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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-000146-21 | EudraCT Number |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| migalastat HCl 150 mg | Experimental | One migalastat 123 milligrams (mg) capsule equivalent to 150 mg migalastat hydrochloride (HCl) (herein referred to as "migalastat") was administered every other day for 12 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Migalastat HCl 150 mg | Drug | migalastat HCl 150 mg capsule |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose) |
| 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. | 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 |
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change In eGFR From Baseline To Month 12 | eGFR was calculated using the modified Schwartz formula for creatinine clearance. | Baseline, Month 12 and last observation (up to Month 12) |
| Annualized Rate Of Change From Baseline |
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Key Inclusion Criteria
Key Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor Clinical Research | Amicus Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Site | Tampa | Florida | 33606 | United States | ||
| Clinical Study Site |
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Participants must have been either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days at the time of screening.
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| ID | Title | Description |
|---|---|---|
| FG000 | Migalastat HCl 150 mg | Migalastat was administered every other day for 12 months. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 13, 2019 | Jul 16, 2021 |
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| 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 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.
| Baseline up to Month 12 |
| 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. | Baseline, Month 12 and last observation (up to Month 12) |
| 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. | Baseline, Month 12 and last observation (up to Month 12) |
| 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. | Baseline, Month 12 and last observation (up to Month 12) |
| 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. | Month 12 and last observation (up to Month 12) |
| 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". | Month 12 |
| 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. | Month 12 |
| 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. | Baseline, Month 12 and last observation (up to Month 12) |
| 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. | Baseline, Month 12 |
| Atlanta |
| Georgia |
| 30322 |
| United States |
| Clinical Study Site | Minneapolis | Minnesota | 55454 | United States |
| Clinical Study Site | Columbia | Missouri | 65212 | United States |
| Clinical Study Site | Cincinnati | Ohio | 45229 | United States |
| Clinical Study Site | Pittsburgh | Pennsylvania | 15224 | United States |
| Clinical Study Site | Fairfax | Virginia | 22030 | United States |
| Clinical Study Site | London | NW3 2QG | United Kingdom |
| Received At Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
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|
All participants who enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Migalastat HCl 150 mg | Migalastat was administered every other day for 12 months. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose) |
|
|
| ||||||||||||||||||||||||||
| 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. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | h*ng/mL | 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 |
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| 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. | 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. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12 |
| |||||||||||||||||||||||||||
| Secondary | Change In eGFR From Baseline To Month 12 | eGFR was calculated using the modified Schwartz formula for creatinine clearance. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints. | Posted | Mean | Standard Deviation | mL/min x 1.73 m^2 | Baseline, Month 12 and last observation (up to Month 12) |
|
| ||||||||||||||||||||||||||
| 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. | All participants who received study drug and had an assessment | Posted | Mean | Standard Deviation | mL/min x 1.73 m^2/year | Baseline up to Month 12 |
|
| ||||||||||||||||||||||||||
| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints. | Posted | Mean | Standard Deviation | mg/L | Baseline, Month 12 and last observation (up to Month 12) |
|
| ||||||||||||||||||||||||||
| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints. | Posted | Mean | Standard Deviation | g/m^2 | Baseline, Month 12 and last observation (up to Month 12) |
|
| ||||||||||||||||||||||||||
| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints. | Posted | Mean | Standard Deviation | ng/mL | Baseline, Month 12 and last observation (up to Month 12) |
|
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| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint. | Posted | Mean | Standard Deviation | units on a scale | Month 12 and last observation (up to Month 12) |
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| 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". | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint. | Posted | Count of Participants | Participants | Month 12 |
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| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint. | Posted | Count of Participants | Participants | Month 12 |
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| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoint. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Month 12 and last observation (up to Month 12) |
|
| ||||||||||||||||||||||||||
| 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. | Participants who received at least 1 dose of study drug and had evaluable data at the specified timepoints. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Month 12 |
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Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Migalastat HCl 150 mg | Migalastat was administered every other day for 12 months. | 0 | 21 | 1 | 21 | 20 | 21 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Suicidal ideation | Psychiatric disorders | MedDRA 21.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Complication associated with device | General disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | MedDRA 21.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Systematic Assessment |
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The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Affairs | Amicus Therapeutics | +1-877-426-4287 (877-4-AMICUS) | MedInfoUSA@amicusrx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 20, 2020 | Jul 16, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
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| ID | Term |
|---|---|
| C090092 | migalastat |
| C525167 | larazotide acetate |
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| Hispanic or Latino |
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| Not Hispanic or Latino |
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| Units | Counts |
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| Participants |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Month 12 |
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| Last observation (up to Month 12) |
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