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Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

NCT00159250 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2019-12-05

Study results available
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Summary

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.

Conditions

Interventions

DRUG

AVI-4658 (PMO)

morpholino antisense oligonucleotide

Sponsors & Collaborators

  • Department of Health, United Kingdom

    collaborator OTHER_GOV

  • Sarepta Therapeutics, Inc.

    collaborator INDUSTRY

  • Imperial College London

    lead OTHER

Principal Investigators

  • Francesco Muntoni, FRCPCH · Dubowitz neuromuscular Centre, Imperial College, London

  • Kate Bushby, MRCP · Institute of Human Genetics, University of Newcastle upon Tyne

  • Volker Straub, FRCPCH · Institute of Human Genetics, University of Newcastle upon Tyne

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
10 Years
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-10-26
Primary Completion
2008-12-31
Completion
2009-03-31

Countries

  • United Kingdom

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00159250 on ClinicalTrials.gov