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| Study 9 of 9 for search of: | "AVI BioPharma, Inc." [Exact] |
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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: | Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study |
| 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. |
| Estimated Enrollment: | 24 |
| Study Start Date: | January 2009 |
| Estimated Study Completion Date: | September 2010 |
| Estimated Primary Completion Date: | March 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
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| Contact: Francesco Muntoni, FRCPCH | 0207 9052869 | f.muntoni@ich.ucl.ac.uk |
| United Kingdom | |
| Dubowitz Neuromuscular Unit, UCL Institute of Child Health | Recruiting |
| London, United Kingdom | |
| Contact: Kanagasabi Ganeshaguru, PhD 020 7905 2221 k.ganeshaguru@ich.ucl.ac.uk | |
| Principal Investigator: Francesco Muntoni, FRCPCH | |
| United Kingdom, England | |
| Royal Victoria Infirmary | Recruiting |
| Newcastle Upon Tyne, England, United Kingdom, NE2 4LP | |
| Contact: Jane Barnes 0191 282 0070 jane.barnes@ncl.ac.uk | |
| Contact: Geoff Bell 0191 241 8649 geoff.bell@nuth.nhs.uk | |
| Principal Investigator: Kate Bushby, MD | |
| Study Director: | Shirley J Leow, MSc | AVI Biopharma, VP Clinical Operations & Project Management |
More Information
| Responsible Party: | University College of London Institute of Child Health ( Professor Francesco Muntoni ) |
| Study ID Numbers: | AVI-4658-28 |
| Study First Received: | December 24, 2008 |
| Last Updated: | August 12, 2009 |
| ClinicalTrials.gov Identifier: | NCT00844597 History of Changes |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency |
|
Muscular Dystrophies Muscular Diseases Genetic Diseases, Inborn Neuromuscular Diseases Musculoskeletal Diseases |
Muscular Disorders, Atrophic Muscular Dystrophy, Duchenne Nervous System Diseases Genetic Diseases, X-Linked |