Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
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ClinicalTrials.gov Identifier: NCT00925301 |
Recruitment Status :
Completed
First Posted : June 22, 2009
Results First Posted : October 30, 2018
Last Update Posted : October 30, 2018
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Sponsor:
Amicus Therapeutics
Information provided by (Responsible Party):
Amicus Therapeutics
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment |
Condition |
Fabry Disease |
Interventions |
Drug: migalastat hydrochloride Drug: Placebo |
Enrollment | 67 |
Participant Flow
Recruitment Details | |
Pre-assignment Details | 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). |
Arm/Group Title | Migalastat (0-6 Months) | Placebo (0-6 Months) | Migalastat (>6-12 Months) | Migalastat (>12-24 Months) |
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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. | 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. | 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. | 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. |
Period Title: 6-Month Double-blind (Stage 1) | ||||
Started | 34 | 33 | 0 [1] | 0 [2] |
Received at Least 1 Dose of Study Drug | 34 | 33 | 0 | 0 |
Intent-to-Treat (ITT) [3] | 34 | 33 | 0 | 0 |
Modified Intent-to-Treat (mITT) [4] | 30 | 30 | 0 | 0 |
Completed | 34 | 30 | 0 | 0 |
Not Completed | 0 | 3 | 0 | 0 |
Reason Not Completed | ||||
Withdrawal by Subject | 0 | 2 | 0 | 0 |
Pregnancy | 0 | 1 | 0 | 0 |
[1]
Zero participants noted because participants did not start Stage 2 yet.
[2]
Zero participants noted because participants did not start the OLE yet.
[3]
All randomized participants regardless of their participation in the study beyond Randomization.
[4]
Participants with Baseline and Month 6 biopsy results.
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Period Title: 6-Month Open-label (Stage 2) | ||||
Started | 0 [1] | 0 [1] | 63 [2] | 0 [3] |
ITT-amenable [4] | 0 | 0 | 47 | 0 |
Completed | 0 | 0 | 60 | 0 |
Not Completed | 0 | 0 | 3 | 0 |
Reason Not Completed | ||||
Withdrawal by Subject | 0 | 0 | 1 | 0 |
Adverse Event | 0 | 0 | 2 | 0 |
[1]
Zero participants noted because participants had completed Stage 1.
[2]
One participant (Migalastat [0-6 Months]) withdrew consent after completing Month 6.
[3]
Zero participants noted because participants did not start the OLE yet.
[4]
Amenable mutations based on Good Laboratory Practice (GLP) Human Embryonic Kidney (HEK) assay.
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Period Title: 12-Month Open-label Extension (Optional) | ||||
Started | 0 [1] | 0 [1] | 0 [2] | 57 [3] |
ITT-amenable [4] | 0 | 0 | 0 | 42 |
Completed | 0 | 0 | 0 | 54 |
Not Completed | 0 | 0 | 0 | 3 |
Reason Not Completed | ||||
Withdrawal by Subject | 0 | 0 | 0 | 1 |
Pregnancy | 0 | 0 | 0 | 1 |
Lost to Follow-up | 0 | 0 | 0 | 1 |
[1]
Zero participants noted because participants had completed Stage 1.
[2]
Zero participants noted because participants had completed Stage 2.
[3]
The OLE was optional for those who completed Stage 2. Reasons for not entering were not recorded.
[4]
Amenable mutations based on GLP HEK assay.
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Baseline Characteristics
Arm/Group Title | Migalastat-Migalastat | Placebo-Migalastat | Total | |
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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. | 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. | Total of all reporting groups | |
Overall Number of Baseline Participants | 34 | 33 | 67 | |
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Safety Population: All randomized participants who received at least 1 dose of study drug.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 34 participants | 33 participants | 67 participants | |
40.0 (13.29) | 44.5 (10.18) | 42.2 (11.99) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 34 participants | 33 participants | 67 participants | |
Female |
22 64.7%
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21 63.6%
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43 64.2%
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Male |
12 35.3%
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12 36.4%
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24 35.8%
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Outcome Measures
Adverse Events
Limitations and Caveats
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.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: | Medical Affairs |
Organization: | Amicus Therapeutics |
Phone: | +1-877-426-4287 (877-4-AMICUS) |
EMail: | MedInfoUSA@amicusrx.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Amicus Therapeutics |
ClinicalTrials.gov Identifier: | NCT00925301 |
Other Study ID Numbers: |
AT1001-011 FACETS ( Other Identifier: Amicus Therapeutics ) 2009-013459-31 ( EudraCT Number ) |
First Submitted: | June 19, 2009 |
First Posted: | June 22, 2009 |
Results First Submitted: | August 10, 2018 |
Results First Posted: | October 30, 2018 |
Last Update Posted: | October 30, 2018 |