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| ID | Type | Description | Link |
|---|---|---|---|
| FACETS | Other Identifier | Amicus Therapeutics | |
| 2009-013459-31 | EudraCT Number |
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The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
This double-blind, randomized, placebo-controlled study was conducted in 67 participants at 46 sites worldwide. The study consisted of 2 stages and an optional open-label treatment extension phase:
Stage 1 included a screening period of up to 2 months followed by a 6-month treatment period which involved 4 visits to the clinic. Participants were randomized in equal proportions to receive either migalastat or placebo.
After completing the 6-month double-blind phase, all participants entered Stage 2 of the study and received migalastat in an open-label manner. Stage 2 treatment lasted for 6 months and involved up to 4 visits to the clinic.
Participants who completed both Stage 1 and Stage 2 of the study as scheduled were offered the opportunity to participate in an open-label treatment extension phase with migalastat. The open-label treatment extension phase lasted 12 months and involved 2 visits to the clinic. A follow-up visit was undertaken 1 month following completion or discontinuation from the open-label treatment extension. Participants completing the 12-month open-label treatment extension and providing consent to enter a separate long-term extension were not required to complete this follow-up visit.
Study assessments included clinical laboratory tests, 12-lead electrocardiogram, kidney biopsy, kidney function testing, echocardiography, and patient-reported outcomes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Migalastat | Experimental | Migalastat 150-mg capsule taken orally every other day (QOD) for 6 months and an open-label 6-month treatment extension, followed by an optional, 12-month, open-label treatment extension. |
|
| Placebo | Placebo Comparator | Placebo capsule taken orally QOD for 6 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| migalastat hydrochloride | Drug | Oral capsule QOD |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. A responder was defined as a participant with a ≥50% reduction from Baseline to Month 6 in the average number of kidney IC GL-3 inclusions. | Baseline, Month 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 Inclusions | Renal biopsies were taken at Month 6 and Month 12. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Month 6 and Month 12. Assessments were made using digital images. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor, Clinical Research | Amicus Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Los Angeles | California | 90048 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32994552 | Derived | Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease. Genet Med. 2021 Jan;23(1):192-201. doi: 10.1038/s41436-020-00968-z. Epub 2020 Sep 30. | |
| 30723321 | Derived |
Not provided
Not provided
During Stage 1 (0-6 Months), all participants were randomized to either Migalastat or Placebo. After Stage 1, all participants progressed to Stage 2 where they received open-label migalastat for 6 months (>6-12 Months). After Stage 2, participants were eligible to enter the optional, 12-month, open-label extension (OLE) (>12-24 Months).
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Migalastat (0-6 Months) | Migalastat hydrochloride (migalastat) 150-milligram (mg) capsule (equivalent to 123 mg of migalastat) given orally every other day (QOD) during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| 6-Month Double-blind (Stage 1) |
|
Not provided
Not provided
Not provided
Sponsor and Assessor were also blinded
| Placebo | Drug | Oral capsule QOD |
|
| Baseline, Month 6 |
| Change From Baseline Through Month 24 In Urine GL-3 Levels | The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages. | Baseline, Months 6, 12, and 24 |
| Month 6, Month 12 |
| San Francisco |
| California |
| 94143 |
| United States |
| Decatur | Georgia | 30033 | United States |
| Chicago | Illinois | 60611 | United States |
| Kansas City | Kansas | 66160 | United States |
| Boston | Massachusetts | 02114 | United States |
| Grand Rapids | Michigan | 49525 | United States |
| New York | New York | 10032 | United States |
| Pittsburgh | Pennsylvania | 15224 | United States |
| Dallas | Texas | 75226 | United States |
| Salt Lake City | Utah | 84132 | United States |
| Springfield | Virginia | 22152 | United States |
| Seattle | Washington | 98195 | United States |
| Buenos Aires | B1629ODT | Argentina |
| Adelaide | 5006 | Australia |
| Parkville | 3050 | Australia |
| Porto Alegre | 90035-903 | Brazil |
| São Paulo | 14048-900 | Brazil |
| Montreal | Quebec | H4J 1C5 | Canada |
| Copenhagen | DK-2100 | Denmark |
| Cairo | 11451 | Egypt |
| Garches | 92380 | France |
| Roma | 00168 | Italy |
| Warsaw | 04-628 | Poland |
| Barcelona | 08025 | Spain |
| Zaragoza | 50009 | Spain |
| Ankara | 06500 | Turkey (Türkiye) |
| Salford | M6 8HD | United Kingdom |
| Germain DP, Nicholls K, Giugliani R, Bichet DG, Hughes DA, Barisoni LM, Colvin RB, Jennette JC, Skuban N, Castelli JP, Benjamin E, Barth JA, Viereck C. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study. Genet Med. 2019 Sep;21(9):1987-1997. doi: 10.1038/s41436-019-0451-z. Epub 2019 Feb 6. |
| 29703262 | Derived | Schiffmann R, Bichet DG, Jovanovic A, Hughes DA, Giugliani R, Feldt-Rasmussen U, Shankar SP, Barisoni L, Colvin RB, Jennette JC, Holdbrook F, Mulberg A, Castelli JP, Skuban N, Barth JA, Nicholls K. Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial. Orphanet J Rare Dis. 2018 Apr 27;13(1):68. doi: 10.1186/s13023-018-0813-7. |
| 27657681 | Derived | Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22. |
| 27509102 | Derived | Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198. |
| FG001 |
| Placebo (0-6 Months) |
Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. |
| FG002 | Migalastat (>6-12 Months) | Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2). Includes all participants who progressed from both the migalastat (0-6 Months) and the placebo (0-6 Months) Stage 1 treatment groups. After Stage 2, participants were eligible to enter the optional, 12-month OLE. |
| FG003 | Migalastat (>12-24 Months) | Migalastat 150-mg capsule given orally QOD for up to 12 months during the optional OLE. Includes all participants who decided to continue treatment after completing the 6-month, open-label Stage 2 treatment period. |
| Received at Least 1 Dose of Study Drug |
|
| Intent-to-Treat (ITT) | All randomized participants regardless of their participation in the study beyond Randomization. |
|
| Modified Intent-to-Treat (mITT) | Participants with Baseline and Month 6 biopsy results. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| 6-Month Open-label (Stage 2) |
|
|
| 12-Month Open-label Extension (Optional) |
|
|
Safety Population: All randomized participants who received at least 1 dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Migalastat-Migalastat | Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1), during the 6-month open-label treatment period (Stage 2), and for up to 12 months during the OLE. |
| BG001 | Placebo-Migalastat | Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2) and for up to 12 months during the OLE. |
| BG002 | Total | Total of all reporting groups |
| 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 |
|
| 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 | Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions | Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. A responder was defined as a participant with a ≥50% reduction from Baseline to Month 6 in the average number of kidney IC GL-3 inclusions. | ITT: All randomized participants regardless of their participation in the study beyond randomization. As per the statistical analysis plan, analysis excluded participants who were missing baseline kidney biopsy results. | Posted | Count of Participants | Participants | Baseline, Month 6 |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 | Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. | ITT: All randomized participants regardless of their participation in the study beyond randomization. | Posted | Mean | Standard Deviation | percent change | Baseline, Month 6 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline Through Month 24 In Urine GL-3 Levels | The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages. | Stage 1: ITT, all randomized participants regardless of participation in the study beyond randomization. Stage 2: participants who completed Stage 1 and entered Stage 2. OLE: participants who completed Stage 2 and entered the optional OLE. Once assay and sample issues were identified, later samples were not further analyzed; all data was listed. | Posted | Mean | Standard Deviation | ng/mg creatinine | Baseline, Months 6, 12, and 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Other Pre-specified | Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 Inclusions | Renal biopsies were taken at Month 6 and Month 12. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Month 6 and Month 12. Assessments were made using digital images. | mITT with amenable mutations: Randomized participants who switched from placebo to migalastat during Stage 2, received at least 1 dose of study drug, and underwent a renal biopsy at both Baseline and Month 6. Amenable mutations are mutant forms of α-galactosidase A (α Gal-A) amenable to migalastat. Amenable mutations based on the GLP HEK assay. | Posted | Least Squares Mean | 95% Confidence Interval | kidney IC GL-3 inclusions | Month 6, Month 12 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Post-Hoc | Change From Baseline To Month 6 In Average Number Of Kidney IC GL-3 Inclusions | Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. Treatment effect was estimated using the LS mean difference between treatments within the context of the ANCOVA model. | ITT-amenable: Randomized participants with amenable mutations who received study drug during Stage 1. Amenable mutations are mutant forms of α Gal-A amenable to migalastat. Amenable mutations based on the GLP HEK assay. Participants were analyzed according to their original randomized treatment group. | Posted | Mean | Standard Deviation | kidney IC GL-3 inclusions | Baseline, Month 6 |
|
Adverse events (AEs) were monitored up to 25 months (±7 days) after the first dose of study drug. AEs were reported from the first dose of study medication at Baseline (Visit 1) in Stage 1 to 1 month (±7 days) after the date of the last study visit, whether during Stage 1, Stage 2, or the optional OLE, or after premature discontinuation from the study.
Stage 1 AEs were any AEs that started between the first dose of Stage 1 and the first dose of Stage 2 (or last Stage 1 administration for participants that discontinued during Stage 1). Stage 2 AEs were any AEs that started between the first dose of Stage 2 and the first dose of the OLE (or the last Stage 2 administration for participants that discontinued during Stage 2). OLE AEs were any AEs that started after the first dose of the OLE.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Migalastat (0-6 Months) | Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. | 2 | 34 | 31 | 34 | ||
| EG001 | Placebo (0-6 Months) | Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. | 4 | 33 | 30 | 33 | ||
| EG002 | All Migalastat (>6-12 Months) | Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2). Includes all participants who progressed from both the migalastat (0-6 Months) and the placebo (0-6 Months) Stage 1 treatment groups. After Stage 2, participants were eligible to enter the optional, 12-month OLE. | 5 | 63 | 50 | 63 | ||
| EG003 | All Migalastat (>12-24 Months) | Migalastat 150-mg capsule given orally QOD for up to 12 months during the optional OLE. Includes all participants who decided to continue treatment after completing the 6-month, open-label Stage 2 treatment period. | 11 | 57 | 46 | 57 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Deep Vein Thrombosis | Vascular disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Multiple Fractures | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Post Procedural Haematoma | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Post Procedural Haemorrhage | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Anaplastic Large Cell Lymphoma T - and Null - Cell Types | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Ventricular Tachycardia | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Amyotrophic Lateral Sclerosis | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Cerebral Haemorrhage | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Transient Ischaemic Attack | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Bulimia Nervosa | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal Pain Lower | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Bone Cyst | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Bacterial Infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Helicobacter Gastritis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Meningitis Viral | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Overdose | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Procedural Pain | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Atrial Fibrillation | Cardiac disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Abdominal Distension | Gastrointestinal disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 15.0 | Systematic Assessment |
| |
| Post Procedural Complication | Injury, poisoning and procedural complications | MedDRA 15.0 | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA 15.0 | Systematic Assessment |
| |
| Torticollis | Musculoskeletal and connective tissue disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Vitamin D Deficiency | Metabolism and nutrition disorders | MedDRA 15.0 | Systematic Assessment |
| |
| Microalbuminuria | Renal and urinary disorders | MedDRA 15.0 | Systematic Assessment |
|
During Phase 3, prior to unblinding, the assay used for enrollment was validated (GLP HEK assay). Mutant forms of α-Gal A that met assay criteria were categorized as amenable; additional analyses were subsequently performed on this target population.
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 for 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 |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| 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 |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| C090092 | migalastat |
| C525167 | larazotide acetate |
Not provided
Not provided
Not provided
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