An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis
4D Molecular Therapeutics
Summary
This is a Phase 1/2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.
Description
This Phase 1/2 trial will evaluate the safety, tolerability, and preliminary efficacy of 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who are ineligible or unable to tolerate CFTR modulator therapy. A sub-study will evaluate 4D-710 in a cohort of adults with CF advanced lung disease and/or frequent pulmonary exacerbation (PE) while on currently available CFTR modulator therapy.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria (Primary Study): 1. 18 years and older 2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including: 1. Sweat chloride ≥ 60 mmol/L 2. Mutation Status * Bi-allelic mutations in the CFTR gene, or * Single mutation in the CFTR gene and clinical manifestations of CF lung disease 3. Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects. 3. Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening 4. Resting…
Interventions
- Biological4D-710
4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).
Locations (20)
- University of Alabama Child Health Research UnitBirmingham, Alabama
- The University of ArizonaTucson, Arizona
- University of California San FranciscoSan Francisco, California
- National Jewish HealthDenver, Colorado
- University of FloridaGainesville, Florida
- University of Miami HospitalMiami, Florida