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| Sponsor: | Imperial College London |
|---|---|
| Collaborator: |
Department of Health, Taiwan |
| Information provided by: | Imperial College London |
| ClinicalTrials.gov Identifier: | NCT00159250 |
Purpose
Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, arises from mutations in the dystrophin gene. Antisense therapy with the use of antisense oligonucleotides (AON) has the potential to restore effectively the production of dystrophin, the defective protein, in >70% of DMD. This could result in increased life expectancy through improved muscle survival and function. Recent scientific research has demonstrated the potential of this technique to skip mutated dystrophin exons, restore the reading frame and generate functional dystrophin protein. Having demonstrated proof-of-principle in human cell culture and animal model studies, we now intend to determine efficacy and safety of this approach to induce dystrophin exon skipping in children with DMD.
The specific aim of this phase I/II study is to assess efficacy (dystrophin production) and safety of intramuscular administered morpholino oligomer directed against exon 51 (AVI-4658 PMO). We are performing parallel preclinical studies to develop methods of systemic delivery that will be necessary for future phase II/III clinical studies.
| Condition | Intervention | Phase |
|---|---|---|
|
Duchenne Muscular Dystrophy |
Drug: AVI-4658 (PMO) |
Phase I Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Single Blind (Subject), Placebo Control, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 |
| Estimated Enrollment: | 9 |
| Study Start Date: | October 2007 |
| Estimated Study Completion Date: | March 2009 |
| Estimated Primary Completion Date: | December 2008 (Final data collection date for primary outcome measure) |
Show Detailed Description
Eligibility| Ages Eligible for Study: | 12 Years to 17 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United Kingdom | |
| Dubowitz Neuromuscular Centre, Hammersmith Hospital and Clinical Trails Unit, St Mary's Hospital | |
| London, United Kingdom, W12 0HS | |
| Principal Investigator: | Francesco Muntoni, FRCPCH | Dubowitz neuromuscular Centre, Imperial College, London |
| Study Director: | Kate Bushby, MRCP | Institute of Human Genetics, University of Newcastle upon Tyne |
| Study Director: | Volker Straub, FRCPCH | Institute of Human Genetics, University of Newcastle upon Tyne |
More Information
| Responsible Party: | Imperial College London ( Mr Gary Roper/Manager Clinical Research Governance Organisation ) |
| Study ID Numbers: | 05/MRE12/32 |
| Study First Received: | September 8, 2005 |
| Last Updated: | July 27, 2009 |
| ClinicalTrials.gov Identifier: | NCT00159250 History of Changes |
| Health Authority: | United Kingdom: Department of Health |
|
Duchenne muscular dystrophy; antisense oligonucleotides; gene restoration; deletion |
|
Muscular Dystrophies Muscular Diseases Genetic Diseases, Inborn Neuromuscular Diseases Musculoskeletal Diseases |
Muscular Disorders, Atrophic Muscular Dystrophy, Duchenne Nervous System Diseases Genetic Diseases, X-Linked |