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| Sponsor: | AVI BioPharma, Inc. |
|---|---|
| Collaborator: |
British Medical Research Council |
| Information provided by: | AVI BioPharma, Inc. |
| ClinicalTrials.gov Identifier: | NCT00844597 |
Purpose
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
| Condition | Intervention | Phase |
|---|---|---|
|
Duchenne Muscular Dystrophy |
Drug: AVI-4658 for Injection |
Phase I Phase II |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Clinical Study to Assess the Safety fo AVI-4658 in Subjects With Duchenne Muscular Dystrophy Due to a Frame-shift Mutation Amenable to Correction by Skipping Exon 51. |
| Enrollment: | 19 |
| Study Start Date: | January 2009 |
| Study Completion Date: | December 2010 |
| Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 1
not applicable
|
Drug: AVI-4658 for Injection
AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline as follows: Cohort 1: 0.5mg/kg once weekly for 12 weeks; Cohort 2: 1.0mg/kg once weekly for 12 weeks; Cohort 3: 2.0mg/kg once weekly for 12 weeks; Cohort 4: 4.0mg/kg once weekly for 12 weeks; Cohort 5: 10.0mg/kg once weekly for 12 weeks; Cohort 6: 20.0mg/kg once weekly for 12 weeks |
Primary outcome is safety, tolerability and dose selection for future studies.
Eligibility| Ages Eligible for Study: | 5 Years to 15 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United Kingdom | |
| Great Ormond Street Hospital | |
| London, England, United Kingdom, WC1N 3JH | |
| Royal Victoria Infirmary | |
| Newcastle Upon Tyne, England, United Kingdom, NE2 4LP | |
| Study Director: | Austen Eddy, MSM | AVI BioPharma, Director, Clinical Operations |
More Information
| Responsible Party: | Professor Francesco Muntoni, University College of London Institute of Child Health |
| ClinicalTrials.gov Identifier: | NCT00844597 History of Changes |
| Other Study ID Numbers: | AVI-4658-28 |
| Study First Received: | December 24, 2008 |
| Last Updated: | April 22, 2011 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency |
|
Muscular Dystrophy, Duchenne Muscular Dystrophies Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn |