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VX-522 phase 1/2 study in adults with cystic fibrosis
NL-OMON59632Phase 2
Key facts
- Status
- Completed
- Start
- Jun 2024
- Countries
- 1
- Sponsor
- Vertex Pharmaceuticals
- Modality
- Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Drugs
- vx-522
- Conditions
- cystic fibrosis (CF)
01
Overview
Phase 2, open-label trial of vx-522 in cystic fibrosis (CF), sponsored by Vertex Pharmaceuticals. Primary endpoint: Safety and tolerability through Week 8. Started Jun 2024; status: completed.
02
Design
- Study design
- Non-randomizedOpen-label
03
Who it enrols
Adults aged 18 to 65 years with stable cystic fibrosis and CFTR mutations on both alleles that are not responsive to CFTR modulator therapy. Participants had to be on a stable CF regimen, have adequate lung function, BMI below 30 kg/m^2, weight above 50 kg, no active significant infection, and no major hepatic, renal, cardiac, viral, or pregnancy-related exclusion factors.
04
Endpoints
Primary
Safety and tolerability through Week 8
Secondary
Change from baseline in ppFEV1 at Day 29 in MAD T1
Change from baseline and pre-run-in baseline in ppFEV1 at Day 29 in MAD T2
Safety through safety follow-up
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