Gene Therapy Study for Cystic Fibrosis
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-710 in Adults with Cystic Fibrosis
- Sex: 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
In people with cystic fibrosis, the instructions for making the CFTR protein contain an error that leads to abnormal production or abnormal function of the CFTR protein. Adeno-associated viruses (AAV) are small viruses that are commonly found in nature and are not known to cause disease in humans. 4D-710 is an AAV that was changed in the laboratory to deliver a normal copy of the CFTR gene to the lungs, thereby providing a copy of instructions for making normal CFTR protein. The process of providing a normal copy of the gene is called gene therapy. 4D-710 has been changed so that it cannot make copies of itself. The purpose of this study is to see if 4D-710 is safe for use in adults with cystic fibrosis and to see if 4D-710 can help lung function.
Who Can Participate
Age: 18 years and older
Locations
UH Cleveland Medical Center
11100 Euclid Avenue
Cleveland OH, 44106
- UH IRB: SITE00001631
- StudyID: 2021-0820
- ClinicalTrials.gov: NCT05248230
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