Fabry Disease
Conditions
Keywords
Lysosomal storage disease, migalastat, AT1001, Galafold
Brief summary
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
Detailed description
This was a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, PK, PD, and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age and weighing ≥ 45 kilograms (99 pounds) with Fabry disease and amenable GLA variants. Participants must have been naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days at the time of screening. Stage 1 was a treatment period of approximately 1 month (4 weeks); Stage 2 was a treatment period of 11 months and a 30-day (untreated) safety follow-up period. There was no break in treatment between Stages 1 and 2. Prior to Stage 1, there was a screening period lasting at least 14 days and up to 30 days (or more, if GLA genotyping was required). Stages 1 and 2 together consisted of a 12-month treatment period, and a 30-day safety follow-up period, for a total of approximately 13 months. Upon study completion, participants had the option to enroll in a long-term extension study conducted under a separate protocol (NCT04049760). Participants were randomly assigned 1:1:1 to 1 of 3 PK sampling groups using interactive response technology (IRT). Four blood samples for the determination of migalastat concentrations in plasma were collected during Stage 1 study drug administration, and 1 PK (trough) sample was collected at Month 6 and again at Month 12.
Interventions
migalastat HCl 150 mg capsule
Sponsors
Amicus Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria * Willing and able to provide written consent or assent (participant and parent/legal guardian, as applicable) * Male or female between 12 and \<18 years of age diagnosed with Fabry disease * Confirmed, amenable GLA variant * Participant weighed at least 45 kg (99 pounds) at screening * Participant had never been treated with ERT or had not received ERT for 14 days prior to screening * Participant had at least 1 complication (such as, laboratory abnormality and/or sign/symptom) of Fabry disease * Participant was able to swallow study medication whole Key
Exclusion criteria
* Had moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) \<60 milliliter/minute/1.73 meter squared (m\^2) at screening) * Had advanced kidney disease requiring dialysis or kidney transplantation * History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol) * Had received any gene therapy at any time or anticipated starting gene therapy during the study period * Required treatment with Glyset (miglitol) and/or Zavesca (miglustat) within 6 months before screening or throughout the study * Required treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study * Participant was treated or had been treated with any investigational/experimental drug, biologic or device within 30 days before screening * Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may have precluded the participant from fulfilling the protocol requirements or suggested to the investigator that the potential participant may have had an unacceptable risk by participating in this study * Pregnant or breast-feeding or planned to become pregnant during the study period * Otherwise unsuitable for the study in the opinion of the investigator
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) | Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose) | TEAEs included adverse events that began on or after the first dose of study drug until 30 days after the last dose. Treatment-related TEAEs were defined as TEAEs that had an investigator-defined relationship to study drug of Definite, Probable, or Possible. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. |
| Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat | 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 | PK sampling was performed on 1 occasion at steady-state during the first month of the study. Adolescent participants were randomized to 3 sparse PK sampling groups based on optimal sampling theory. Each blood sample could have been taken at any time within the following time point ranges. Time point ranges for all 3 sparse sampling groups are arranged here in chronological order: 1 to 1.25 hours (h), 1.5 to 2h, 2.75 to 3.25h, 3.25 to 3.75h, 3.75 to 4.25h, 5 to 5.5h, 5.25 to 5.75h, 6.5 to 7h, 8.25 to 8.75h, 8.75 to 9.25h, and 10.75 to 11.25h post-dose. The combined sampling scheme resulted in rich data over an approximate 12-hour time course for AUC estimation. Additional trough samples were taken at Months 6 and 12. These trough samples (48h) assisted in estimating the terminal elimination phase for the AUC. Simulations were conducted to predict steady-state PK profiles and parameters for adolescent age subgroups 12 to \<16 years, 16 to \<18 years, and overall, 12 to \<18 years. |
| PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat | 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 | PK sampling was performed on 1 occasion at steady-state during the first month of the study. Adolescent participants were randomized to 3 sparse PK sampling groups based on optimal sampling theory. Each blood sample could have been taken at any time within the following time point ranges. Time point ranges for all 3 sparse sampling groups are arranged here in chronological order: 1 to 1.25h, 1.5 to 2h, 2.75 to 3.25h, 3.25 to 3.75h, 3.75 to 4.25h, 5 to 5.5h, 5.25 to 5.75h, 6.5 to 7h, 8.25 to 8.75h, 8.75 to 9.25h, and 10.75 to 11.25h post-dose. The combined sampling scheme resulted in rich data over an approximate 12-hour time course for AUC and Cmax estimation. Additional trough samples were taken at Months 6 and 12. These trough samples (48h) assisted in estimating the terminal elimination phase for the AUC. Simulations were conducted to predict steady-state PK profiles and parameters for adolescent age subgroups 12 to \<16 years, 16 to \<18 years, and overall, 12 to \<18 years. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline In Left Ventricular Mass Index (LVMi) | Baseline, Month 12 and last observation (up to Month 12) | LVMi was assessed as a measure of cardiac impairment in the study participants. LVMi values for both M Mode and 2D views are presented. |
| Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) | Baseline, Month 12 and last observation (up to Month 12) | Blood samples were collected for measurement of lyso-Gb3 levels in plasma. Plasma levels of lyso-Gb3 were measured using a validated liquid chromatography-mass spectrometry assay. |
| FABPRO-GI And Pain Scores | Month 12 and last observation (up to Month 12) | The Fabry Disease Patient-Reported Outcome - Gastrointestinal Signs And Symptoms (FABPRO-GI) And Pain Questionnaire For Clinical Trials (24-hr Version) consists of questions regarding gastrointestinal signs and symptoms and pain relative to the past 24 hours. Participants rated the severity of their symptoms and pain from 0 (none) to 10 (worst possible). The monthly average score at Month 12 and at last observation are presented. A higher score indicated higher levels of symptoms and pain. |
| Change In eGFR From Baseline To Month 12 | Baseline, Month 12 and last observation (up to Month 12) | eGFR was calculated using the modified Schwartz formula for creatinine clearance. |
| Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) | Month 12 | The assessment of In the last 3 months how many times did you experience sudden onset of pain? using the FPHPQ for ages 13 to 18 is presented. |
| Change From Baseline In FPHPQ Score For Pain Intensity | Baseline, Month 12 and last observation (up to Month 12) | The assessment of How bad is your pain today? using the FPHPQ for ages 13 to 18 is presented. Pain intensity was measured on a 10-point scale, 0 (no pain) to 10 (pain as bad as you can imagine). A decrease from baseline indicates an improvement in the condition. |
| Change From Baseline In Pediatric Quality Of Life (PedsQL) At Month 12 | Baseline, Month 12 | The PedsQL is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The psychosocial score for the PedsQL encompassed 15 questions relating to the participants' feelings, social interaction with others, and school. The physical score was derived from answers to 8 questions about the participants' ease of managing physical activity. All components of the PedsQL were scored based on a scale of 0 (never) to 4 (almost always) and linearly transformed to a 0-100 scale as follows: 0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0. Both categories were combined for a total score. Total scores for the child report ages 13 to 18 years old and parent report are presented at Month 12. |
| Patient's Global Impression Of Change (PGI-C) Scores | Month 12 | The PGI-C consists of 4 questions regarding diarrhea, abdominal pain, overall pain, and daily living. Participants rated their status based on improvement, worsening, or the same. Improved status includes Much better, Better and A little better; worsened status includes A little worse, Worse and Much worse. |
| Annualized Rate Of Change From Baseline | Baseline up to Month 12 | Annualized rate of change from baseline of eGFR was defined as change from baseline to last visit divided by the duration from baseline to the last visit (Last assessment date - First dose date +1) and multiplied by 365.25. Baseline was defined as the last non-missing assessment prior to the first dose of study drug. |
| Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 | Baseline, Month 12 and last observation (up to Month 12) | Renal function was assessed by urine protein and urine albumin levels. Urine samples were collected as part of urinalysis to measure protein and albumin levels. |
Countries
United Kingdom, United States
Participant flow
Recruitment details
Participants must have been either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days at the time of screening.
Participants by arm
| Arm | Count |
|---|---|
| Migalastat HCl 150 mg Migalastat was administered every other day for 12 months. | 22 |
| Total | 22 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Withdrawal by Parent or Legally-authorized Representative | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Migalastat HCl 150 mg |
|---|---|
| Age, Continuous | 14.6 years STANDARD_DEVIATION 1.62 |
| Race/Ethnicity, Customized Hispanic or Latino | 3 participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 19 participants |
| Race/Ethnicity, Customized Other | 2 participants |
| Race/Ethnicity, Customized White | 20 participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 21 |
| other Total, other adverse events | 20 / 21 |
| serious Total, serious adverse events | 1 / 21 |
Outcome results
Primary
Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs)
TEAEs included adverse events that began on or after the first dose of study drug until 30 days after the last dose. Treatment-related TEAEs were defined as TEAEs that had an investigator-defined relationship to study drug of Definite, Probable, or Possible. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Time frame: Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
Population: Participants who received at least 1 dose of study drug.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Migalastat HCl 150 mg | Number Of Participants Who Experienced Treatment-related Treatment-emergent Adverse Events (TEAEs) | 5 Participants |
Primary
Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat
PK sampling was performed on 1 occasion at steady-state during the first month of the study. Adolescent participants were randomized to 3 sparse PK sampling groups based on optimal sampling theory. Each blood sample could have been taken at any time within the following time point ranges. Time point ranges for all 3 sparse sampling groups are arranged here in chronological order: 1 to 1.25 hours (h), 1.5 to 2h, 2.75 to 3.25h, 3.25 to 3.75h, 3.75 to 4.25h, 5 to 5.5h, 5.25 to 5.75h, 6.5 to 7h, 8.25 to 8.75h, 8.75 to 9.25h, and 10.75 to 11.25h post-dose. The combined sampling scheme resulted in rich data over an approximate 12-hour time course for AUC estimation. Additional trough samples were taken at Months 6 and 12. These trough samples (48h) assisted in estimating the terminal elimination phase for the AUC. Simulations were conducted to predict steady-state PK profiles and parameters for adolescent age subgroups 12 to \<16 years, 16 to \<18 years, and overall, 12 to \<18 years.
Time frame: 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Population: Participants with at least 1 quantifiable concentration and a known weight and estimated glomerular filtration rate (eGFR). A population PK model was used for assessment. All migalastat concentration-time data were combined and included in a population PK analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat | 12 to <16 years old | 8920 h*ng/mL | Geometric Coefficient of Variation 47.2 |
| Migalastat HCl 150 mg | Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat | 16 to <18 years old | 8430 h*ng/mL | Geometric Coefficient of Variation 41.7 |
| Migalastat HCl 150 mg | Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) Of Migalastat | 12 to <18 years old | 8740 h*ng/mL | Geometric Coefficient of Variation 44.2 |
Primary
PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat
PK sampling was performed on 1 occasion at steady-state during the first month of the study. Adolescent participants were randomized to 3 sparse PK sampling groups based on optimal sampling theory. Each blood sample could have been taken at any time within the following time point ranges. Time point ranges for all 3 sparse sampling groups are arranged here in chronological order: 1 to 1.25h, 1.5 to 2h, 2.75 to 3.25h, 3.25 to 3.75h, 3.75 to 4.25h, 5 to 5.5h, 5.25 to 5.75h, 6.5 to 7h, 8.25 to 8.75h, 8.75 to 9.25h, and 10.75 to 11.25h post-dose. The combined sampling scheme resulted in rich data over an approximate 12-hour time course for AUC and Cmax estimation. Additional trough samples were taken at Months 6 and 12. These trough samples (48h) assisted in estimating the terminal elimination phase for the AUC. Simulations were conducted to predict steady-state PK profiles and parameters for adolescent age subgroups 12 to \<16 years, 16 to \<18 years, and overall, 12 to \<18 years.
Time frame: 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Population: Participants with at least 1 quantifiable concentration and a known weight and eGFR. A population PK model was used for assessment. All migalastat concentration-time data were combined and included in a population PK analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat | 12 to <16 years old | 1220 ng/mL | Geometric Coefficient of Variation 60.9 |
| Migalastat HCl 150 mg | PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat | 16 to <18 years old | 1160 ng/mL | Geometric Coefficient of Variation 39.2 |
| Migalastat HCl 150 mg | PK: Maximum Observed Plasma Concentration (Cmax) Of Migalastat | 12 to <18 years old | 1200 ng/mL | Geometric Coefficient of Variation 52.7 |
Secondary
Annualized Rate Of Change From Baseline
Annualized rate of change from baseline of eGFR was defined as change from baseline to last visit divided by the duration from baseline to the last visit (Last assessment date - First dose date +1) and multiplied by 365.25. Baseline was defined as the last non-missing assessment prior to the first dose of study drug.
Time frame: Baseline up to Month 12
Population: All participants who received study drug and had an assessment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Migalastat HCl 150 mg | Annualized Rate Of Change From Baseline | -1.5 mL/min x 1.73 m^2/year | Standard Deviation 15.11 |
Secondary
Change From Baseline In FPHPQ Score For Pain Intensity
The assessment of How bad is your pain today? using the FPHPQ for ages 13 to 18 is presented. Pain intensity was measured on a 10-point scale, 0 (no pain) to 10 (pain as bad as you can imagine). A decrease from baseline indicates an improvement in the condition.
Time frame: Baseline, Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change From Baseline In FPHPQ Score For Pain Intensity | Month 12 | 0.4 units on a scale | Standard Deviation 1.82 |
| Migalastat HCl 150 mg | Change From Baseline In FPHPQ Score For Pain Intensity | Last observation (up to Month 12) | 0.4 units on a scale | Standard Deviation 1.77 |
Secondary
Change From Baseline In Left Ventricular Mass Index (LVMi)
LVMi was assessed as a measure of cardiac impairment in the study participants. LVMi values for both M Mode and 2D views are presented.
Time frame: Baseline, Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change From Baseline In Left Ventricular Mass Index (LVMi) | 2D: Month 12 | 4.9 g/m^2 | Standard Deviation 9.12 |
| Migalastat HCl 150 mg | Change From Baseline In Left Ventricular Mass Index (LVMi) | M Mode: Month 12 | -3.9 g/m^2 | Standard Deviation 13.53 |
| Migalastat HCl 150 mg | Change From Baseline In Left Ventricular Mass Index (LVMi) | M Mode: Last observation (up to Month 12) | -4.4 g/m^2 | Standard Deviation 13.31 |
| Migalastat HCl 150 mg | Change From Baseline In Left Ventricular Mass Index (LVMi) | 2D: Last observation (up to Month 12) | 4.3 g/m^2 | Standard Deviation 9.34 |
Secondary
Change From Baseline In Pediatric Quality Of Life (PedsQL) At Month 12
The PedsQL is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The psychosocial score for the PedsQL encompassed 15 questions relating to the participants' feelings, social interaction with others, and school. The physical score was derived from answers to 8 questions about the participants' ease of managing physical activity. All components of the PedsQL were scored based on a scale of 0 (never) to 4 (almost always) and linearly transformed to a 0-100 scale as follows: 0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0. Both categories were combined for a total score. Total scores for the child report ages 13 to 18 years old and parent report are presented at Month 12.
Time frame: Baseline, Month 12
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change From Baseline In Pediatric Quality Of Life (PedsQL) At Month 12 | Child Report | 2.2 units on a scale | Standard Deviation 6.13 |
| Migalastat HCl 150 mg | Change From Baseline In Pediatric Quality Of Life (PedsQL) At Month 12 | Parent Report | 3.1 units on a scale | Standard Deviation 10.29 |
Secondary
Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12
Renal function was assessed by urine protein and urine albumin levels. Urine samples were collected as part of urinalysis to measure protein and albumin levels.
Time frame: Baseline, Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 | Urine Albumin: Month 12 | 16.2 mg/L | Standard Deviation 28.27 |
| Migalastat HCl 150 mg | Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 | Urine Albumin: Last observation (up to Month 12) | 15.6 mg/L | Standard Deviation 27.67 |
| Migalastat HCl 150 mg | Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 | Total Urine Protein: Month 12 | 36.0 mg/L | Standard Deviation 111.61 |
| Migalastat HCl 150 mg | Change From Baseline In Total Urine Protein And Urine Albumin Levels At Month 12 | Total Urine Protein: Last observation (up to Month 12) | 36.2 mg/L | Standard Deviation 108.64 |
Secondary
Change In eGFR From Baseline To Month 12
eGFR was calculated using the modified Schwartz formula for creatinine clearance.
Time frame: Baseline, Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change In eGFR From Baseline To Month 12 | Month 12 | -1.6 mL/min x 1.73 m^2 | Standard Deviation 15.4 |
| Migalastat HCl 150 mg | Change In eGFR From Baseline To Month 12 | Last observation (up to Month 12) | -1.6 mL/min x 1.73 m^2 | Standard Deviation 14.99 |
Secondary
Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3)
Blood samples were collected for measurement of lyso-Gb3 levels in plasma. Plasma levels of lyso-Gb3 were measured using a validated liquid chromatography-mass spectrometry assay.
Time frame: Baseline, Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) | Month 12 | -14.0 ng/mL | Standard Deviation 23.13 |
| Migalastat HCl 150 mg | Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) | Last Observation (up to Month 12) | -14.0 ng/mL | Standard Deviation 23.13 |
| Migalastat HCl 150 mg: ERT Experienced | Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) | Month 12 | 12.5 ng/mL | Standard Deviation 36.33 |
| Migalastat HCl 150 mg: ERT Experienced | Change In Plasma Levels Of Globotriaosylsphingosine (Lyso-Gb3) | Last Observation (up to Month 12) | 11.3 ng/mL | Standard Deviation 34.67 |
Secondary
FABPRO-GI And Pain Scores
The Fabry Disease Patient-Reported Outcome - Gastrointestinal Signs And Symptoms (FABPRO-GI) And Pain Questionnaire For Clinical Trials (24-hr Version) consists of questions regarding gastrointestinal signs and symptoms and pain relative to the past 24 hours. Participants rated the severity of their symptoms and pain from 0 (none) to 10 (worst possible). The monthly average score at Month 12 and at last observation are presented. A higher score indicated higher levels of symptoms and pain.
Time frame: Month 12 and last observation (up to Month 12)
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Daily Ratings of Severity in Constipation: Month 12 | 0.9 units on a scale | Standard Deviation 1.91 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Daily Ratings of Severity in Constipation: Last observation (up to Month 12) | 0.4 units on a scale | Standard Deviation 1 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Daily Ratings of Severity in Diarrhea: Month 12 | 1.0 units on a scale | Standard Deviation 1.65 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Daily Ratings of Severity in Diarrhea: Last Observation (up to Month 12) | 0.4 units on a scale | Standard Deviation 0.91 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Overall Pain: Month 12 | 0.6 units on a scale | Standard Deviation 0.71 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Overall Pain: Last Observation (up to Month 12) | 1.2 units on a scale | Standard Deviation 1.5 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Worst Tummy Pain: Month 12 | 0.3 units on a scale | Standard Deviation 0.38 |
| Migalastat HCl 150 mg | FABPRO-GI And Pain Scores | Worst Tummy Pain: Last Observation (up to Month 12) | 0.9 units on a scale | Standard Deviation 1.52 |
Secondary
Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ)
The assessment of In the last 3 months how many times did you experience sudden onset of pain? using the FPHPQ for ages 13 to 18 is presented.
Time frame: Month 12
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Migalastat HCl 150 mg | Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) | 0 times | 4 Participants |
| Migalastat HCl 150 mg | Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) | 1-3 times | 5 Participants |
| Migalastat HCl 150 mg | Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) | 4-6 times | 4 Participants |
| Migalastat HCl 150 mg | Number Of Participants Who Experienced Sudden Onset Of Pain As Assessed Using The Fabry-Specific Pediatric Health And Pain Questionnaire (FPHPQ) | > 6 times | 3 Participants |
Secondary
Patient's Global Impression Of Change (PGI-C) Scores
The PGI-C consists of 4 questions regarding diarrhea, abdominal pain, overall pain, and daily living. Participants rated their status based on improvement, worsening, or the same. Improved status includes Much better, Better and A little better; worsened status includes A little worse, Worse and Much worse.
Time frame: Month 12
Population: Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Diarrhea: Status Improved | 12 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Diarrhea: Status Same | 7 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Diarrhea: Status Worse | 0 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Abdominal pain: Status Improved | 10 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Abdominal pain: Status Same | 8 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Abdominal pain: Status Worse | 1 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Overall pain: Status Improved | 10 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Overall pain: Status Same | 8 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Overall pain: Status Worse | 1 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Daily living: Status Improved | 10 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Daily living: Status Same | 8 Participants |
| Migalastat HCl 150 mg | Patient's Global Impression Of Change (PGI-C) Scores | Daily living: Status Worse | 1 Participants |