4D-710-C001 in CF
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults with Cystic Fibrosis
- Sex at Birth: Any
- Age: Adult (18 - 64), Older Adult (65+)
- Accepting Healthy People: No
- Type: Interventional / Therapeutic
- Trial Phase: Phase I/II
- Conditions Being Studied: Cystic Fibrosis (CF)
Study Purpose
The purpose of this study is to evaluate the safety and tolerability of a single dose of 4D-710 delivered via nebulization in people with cystic fibrosis who are not eligible for CFTR modulator therapy or could not tolerate CFTR modulator therapy due to side effects. 4D-710 contains a modified copy of the human CFTR gene packaged in an adeno-associated virus (AAV) capsid that cannot replicate. Absorption of 4D-710 by airway cells should allow them to make functional CFTR protein. Participants in the study undergo a bronchoscopy procedure under sedation during which samples of airway cells are collected to see if 4D-710 works as planned.
Locations
UH Cleveland Medical Center
11100 Euclid Avenue
Cleveland OH, 44106
- UH IRB: SITE00001631
- StudyID: 2021-0820
- ClinicalTrials.gov: NCT05248230
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