Open-Label Phase 3 Long-Term Safety Study of Migalastat (AT1001-041)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01458119 |
|
Recruitment Status :
Terminated
(Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons.)
First Posted : October 24, 2011
Results First Posted : October 2, 2018
Last Update Posted : October 2, 2018
|
- Study Details
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fabry Disease | Drug: migalastat hydrochloride | Phase 3 |
Study AT1001-041 was an open-label, noncomparative, multicenter, long-term extension study for participants with Fabry disease who completed treatment in one of three previous trials of migalastat (AT1001-011 [NCT00925301], AT1001-012 [NCT01218659], or FAB-CL-205 [NCT00526071]). In these trials, migalastat was given as monotherapy. This was an extension study designed to evaluate the long-term safety and efficacy of migalastat for the treatment of Fabry disease. Study visits occurred every 6 months (m). Visit evaluations included physical examinations, clinical laboratory parameters, adverse events, and participant reported outcomes.
The study consisted of a Baseline Visit, which was performed at the time of the final visit of the previous study, followed by clinic visits every 6 m for each year of the study. Study assessments included a physical examination, echocardiography, laboratory parameters, and participant-reported outcomes. Since participants enrolled in the study at varying time points based on the completion of the preceding migalastat study, treatment duration varied among participants. No maximum treatment duration was defined. There were no control groups in this study; all participants received migalastat as a 150-mg capsule taken orally once every other day (QOD) and inactive reminder capsules on alternate days.
The sponsor (Amicus Therapeutics) discontinued Study AT1001-041 for logistical reasons and not due to either safety concerns or lack of efficacy. For participants who were ongoing in Study AT1001-041 at the time of discontinuation, the investigators were offered participation in a similar open-label, long-term migalastat treatment study (AT1001-042 [NCT02194985]) for participants ongoing at discontinuation.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 85 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease |
| Actual Study Start Date : | October 14, 2011 |
| Actual Primary Completion Date : | February 17, 2016 |
| Actual Study Completion Date : | February 17, 2016 |
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Migalastat
Migalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.
|
Drug: migalastat hydrochloride
Oral capsule QOD
Other Names:
|
- Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months. ]An adverse event (AE) was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at the time of reporting; visits typically occurred every 6 months. A TEAE was defined as an AE starting on or after the first study drug administration date. Serious AEs were life-threatening or resulted in death, persistent or significant incapacitation, inpatient or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: minimal discomfort, does not interfere with normal everyday activities; Moderate: sufficiently discomforting, interferes with normal everyday activities; Severe: prevents normal everyday activities. The number of participants experiencing TEAEs is presented for those who received migalastat treatment. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
- Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline, Every 6 m until the End of Study (42 m) ]
The annualized rate of change of the eGFR was assessed per participant by the slope of the simple linear regression between the observed values and the assessment times. It was calculated by using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation (eGFR [CKD-EPI]) and the Modification of Diet in Renal Disease (MDRD) equation (eGFR [MDRD]). The equations are as follows:
eGFR [MDRD] = 175 * (Serum Creatinine)^-1.154 * (Age)^-0.203 * 1.212 (if black or African American) * 0.742 (if female); eGFR [CKD-EPI] = 141 * min(serum creatinine/kappa,1)^alpha * max(serum creatinine/kappa, 1)^-1.209 * 0.993^age * 1.1018(if female) * 1.159(if black or African American), where kappa is 0.7 for females and 0.9 for males, alpha is -0.329 for females and -0.411 for males, min is minimum of serum creatinine/kappa or 1, and max is the maximum of serum creatinine/kappa or 1. The number of participants with at least a Baseline and a post-Baseline value are presented.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 16 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Completed migalastat treatment in a previous Fabry disease protocol
- Both male and female participants were enrolled
- Age 16 years or older
- Male and female participants had to agree to use protocol-identified acceptable contraception
Exclusion Criteria:
- Estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliters/minute/1.73 square meters (mL/min/1.73 m^2) unless there was a measured GFR available within 3 m of the Baseline Visit that was >30 mL/min/1.73 m^2
- Had undergone, or was scheduled to undergo, kidney transplantation or was currently on dialysis
- Pregnant or breast feeding
- Treated with another investigational drug (except migalastat) within 30 days of study start
- Unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator
- Had documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 m before the Baseline Visit
- Had clinically significant, unstable cardiac disease in the opinion of the investigator
- Had a history of allergy or sensitivity to migalastat (including excipients) or to other iminosugars
- Required treatment with Glyset (miglitol) or Zavesca (miglustat)
- Had any intercurrent illness or condition that may have precluded the participant from fulfilling the protocol requirements
- Had a severe or unsuitable concomitant medical condition
- Had a clinically significant abnormal laboratory value and a clinically significant electrocardiogram finding at the Baseline Visit.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01458119
| United States, Georgia | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Chicago, Illinois, United States, 60611 | |
| United States, Kansas | |
| Kansas City, Kansas, United States, 66160 | |
| United States, Massachusetts | |
| Boston, Massachusetts, United States, 02114 | |
| United States, Michigan | |
| Grand Rapids, Michigan, United States, 49525 | |
| United States, New York | |
| New York, New York, United States, 10016 | |
| United States, Oregon | |
| Portland, Oregon, United States, 97239 | |
| United States, Pennsylvania | |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| United States, Texas | |
| Dallas, Texas, United States, 75246 | |
| United States, Virginia | |
| Fairfax, Virginia, United States, 22030 | |
| United States, Washington | |
| Seattle, Washington, United States, 98195 | |
| Argentina | |
| Pilar, Argentina, B1629ODT | |
| Australia | |
| Adelaide, Australia, 5000 | |
| Parkville, Australia, 3050 | |
| Belgium | |
| Edegem, Belgium, 2650 | |
| Brazil | |
| Porto Alegre, Brazil, 90035-903 | |
| Canada | |
| Montreal, Canada, H4J 1C5 | |
| Denmark | |
| Copenhagen, Denmark, 2100 | |
| Egypt | |
| Cairo, Egypt, 11451 | |
| France | |
| Garches, France, 92380 | |
| Italy | |
| Roma, Italy, 00168 | |
| Spain | |
| Barcelona, Spain, 08025 | |
| Turkey | |
| Ankara, Turkey, 06500 | |
| United Kingdom | |
| London, United Kingdom, NW3 2QG | |
| Salford, United Kingdom, M6 8HD | |
| Study Director: | Medical Monitor, Clinical Research | Amicus Therapeutics |
| Responsible Party: | Amicus Therapeutics |
| ClinicalTrials.gov Identifier: | NCT01458119 |
| Other Study ID Numbers: |
AT1001-041 2011-004800-40 ( EudraCT Number ) |
| First Posted: | October 24, 2011 Key Record Dates |
| Results First Posted: | October 2, 2018 |
| Last Update Posted: | October 2, 2018 |
| Last Verified: | October 2018 |
|
Amicus Therapeutics AT1001 Galafold Migalastat |
|
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |