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A Gene Delivery Study to Evaluate the Safety and Expression of Elevidys in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

A Gene Delivery Study to Evaluate the Safety and Expression of Elevidys in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)
Active, Not RecruitingPhase 2Gene Therapy

Study Overview

Age
2–3 years
Phase
Phase 2
Sponsor
Hoffmann-La Roche
Therapeutic Approach
Gene Therapy
Variant Requirement
Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
Eligible Sex
Male
Ambulation
Ambulatory and Non-Ambulatory
Study Start (Actual)
2023-11-29
Primary Completion (Estimated)
2026-03-04
Study Completion (Estimated)
2030-02-18
Enrollment (Estimated)
13
Countries
BelgiumFranceGermanyItalySpainUnited Kingdom

Study Requirements and Criteria

Steroid Use

Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline

Inclusion Criteria

  • Cohort A: >=3 years of age to <4 years of age
  • Cohort B: >=2 years of age to <3 years of age
  • Able to cooperate with age-appropriate motor assessment testing
  • A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive)

Exclusion Criteria

  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression, within protocol-specified time limits
  • Recombinant Adeno-Associated Virus Serotype rh74 (rAArh74) antibody titers are elevated, as per protocol-specified criteria
  • Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer
  • Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant's ability to comply with the protocol required testing or procedures, or compromise the participant's well-being or safety, or clinical interpretability

Contacts and Locations

Contacts and Locations

Contact

Not yet provided

Locations

This study has 7 locations

Belgium

Liège, Belgium, 3500

Chr de La Citadelle

France

Paris, France, 75015

Hôpital Necker-Enfants Malades

Germany

Essen, Germany, 45147

Universitätsklinikum Essen

Italy

Lazio Locations

Rome, Lazio, Italy, 00168

PU A. Gemelli, Università Cattolica del Sacro Cuore

Spain

Barcelona Locations

Esplugues de Llobregas, Barcelona, Spain, 08950

Hospital Sant Joan De Deu

United Kingdom

London, United Kingdom, WC1N 3JH

Great Ormond Street Hospital for Children

Oxford, United Kingdom, OX3 9DU

John Radcliffe Hospital

Clinical Trial Registry

Learn More

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Elevidys Envol Phase 2 Clinical Trial | DMD Warrior