Precision BioSciences announced the commencement of a Phase 1/2a trial of PBGENE-DMD (NCT07429240) in ambulatory patients with Duchenne aged 2 to 7 years. In this trial, single dose of FUNCTION-DMD will be studied over 130 weeks. Read More: Precision BioSciences Receives U.S. FDA Clearance of Investigational New Drug Application
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This Phase 1/2a study is designed to assess the safety, tolerability, and early signs of effectiveness of PBGENE-DMD in individuals with DMD who have mutations suitable for excision of exons 45–55. In light of the limitations of current treatment options, PBGENE-DMD offers a promising new strategy aimed at delivering a potentially one-time, long-lasting correction of the underlying genetic defect in the largest molecular subgroup of people with DMD.
A total of up to 18 participants may be enrolled in this trial. Total duration of trial participation for each participant: approximately 130 weeks.
What is PBGENE-DMD Phase 1/2a Clinical Trial?
This is an early-stage clinical trial testing a new treatment called PBGENE-DMD in boys and men with Duchenne muscular dystrophy (DMD) who have specific genetic changes that may respond to this therapy. Participants will receive a single dose of the treatment through an IV (a vein in the arm). Doctors will closely monitor safety, side effects, and early signs that the treatment is working.
To help reduce possible immune reactions, participants will also receive short-term medications around the time of treatment.
The study has two parts. In Part 1, researchers will determine a safe and well-tolerated dose. If that dose is confirmed, Part 2 will include additional participants to further evaluate the treatment.
Up to 18 participants will take part in the study. Each person will be followed for about 2½ years (approximately 130 weeks).
Learn More: PBGENE-DMD Gene Editing Therapy: Benefits, Eligibility, and FAQs
PBGENE-DMD Phase 1/2a – NCT07429240
A Phase 1/2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)
Study Start (Estimated): 2026-03
Primary Completion (Estimated): 2029-11
Study Completion (Estimated): 2029-12
Number of Participants (Estimated): 18
Study Type: Interventional
Location: Arkansas Children’s Hospital
Ages Eligible for Study: 2 Years to 7 Years (Child)
Principal inclusion and exclusion criteria. – NCT07429240
How to Participate in the PBGENE-DMD Phase 1/2a Clinical Trial
To participate in the PBGENE-DMD Phase 1/2a clinical trial, you can use the contact information below. Learn More: How to Participate in Clinical Trials for Duchenne (DMD)?
Name: Eileen Bristow
Phone Number: +1-610-462-1004
Email: [email protected]
Read More: Clinical Trials for Duchenne (List of All Researches)
