Glucose Transporter Type 1 Deficiency Syndrome
Conditions
Keywords
Glut 1 DS
Brief summary
The primary objective of the study is to evaluate the long-term safety of UX007 in Glut1 DS participants.
Detailed description
The study will enroll up to 40 pediatric, adolescent and adult Glut 1 DS participants who have completed the UX007G-CL201 (NCT019933186) study and, at the discretion of the Sponsor, additional participants from other clinical studies, investigator sponsored trials (ISTs), or expanded access/compassionate use treatment.
Interventions
DRUGUX007
UX007 is a liquid intended for oral (PO) administration.
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Diagnosis of Glut 1 DS confirmed by cerebrospinal fluid glucose concentration. erythrocyte 3-O-methyl-D-glucose uptake assay, or solute carrier family 2 member 1 (SLC2A1) molecular genetic testing (Information obtained from Medical Records) * Males and females aged at least 1 year old at the time of informed consent * Completion of UX007G-CL201 study (NCT01993186). Glut1 DS patients who received UX007/triheptanoin treatment as apart of clinical studies, ISTs or expanded access/compassionate use treatment programs may be eligible at the discretion of the Sponsor * Provide written informed consent or verbal assent (if possible) with written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research related procedures * Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, and comply with accurate completion of the seizure diary * Females of childbearing potential must have a negative urine 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 not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause), or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy. * Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly-effective method of contraception as determined by the investigator from the period following the signing of the informed consent through 30 days after last dose of study drug.
Exclusion criteria
* Any known hypersensitivity to triheptanoin, that in the judgement of the investigator, places the subject at an increased risk for adverse effects * History of, or current suicidal ideation, behavior and/or attempts * Pregnant and/or breast feeding an infant * Unwilling or unable to discontinue use of prohibited medication (barbiturates, pancreatic lipase inhibitors) or other substance that may confound study objectives. Use of up to 3 concomitant antiepileptic drugs is allowed, provided dose has been stable at least 14 days prior to Baseline * Use of any Investigational Product, drug or supplement (other than UX007) within 30 days prior to Baseline, or at any time during the study * Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment * Has a concurrent disease or condition, or laboratory abnormality that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or introduce additional safety concerns
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days. | An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline Over Time in CNS Total Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36 | The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance & Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone & Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function. |
| Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. |
| Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36 | The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence. |
| Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. |
| Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18 | SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life. |
| Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30 | The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life. |
Countries
Australia, Denmark, Spain, United Kingdom, United States
Participant flow
Recruitment details
Study enrolled pediatric, adolescent, and adult glucose transporter type 1 deficiency syndrome (Glut1 DS) participants who completed the UX007G-CL201 study (NCT01993186; rollover participants). No non-rollover participants (those from other clinical studies, investigator sponsored trials, or expanded access/compassionate use treatment) enrolled.
Pre-assignment details
For continuing UX007G-CL201 participants, the Week 52 visit of that study may have been conducted in conjunction with the Baseline visit for this study to avoid duplication of assessments.
Participants by arm
| Arm | Count |
|---|---|
| UX007 (Triheptanoin) UX007 dosing was targeted and/or maintained at 35% of total daily caloric intake. | 15 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Discontinuation of Study by Sponsor | 9 |
| Overall Study | Other, Not Specified | 3 |
| Overall Study | Subject Non-Compliance | 2 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | UX007 (Triheptanoin) |
|---|---|
| Age, Continuous | 15.24 years STANDARD_DEVIATION 6.106 |
| Age, Customized 12 to < 18 years | 5 Participants |
| Age, Customized 18 to < 65 years | 5 Participants |
| Age, Customized 2 to < 12 years | 5 Participants |
| Baseline (NCT01993186) Columbia Neurological Score (CNS) Total Score | 38.63 score on a scale STANDARD_DEVIATION 25.428 |
| Baseline (NCT01993186) SF-10 Health Survey for Children Psychosocial Summary Score | 48.29 T-score STANDARD_DEVIATION 10.196 |
| Baseline (NCT01993186) SF-12v2 Health Survey MCS Score | 43.49 T-score STANDARD_DEVIATION 4.356 |
| Baseline (NCT01993186) SF12 Version 2 (SF-12v2) Health Survey Physical Component Summary (PCS) Score | 39.37 T-score STANDARD_DEVIATION 0.523 |
| Baseline (NCT01993186) Short Form (SF) 10 (SF-10) Health Survey for Children Physical Summary Score | 32.63 T-score STANDARD_DEVIATION 17.027 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 13 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Overall Seizure Frequency Per 4 Weeks | 312.17 seizures per 4 weeks STANDARD_DEVIATION 528.057 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Asian | 1 Participants |
| Race/Ethnicity, Customized Black or African American | 1 Participants |
| Race/Ethnicity, Customized Other, Not Specified | 1 Participants |
| Race/Ethnicity, Customized White | 11 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 11 Participants |
| Sex: Female, Male Female | 11 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 15 |
| other Total, other adverse events | 13 / 15 |
| serious Total, serious adverse events | 2 / 15 |
Outcome results
Primary
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths
An adverse event (AE) was defined as any untoward medical occurrence, whether or not considered drug related. Serious adverse events (SAEs) are AEs that at any dose, in the view of either the investigator or sponsor, results in any of the following outcomes: death; a life-threatening AE; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant incapacity or disability; a congenital anomaly/birth defect; other important medical event. An AE was considered a TEAE if it occurred on or after the first dose in this study, and was not present prior to the first dose in this study, or it was present at the first dose in this study but increased in severity during the study. Severity was based on Common Terminology Criteria for Adverse Events (CTCAE): 1 = mild, 2 = moderate, 3 = severe, 4 = life threatening, and 5 = death related to AE.
Time frame: From first dose of study drug up to 36 months. The mean (SD) treatment duration was 667.9 (357) days.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | TEAEs | 13 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | Serious TEAEs | 2 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | Related TEAEs | 10 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | Serious and Related TEAEs | 0 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | Grade 3 or 4 TEAEs | 1 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | Gastrointestinal TEAEs | 9 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | TEAEs Leading to Treatment Discontinuation | 0 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | TEAEs Leading to Study Discontinuation | 0 Participants |
| UX007 (Triheptanoin) | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Discontinuations Due to TEAEs, and Deaths | TEAEs Leading to Death | 0 Participants |
Secondary
Change From Baseline Over Time in CNS Total Score
The CNS evaluates measures of neurological function and development delay, and is the sum of scores for the following domains: Weight, Height, Head Circumference, General Medical Exam, Funduscopic Exam, Cranial Nerves, Stance & Gait, Involuntary Movements, Sensation, Cerebellar Function, Muscle Bulk, Tone & Strength, Myotatic Reflexes, Toe Sign, Other Findings. The CNS is only scored when all domains are measured and ranges from 0 (abnormal exam) to 76 (normal exam). Higher scores are associated with higher neurological function.
Time frame: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 24, Month 36
Population: Participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in CNS Total Score | Change at Month 0 | 11.85 score on a scale | Standard Deviation 20.331 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in CNS Total Score | Change at Month 6 | 9.36 score on a scale | Standard Deviation 18.495 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in CNS Total Score | Change at Month 12 | 13.64 score on a scale | Standard Deviation 21.371 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in CNS Total Score | Change at Month 24 | 3.38 score on a scale | Standard Deviation 3.092 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in CNS Total Score | Change at Month 36 | 0.00 score on a scale | — |
Secondary
Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks
The number of observable seizures were recorded by the subject or caregiver via diary throughout the study. Observable seizures were defined as: generalized tonic-clonic; generalized tonic; generalized clonic; generalized atonic; partial/focal with secondary generalization; myoclonic, myoclonic (astatic) atonic, myoclonic tonic; complex partial/focal; simple partial/focal motor; absence.
Time frame: Baseline (from NCT01993186), Month 0-3, Month 4-6, Month 7-9, Month 10-12, Month 13-18, Month 19-24, Month 25-30, Month 31-36
Population: Participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 0-3 | -64.22 seizures per 4 weeks | Standard Deviation 185.564 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 4-6 | -64.19 seizures per 4 weeks | Standard Deviation 142.46 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 7-9 | -61.81 seizures per 4 weeks | Standard Deviation 164.77 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 10-12 | -91.93 seizures per 4 weeks | Standard Deviation 216.834 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 13-18 | -75.56 seizures per 4 weeks | Standard Deviation 206.406 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 19-24 | -110.12 seizures per 4 weeks | Standard Deviation 233.492 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 25-30 | -135.60 seizures per 4 weeks | Standard Deviation 249.871 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in Overall Seizure Frequency Per 4 Weeks | Change at Month 31-36 | -51.88 seizures per 4 weeks | Standard Deviation 101.378 |
Comparison: Month 0-3p-value: <0.000195% CI: [-105, -60.46]generalized estimating equation (GEE)
Comparison: Month 4-6p-value: 0.000695% CI: [-86.46, -23.36]GEE model
Comparison: Month 7-9p-value: 0.001995% CI: [-85.62, -19.45]GEE model
Comparison: Month 10-12p-value: <0.000195% CI: [-113.13, -52.17]GEE model
Secondary
Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score
The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.
Time frame: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
Population: Pediatric participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 0 | 0.25 T-score | Standard Deviation 16.917 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 6 | 9.56 T-score | Standard Deviation 21.522 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 12 | -2.67 T-score | Standard Deviation 9.475 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 18 | -9.35 T-score | Standard Deviation 12.702 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 24 | -8.96 T-score | Standard Deviation 20.075 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Physical Summary Score | Change at Month 30 | 7.74 T-score | Standard Deviation 2.273 |
Secondary
Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score
The SF-10 Health Survey for Children was administered to caregivers of participants aged 5-17 years. Responses are used to generate 2 component summary scores: Physical Summary Score and the Psychosocial Summary Score. The T-score based scale scores were centered so that a score of 50 corresponds to the average score in a comprehensive 2006 sample (a combination of general population and supplemental disability and chronic condition samples). Higher scores are associated with better quality of life.
Time frame: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18, Month 24, Month 30
Population: Pediatric participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 0 | 0.59 T-score | Standard Deviation 11.049 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 6 | -2.29 T-score | Standard Deviation 12.366 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 12 | -7.43 T-score | Standard Deviation 15.514 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 18 | -6.60 T-score | Standard Deviation 14.987 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 24 | -8.25 T-score | Standard Deviation 15.316 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-10 Health Survey for Children Psychosocial Summary Score | Change at Month 30 | 8.91 T-score | Standard Deviation 12.599 |
Secondary
Change From Baseline Over Time in SF-12v2 Health Survey MCS Score
SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.
Time frame: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18
Population: Adult participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Change at Month 0 | 8.63 T-score | Standard Deviation 2.249 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Change at Month 6 | 5.84 T-score | Standard Deviation 3.316 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Change at Month 12 | 6.90 T-score | Standard Deviation 5.706 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey MCS Score | Change at Month 18 | 16.96 T-score | — |
Secondary
Change From Baseline Over Time in SF-12v2 Health Survey PCS Score
SF-12v2 was assessed for adults 18 years of age and older. Eight domain scores were used to generate 2 component summary scores: physical health (PCS) and mental health (MCS). The PCS and MCS scores have mean of 50 and SD of 10. The T-score based scoring method scores the data in relation to U.S. general population T-scores. Therefore, all scores obtained that are below 50 can be interpreted as below the U.S. general population T-score and scores above 50 can be interpreted as above the U.S. general population T-score. Higher global scores are associated with better quality of life.
Time frame: Baseline (from NCT01993186), Month 0, Month 6, Month 12, Month 18
Population: Adult participants with a baseline and postbaseline assessment at given time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Change at Month 0 | 10.25 T-score | Standard Deviation 5.077 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Change at Month 6 | 12.37 T-score | Standard Deviation 5.367 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Change at Month 12 | 5.09 T-score | Standard Deviation 1.619 |
| UX007 (Triheptanoin) | Change From Baseline Over Time in SF-12v2 Health Survey PCS Score | Change at Month 18 | -0.35 T-score | — |