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| Sponsors and Collaborators: |
Imperial College London Department of Health |
| 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 |
| Genetics Home Reference related topics: | Duchenne and Becker muscular dystrophy L1 syndrome |
| MedlinePlus related topics: | Muscular Dystrophy |
| 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: | December 2008 |
| Estimated Primary Completion Date: | September 2008 (Final data collection date for primary outcome measure) |
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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| Contact: Francesco Muntoni, FRCPCH | +442079052111 | f.muntoni@imperial.ac.uk |
| Contact: Kinali Maria, MRCPCH | + 44 2079052111 | m.kinali@imperial.ac.uk |
| United Kingdom | |||||
| Dubowitz Neuromuscular Centre, Hammersmith Hospital and Clinical Trails Unit, St Mary's Hospital | Recruiting | ||||
| London, United Kingdom, W12 0HS | |||||
| Contact: Francesco Muntoni, FRCPCH +44 20 7905 2111 f.muntoni@imperial.ac.uk | |||||
| Contact: Maria Kinali, MRCPCH +44 20 7905 2111 | |||||
| Principal Investigator: Francesco Muntoni, FRCPCH | |||||
| Sub-Investigator: Mary Rutherford, FRCR | |||||
| Sub-Investigator: Maria Kinali, MRCPCH | |||||
| Sub-Investigator: Virginia Arechavala, PhD | |||||
| Sub-Investigator: Caroline Sewry, PhD | |||||
| Sub-Investigator: Lucy Feng, PhD | |||||
| Imperial College London |
| Department of Health |
| 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
Department of Health Funding for "molecular patches" research 
  |
AVI biopharma 
  |
| Responsible Party: | Imperial College London ( Mr Gary Roper/Manager Clinical Research Governance Organisation ) |
| Study ID Numbers: | 05/MRE12/32 |
| First Received: | September 8, 2005 |
| Last Updated: | May 8, 2008 |
| ClinicalTrials.gov Identifier: | NCT00159250 |
| Health Authority: | United Kingdom: Department of Health |
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