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Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

NCT00844597 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2015-10-06

Study results available
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Summary

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).

Conditions

Interventions

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 in 6 dose cohorts.

Sponsors & Collaborators

  • British Medical Research Council

    collaborator OTHER_GOV

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Austen Eddy, MSM · AVI BioPharma, Director, Clinical Operations

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
15 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-01-31
Primary Completion
2010-06-30
Completion
2010-12-31

Countries

  • United Kingdom

Study Locations

More Related Trials

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 NCT00844597 on ClinicalTrials.gov