 9: Yes | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Termination: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 9: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 27: Yes | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 12: Yes | 6 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 36: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 12: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 27: No | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 15: Yes | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 24: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 15: No | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Experienced Any New Conditions or Exacerbations of an Existing Condition Since Last Study Visit? | Part II-IV Month 33: Yes | 2 Participants |
Primary
Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit?
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Time frame: Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination
Population: All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 18: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Baseline: No | 37 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 18: No | 35 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 6: Yes | 9 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 21: Yes | 3 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I Month 6: Yes | 3 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 21: No | 27 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 6: No | 37 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 24: Yes | 2 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 1: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 27: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 9: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 27: No | 38 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I Month 6: No | 3 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 33: Yes | 3 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 9: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 33: No | 13 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 1: No | 42 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 36: Yes | 3 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 12: Yes | 5 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 36: No | 20 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Baseline: Yes | 9 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 12: No | 41 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Termination: No | 1 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 3: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 24: No | 12 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): Yes | 5 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 15: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): No | 41 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 15: No | 5 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 3: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 24: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Baseline: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Baseline: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 1: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 1: No | 13 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 3: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 3: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 6: Yes | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 6: No | 7 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 9: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 9: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 12: Yes | 2 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 12: No | 8 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 15: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 15: No | 8 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 18: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 18: No | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 21: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 21: No | 11 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 24: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 27: Yes | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 27: No | 9 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 33: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 33: No | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 36: Yes | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Month 36: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Receiving Any New Therapy or Discontinued Any Therapies Since Last Study Visit? | Part II-IV Termination: No | 0 Participants |
Primary
Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit?
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Time frame: Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 3, 6, 9, 12, 15, 18, 21, 24, 27, 33, 36, study termination
Population: All participants who received at least one dose of study drug. Participant n at each time point includes those with a yes or no response. Naïve Participants did not enter the study until Part II.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 6: Yes | 24 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 21: Yes | 19 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 1: Yes | 15 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 21: No | 11 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 6: No | 22 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Baseline: Yes | 23 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 24: No | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 9: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 27: Yes | 19 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 27: No | 23 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 1: No | 31 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 33: Yes | 12 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 9: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 33: No | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I Month 6: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 36: Yes | 12 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 12: Yes | 27 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 36: No | 11 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 3: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 12: No | 19 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Termination: No | 1 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Baseline: No | 23 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): Yes | 21 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 15: No | 1 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 15: Yes | 4 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 24: Yes | 10 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I Baseline (Study UX001-CL201 Week 48): No | 25 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 3: No | 0 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 18: Yes | 17 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part I Month 6: No | 2 Participants |
| Crossover Participants; 6 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 18: No | 22 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 27: Yes | 6 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Baseline: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Baseline: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 1: Yes | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 1: No | 9 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 3: Yes | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 3: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 6: Yes | 7 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 6: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 9: Yes | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 9: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 12: Yes | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 15: Yes | 3 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 15: No | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 18: Yes | 2 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 18: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 21: Yes | 7 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 21: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 24: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 24: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 27: No | 4 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 33: Yes | 2 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 33: No | 1 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 36: Yes | 5 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 36: No | 2 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Termination: Yes | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Termination: No | 0 Participants |
| Crossover Participants: 12 g/Day | Interval History: Has the Participant Started Taking Any New Medications or Discontinued Any Medications Since the Study Visit? | Part II-IV Month 12: No | 5 Participants |
Primary
Interval History: Typical Number of Falls Per Year
Each interval history was intended to record any signs, symptoms, or events (e.g., falls, changes in medications or therapies) experienced by the participant since the prior study visit that were not related to study procedure(s) performed at prior study visits or study drug. Interval history may have included exacerbation or improvement in existing medical conditions (including the clinical manifestations of GNE myopathy) that might have interfered with study participation, safety, and/or positively or negatively impact performance of functional assessments.
Time frame: Part I: Baseline (Study UX001-CL201 Week 48), Month 6; Part II-IV: Baseline, Months 1, 6, 12, 18, 24, 30, 36, study termination
Population: All participants who received at least one dose of study drug. Naïve Participants did not enter the study until Part II.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part I Baseline (Study UX001-CL201 Week 48) | 11.0 falls | Standard Deviation 53.63 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part I Month 6 | 3.8 falls | Standard Deviation 3.54 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Baseline | 14.1 falls | Standard Deviation 53.66 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 1 | 6.8 falls | Standard Deviation 9.6 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 6 | 8.0 falls | Standard Deviation 11.87 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 12 | 5.4 falls | Standard Deviation 8.94 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 18 | 7.8 falls | Standard Deviation 14.58 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 24 | 9.4 falls | Standard Deviation 28.23 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 30 | 6.2 falls | Standard Deviation 10.03 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 36 | 5.4 falls | Standard Deviation 9.03 |
| Crossover Participants; 6 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Termination | 4.0 falls | — |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 18 | 6.0 falls | Standard Deviation 4.15 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Baseline | 6.0 falls | Standard Deviation 6.19 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Termination | 12.0 falls | — |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 1 | 7.3 falls | Standard Deviation 6.31 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 24 | 4.5 falls | Standard Deviation 3.05 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 6 | 5.6 falls | Standard Deviation 6.39 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 36 | 7.6 falls | Standard Deviation 10.88 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 12 | 5.1 falls | Standard Deviation 3.51 |
| Crossover Participants: 12 g/Day | Interval History: Typical Number of Falls Per Year | Part II-IV Month 30 | 5.0 falls | Standard Deviation 6.56 |
Primary
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation
An adverse event (AE) is defined as any untoward medical occurrence, whether or not considered drug related. A serious AE (SAE) is an AE that at any dose results in any of the following: death, a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; a congenital anomaly/birth defect. TEAEs include all adverse events that start on or after the first dose of study medication, or adverse events that are present prior to the first dose of study medication, but their severity or relationship increases after the first dose of study medication up to and including 30 days after the final study medication dosing date. TEAEs were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). TEAE relationship to study medication was classified as not related, possibly related, or probably related.
Time frame: From first dose of study drug until up to 30 days after the last dose of study drug. Mean (SD) duration of treatment was 1170.0 (170.2) days for Crossover Participants and 897 (380) days for Naïve Participants
Population: All participants who received at least one dose of study drug.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 22 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 26 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 43 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 2 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 2 Participants |
| Crossover Participants; 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 19 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 23 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 40 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 46 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 0 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 0 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 23 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Crossover Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 15 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 2 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 29 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 2 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 45 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 46 Participants |
| Crossover Participants: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 1 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 1 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Naïve Participants: 6 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 1 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 1 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 13 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 5 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 1 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 11 Participants |
| Naïve Participants: 12 g/Day | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 7 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Serious TEAEs | 3 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 1 | 20 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 2 | 36 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | >/= 1 TEAE | 59 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 4 | 0 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 3 | 3 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Treatment Related TEAEs | 56 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAE Maximum Severity = Grade 5 | 0 Participants |
| Overall: Any Dose | Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | TEAEs Causing Discontinuation | 0 Participants |