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| Sponsored by: |
Amicus Therapeutics |
| Information provided by: | Amicus Therapeutics |
| ClinicalTrials.gov Identifier: | NCT00688597 |
Purpose
The main purpose of this study is to determine the safety and tolerability of three different doses of AT2220 in people affected by Pompe disease. The study will also evaluate the effects of AT2220 on functional parameters in Pompe disease.
| Condition | Intervention | Phase |
|
Pompe Disease |
Drug: AT2220 |
Phase II |
| Genetics Home Reference related topics: | Pompe disease |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety Study |
| Official Title: | An Open-Label, Multicenter, Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease |
| Estimated Enrollment: | 18 |
| Study Start Date: | July 2008 |
| Estimated Study Completion Date: | May 2009 |
| Estimated Primary Completion Date: | March 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|
Cohort 1: Experimental
AT2220 low dose, regimen 1, for 11 weeks
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Drug: AT2220
low dose regimen 1 powder in a bottle for dissolution in water for oral administration |
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Cohort 2: Experimental
AT2220 high dose, regimen 1, for 11 weeks
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Drug: AT2220
high dose, regimen 1
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Cohort 3: Experimental
AT2220 high dose, regimen 2, for 11 weeks
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Drug: AT2220
high dose, regimen 2
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Subjects meeting all eligibility criteria will undergo physical examination, electrocardiogram (ECG), spirometry, muscular strength test, functional muscle test, 6-minute walk test (6MWT) (when appropriate), laboratory tests, MRI and muscle (needle) biopsy. Quality of life will be assessed via SF-36 questionnaire. Functional ability and level of handicap will be assessed by Rotterdam handicap scale.
Eligibility
| Ages Eligible for Study: | 18 Years to 74 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations![]() |
Show 20 Study Locations |
| Amicus Therapeutics |
| Study Director: | Andrew Shenker, MD, PhD | Amicus Therapeutics |
More Information
| Responsible Party: | Amicus Therapeutics ( Andrew Shenker, MD, PhD, Vice President, Clinical Research ) |
| Study ID Numbers: | POM-CL-201 |
| First Received: | May 30, 2008 |
| Last Updated: | June 16, 2008 |
| ClinicalTrials.gov Identifier: | NCT00688597 |
| Health Authority: | United States: Food and Drug Administration |
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