What side effects are associated with this type of intervention?

Gene transfer therapies, such as delandistrogene moxeparvovec, are still under investigation, but potential side effects may include immune responses to the viral vector used for gene delivery. Exon-skipping therapies like eteplirsen and golodirsen can cause balance disorders, vomiting, contact dermatitis, and upper respiratory tract infections. Glucocorticoids, such as prednisone and deflazacort, are associated with weight gain, osteoporosis, and increased risk of fractures, but they also offer benefits in motor function and delaying disease progression.

What prior FDA approvals exist?

The FDA has approved several genetic therapies for the treatment of Duchenne Muscular Dystrophy (DMD). Eteplirsen, approved in September 2016, is an exon 51 skipping drug that increases dystrophin production in patients with specific deletion mutations. Viltolarsen, approved in August 2020, targets exon 53 skipping to increase dystrophin levels. Golodirsen, another exon 53 skipping therapy, was approved based on its ability to increase dystrophin production. These therapies use antisense oligonucleotides to modify the splicing of dystrophin pre-mRNA, thereby increasing the production of functional dystrophin protein. While these treatments are not gene transfer therapies like delandistrogene moxeparvovec, they share the goal of restoring dystrophin function in DMD patients.

What other types of research exist?

Promising alternatives to Delandistrogene Moxeparvovec for DMD include Viltolarsen and Eteplirsen. Viltolarsen is an antisense oligonucleotide designed for exon 53 skipping, showing increased dystrophin levels and functional improvements in clinical trials. Eteplirsen, another antisense oligonucleotide for exon 51 skipping, has demonstrated increased dystrophin production and functional benefits in patients. Both therapies have shown favorable safety profiles and are FDA-approved for specific DMD mutations.

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Gene Therapy

Gene Therapy for Duchenne Muscular Dystrophy (ENDEAVOR Trial)

Q: What is the mechanism of action of this treatment?

The most common treatments for Duchenne Muscular Dystrophy (DMD) include gene transfer therapy, exon skipping drugs, and glucocorticoids. Gene transfer therapy, such as delandistrogene moxeparvovec, delivers a functional copy of the dystrophin gene to muscle cells, potentially restoring dystrophin production. Exon skipping drugs, like eteplirsen and golodirsen, skip specific exons in the dystrophin gene to produce a shorter but functional dystrophin protein. Glucocorticoids, such as prednisone and deflazacort, improve motor function and reduce inflammation. These treatments are vital for DMD patients as they address the genetic defect or its effects, aiming to slow disease progression and enhance quality of life.

Phase 1

Waitlist Available

Research Sponsored by Sarepta Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing

Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new gene therapy called delandistrogene moxeparvovec in people with Duchenne Muscular Dystrophy (DMD). The therapy aims to insert a healthy gene into the body to help improve muscle function. The study will last several years to evaluate safety and effectiveness.

Who is the study for?

This trial is for boys with Duchenne Muscular Dystrophy (DMD). Different age groups can join: 4-8, ≥8 to <18 years old, and some who don't need steroids yet. They must be able to do motor tests and not have taken gene therapy or certain drugs recently. Those on stable steroid doses can also participate.

What is being tested?

The study is testing delandistrogene moxeparvovec, a gene transfer therapy for DMD. It's an open-label study where everyone gets the treatment to see if it's safe and how well it works over about three years.

What are the potential side effects?

Possible side effects aren't listed here but typically include immune reactions due to gene transfer, potential liver issues from viral vectors used in the therapy, and general risks associated with genetic therapies.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been officially diagnosed with Duchenne Muscular Dystrophy.

Select...

I have been officially diagnosed with Duchenne Muscular Dystrophy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2023 Phase 1 & 2 trial • 4 Patients • NCT03375164

100%

Upper respiratory tract infection

100%

Vomiting

75%

Hepatic enzyme increased

50%

COVID-19

50%

Gastrooesophageal reflux disease

50%

Procedural pain

50%

Decreased appetite

50%

Fatigue

50%

Cough

25%

Pyrexia

25%

Abdominal discomfort

25%

Abdominal distension

25%

Abdominal pain upper

25%

Gastroenteritis

25%

Headache

25%

Dermatitis contact

25%

Anal incontinence

25%

Diarrhoea

25%

Nausea

25%

Eye irritation

25%

Proteinuria

25%

Gastroenteritis viral

25%

Subcutaneous abscess

25%

Viral infection

25%

Asthenia

25%

Clavicle fracture

25%

Cardiomyopathy

25%

Skin papilloma

25%

Influenza A virus test positive

25%

Back pain

25%

Bone pain

25%

Pain in extremity

25%

Asthma

25%

Irritability

100%

80%

60%

40%

20%

Study treatment Arm

Delandistrogene Moxeparvovec

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Delandistrogene MoxeparvovecExperimental Treatment1 Intervention

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

delandistrogene moxeparvovec

2018

Completed Phase 2

~50

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Duchenne Muscular Dystrophy (DMD) include gene transfer therapy, exon skipping drugs, and glucocorticoids. Gene transfer therapy, such as delandistrogene moxeparvovec, delivers a functional copy of the dystrophin gene to muscle cells, potentially restoring dystrophin production. Exon skipping drugs, like eteplirsen and golodirsen, skip specific exons in the dystrophin gene to produce a shorter but functional dystrophin protein. Glucocorticoids, such as prednisone and deflazacort, improve motor function and reduce inflammation. These treatments are vital for DMD patients as they address the genetic defect or its effects, aiming to slow disease progression and enhance quality of life.