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| Tracking Information | |||||
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| First Received Date ICMJE | September 13, 2005 | ||||
| Last Updated Date | June 4, 2008 | ||||
| Start Date ICMJE | September 2005 | ||||
| Primary Completion Date | January 2008 (final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures ICMJE |
Safety and tolerability of 3 dose levels of oral AT1001 | ||||
| Original Primary Outcome Measures ICMJE | Same as current | ||||
| Change History | Complete list of historical versions of study NCT00214500 on ClinicalTrials.gov Archive Site | ||||
| Current Secondary Outcome Measures ICMJE |
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| Original Secondary Outcome Measures ICMJE | Same as current | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | A Study of AT1001 in Patients With Fabry Disease | ||||
| Official Title ICMJE | A Phase 2, Open-Label, Multicenter, Ascending-Dose, 12-Week Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AT1001 in Patients With Fabry Disease | ||||
| Brief Summary | The purpose of this study is to determine whether AT1001 (migalastat hydrochloride) is safe and effective in patients with Fabry disease. |
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| Detailed Description | This open-label study will be conducted in up to 20 patients at five clinical sites in the United States. Patients will undergo a 28-day screening period, including a 14-day run-in with AT1001 to assess eligibility for the study. Patients receiving enzyme replacement therapy or substrate depletion therapy for Fabry disease must undergo a 2-week washout of prior therapy before the 28-day screening period. Patients will receive a daily dose of AT1001 for 12 weeks during the treatment phase. The pharmacokinetics of AT1001 in plasma and urine will be assessed at regular intervals. Safety will be evaluated throughout the treatment period. At the end of the 12-week treatment period, the effect of AT1001 on pharmacodynamic parameters (alpha-Gal A in leukocytes, GL-3 in plasma and urine, and alpha-Gal A and GL-3 in skin biopsy samples) and functional parameters (cardiac function as assessed by electrocardiogram, echocardiogram, and MRI, and renal function as assessed by blood and urine tests, and nerve conduction as assessed by QSART and CASE IV tests) will be evaluated. If the safety profile is acceptable, patients will be permitted to enter a 36-week treatment extension period and continue to receive AT1001, with safety, pharmacodynamic, and functional assessments performed at 12-week intervals. |
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| Study Phase | Phase II | ||||
| Study Type ICMJE | Interventional | ||||
| Study Design ICMJE | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study | ||||
| Condition ICMJE | Fabry Disease | ||||
| Intervention ICMJE | Drug: AT1001 (migalastat hydrochloride) | ||||
| Study Arms / Comparison Groups | |||||
| Publications * | |||||
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* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Completed | ||||
| Enrollment ICMJE | 20 | ||||
| Completion Date | January 2008 | ||||
| Primary Completion Date | January 2008 (final data collection date for primary outcome measure) | ||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Male | ||||
| Ages | 18 Years to 55 Years | ||||
| Accepts Healthy Volunteers | No | ||||
| Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
| Location Countries ICMJE | United States | ||||
| Administrative Information | |||||
| NCT ID ICMJE | NCT00214500 | ||||
| Responsible Party | |||||
| Study ID Numbers ICMJE | AA1565520 (FAB-CL-201) | ||||
| Study Sponsor ICMJE | Amicus Therapeutics | ||||
| Collaborators ICMJE | |||||
| Investigators ICMJE |
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| Information Provided By | Amicus Therapeutics | ||||
| Verification Date | June 2008 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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