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| ID | Type | Description | Link |
|---|---|---|---|
| ATTRACT | Other Identifier | Amicus Therapeutics | |
| 2010-022636-37 | EudraCT Number |
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Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.
This was a Phase 3, randomized, open-label, active-controlled study to evaluate the efficacy and safety of 150 milligrams (mg) of migalastat hydrochloride (migalastat) (equivalent to 123 mg of migalastat) once every other day (QOD) and ERT in male and female participants with Fabry disease who were receiving ERT and who have an α Gal-A mutation that is amenable to migalastat, based on the clinical trial HEK assay. This was a 2-part study. Part 1, the 18-month randomized phase, evaluated participants who received either migalastat 150 mg QOD or ERT per prescribing physicians' instructions for efficacy and safety. Part 2, the optional 12-month open-label extension (OLE) phase in which all participants received migalastat, also explored efficacy and safety. For Part 2, all participants who received ERT in Part 1 were given migalastat. Data presented in this posting include efficacy data from the 18-month randomized period and safety data from the entire study (18-month randomized period and 12-month optional OLE [total of 30 months]).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Migalastat | Experimental | Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period and the optional 12-month OLE period. Participants received an inactive reminder capsule on alternate days during both treatment periods. |
|
| ERT | Active Comparator | Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| migalastat hydrochloride | Drug | 150-mg capsule administered orally QOD |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of <2.2 milliliter (mL)/minute (min)/1.73 meter squared (m^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months. | Baseline to Month 18 |
| Annualized Rate Of Change From Baseline To Month 18 In eGFR | The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following: eGFR-CKD-EPI = 141 x min (Serum Creatinine/κ,1)^(α) x max(Serum Creatinine/κ,1)^(-1.209) x 0.993^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Serum Creatinine/κ or 1; max indicates the maximum of Serum Creatinine/κ or 1. The annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of <2.2 mL/min/1.73m^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months. | Baseline to Month 18 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)^(-1.154) x (Age)^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female). The eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. |
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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 |
|---|---|---|---|
| 33012654 | Derived | Feldt-Rasmussen U, Hughes D, Sunder-Plassmann G, Shankar S, Nedd K, Olivotto I, Ortiz D, Ohashi T, Hamazaki T, Skuban N, Yu J, Barth JA, Nicholls K. Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study. Mol Genet Metab. 2020 Sep-Oct;131(1-2):219-228. doi: 10.1016/j.ymgme.2020.07.007. Epub 2020 Aug 15. | |
| 32994552 |
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The All Migalastat group was comprised of participants who received migalastat during the randomized treatment period (0-18 mo.) and who received enzyme replacement therapy (ERT) during the randomized treatment period (0-18 mo.) and switched to migalastat during the 12-mo. open-label extension (OLE). 8 participants enrolled but did not randomize.
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| ID | Title | Description |
|---|---|---|
| FG000 | Migalastat (0-18 Months) | Participants received 150 milligrams (mg) of migalastat hydrochloride (migalastat) (equivalent to 123 mg of migalastat) orally once every other day (QOD) during the 18-month randomized treatment period (0-18 months). Participants received an inactive reminder capsule on alternate days during both treatment periods. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| 18-Month Randomized Period |
|
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| agalsidase | Biological | Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information |
|
|
| Baseline to Month 18 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Decatur | Georgia | 30033 | United States |
| Grand Rapids | Michigan | 49525 | United States |
| Portland | Oregon | 97239 | United States |
| Pittsburgh | Pennsylvania | 15224 | United States |
| Fairfax | Virginia | 22030 | United States |
| Milwaukee | Wisconsin | 53226 | United States |
| Parkville | Victoria | 03050 | Australia |
| Perth | Western Australia | 6000 | Australia |
| Vienna | 1090 | Austria |
| Edegem | 2650 | Belgium |
| São Paulo | 14048-900 | Brazil |
| Copenhagen | 2100 | Denmark |
| Garches | 92380 | France |
| Lille | 59037 | France |
| Florence | 50129 | Italy |
| Niigata | 951-8520 | Japan |
| Osaka | 545-8586 | Japan |
| Osaka | 565-0871 | Japan |
| Tokyo | 105-8471 | Japan |
| Cambridge | CB2 0QQ | United Kingdom |
| London | NW3 2QG | United Kingdom |
| London | WC1N 3BG | United Kingdom |
| Salford | M6 8HD | United Kingdom |
| 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. |
| 31934472 | Derived | Haninger-Vacariu N, El-Hadi S, Pauler U, Foretnik M, Kain R, Prohaszka Z, Schmidt A, Skuban N, Barth JA, Sunder-Plassmann G. Pregnancy Outcome after Exposure to Migalastat for Fabry Disease: A Clinical Report. Case Rep Obstet Gynecol. 2019 Dec 21;2019:1030259. doi: 10.1155/2019/1030259. eCollection 2019. |
| 31889231 | Derived | Narita I, Ohashi T, Sakai N, Hamazaki T, Skuban N, Castelli JP, Lagast H, Barth JA. Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study. Clin Exp Nephrol. 2020 Feb;24(2):157-166. doi: 10.1007/s10157-019-01810-w. Epub 2019 Dec 30. |
| 27834756 | Derived | Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T, Olivotto I, Sakai N, Deegan P, Dimmock D, Eyskens F, Germain DP, Goker-Alpan O, Hachulla E, Jovanovic A, Lourenco CM, Narita I, Thomas M, Wilcox WR, Bichet DG, Schiffmann R, Ludington E, Viereck C, Kirk J, Yu J, Johnson F, Boudes P, Benjamin ER, Lockhart DJ, Barlow C, Skuban N, Castelli JP, Barth J, Feldt-Rasmussen U. Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study. J Med Genet. 2017 Apr;54(4):288-296. doi: 10.1136/jmedgenet-2016-104178. Epub 2016 Nov 10. |
| 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. |
| FG001 |
| ERT (0-18 Months) |
Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period (0-18 months). Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. |
| FG002 | All Migalastat (0-30 Months) | Participants in this group are composed of participants from the Migalastat (0-18 months) and the ERT (0-18 months) groups. Therefore, participants in the All Migalastat group received at least 1 dose of 150 mg migalastat orally QOD during the 18-month randomization period or the optional 12-month OLE (for a total time period of up to 30 months), regardless of whether or not the participant discontinued the study early, and regardless of whether they were previously randomized to migalastat or ERT during the 18-month Randomized Period (0-18 months). |
| Safety Population | Safety: received at least 1 dose of migalastat or ERT. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| 12-Month OLE |
|
|
Safety Population: All randomized participants who received at least 1 dose of migalastat or ERT.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Migalastat (0-18 Months) | Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days during both treatment periods. |
| BG001 | ERT (0-18 Months) | Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. Participants received an inactive reminder capsule on alternate days 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 | Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate | To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of <2.2 milliliter (mL)/minute (min)/1.73 meter squared (m^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months. | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | Posted | Least Squares Mean | 95% Confidence Interval | mL/min/1.73 m^2/year | Baseline to Month 18 |
|
|
| ||||||||||||||||||||||||||||
| Primary | Annualized Rate Of Change From Baseline To Month 18 In eGFR | The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following: eGFR-CKD-EPI = 141 x min (Serum Creatinine/κ,1)^(α) x max(Serum Creatinine/κ,1)^(-1.209) x 0.993^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: κ is 0.7 for females and 0.9 for males; α is -0.329 for females and -0.411 for males; min indicates the minimum of Serum Creatinine/κ or 1; max indicates the maximum of Serum Creatinine/κ or 1. The annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of <2.2 mL/min/1.73m^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months. | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | Posted | Least Squares Mean | 95% Confidence Interval | mL/min/1.73 m^2/year | Baseline to Month 18 |
| ||||||||||||||||||||||||||||||
| Secondary | Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation | The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)^(-1.154) x (Age)^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female). The eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. | mITT Population: All randomized participants with an α Gal-A mutation that is amenable to migalastat, based on the GLP HEK assay, who received at least 1 dose of study drug, had Baseline and postbaseline mGFR-iohexol values, and postbaseline measure of the estimated GFR using the CKD-EPI equation. | Posted | Least Squares Mean | 95% Confidence Interval | mL/min/1.73 m^2/year | Baseline to Month 18 |
|
Adverse Event (AE) data were collected from the time of signing informed consent at screening/baseline through the follow-up visit (1 mo. after the last treatment in the optional OLE/after the 18-month Randomized Period if the participant did not enroll in the OLE), for up to 30 months. Because participants in the Migalastat (0-18 mo.) and ERT (0-18 mo.) groups were combined into the All Migalastat (0-30 mo.) group, AEs for those groups are included in the All Migalastat (0-30 mo.) group AEs.
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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 (0-18 Months) | Participants received 150 mg of migalastat orally QOD during the 18-month randomized treatment period (0-18 months). Participants received an inactive reminder capsule on alternate days during both treatment periods. | 7 | 36 | 34 | 36 | ||
| EG001 | ERT (0-18 Months) | Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period (0-18 months). Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. | 7 | 21 | 20 | 21 | ||
| EG002 | All Migalastat (0-30 Months) | Participants in this group are composed of participants from the Migalastat (0-18 month) and the ERT (0-18 month) groups. Therefore, participants in the All Migalastat group received at least 1 dose of 150 mg migalastat orally QOD during the 18-month randomized treatment period or the optional 12-month OLE (for a total time period of up to 30 months), regardless of whether or not the participant discontinued the study early, and regardless of whether they were previously randomized to migalastat or ERT during the 18-month randomized phase (0-18 months). | 16 | 51 | 50 | 51 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Perineal abscess | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Device malfunction | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Embolic stroke | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Ventricular tachycardia | Cardiac disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Cardiac failure chronic | Cardiac disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Hernial eventration | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Phaeochromocytoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Palpitations | Cardiac disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Herpes Zoster | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (16.1) | Systematic Assessment |
| |
| Protein urine present | Investigations | MedDRA (16.1) | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA (16.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA (16.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (16.1) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (16.1) | Systematic Assessment |
|
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 |
| 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 |
| C459420 | agalsidase beta |
| C000627036 | agalsidase alfa |
Not provided
Not provided
Not provided
| Lost to Follow-up |
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| Pregnancy |
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| Lack of Efficacy |
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| Declined to participate in the OLE |
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| Male |
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| OG001 | ERT (0-18 Months) | Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE. |
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