VX-272 dose escalation in healthy adults

A Phase 1, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study Evaluating Safety and Pharmacokinetics of VX-272 in Healthy Subjects

This will test single and multiple increasing doses of the experimental drug VX-272 in healthy adults to see how safe it is and how the body handles it.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 128 (estimated)
• Ages: 18 Years to 55 Years
• Sex: All
• Sponsor: Vertex Pharmaceuticals Incorporated (Industry-sponsored)
• Locations: 1 site (Tempe, Arizona)
• Trial ID: NCT07437105 on ClinicalTrials.gov

What this trial studies

This Phase 1, randomized placebo-controlled trial gives single and multiple ascending doses of VX-272 to healthy adult volunteers to collect safety, tolerability, and pharmacokinetic data. Participants must meet weight and BMI criteria and be non-childbearing potential, and those with recent febrile illness or conditions affecting absorption are excluded. Dosing cohorts will escalate based on predefined safety criteria and pharmacokinetic results. The data will inform dose selection and safety monitoring for later studies in people with cystic fibrosis.

Who should consider this trial

Good fit: Ideal candidates are healthy adults over 50 kg with a BMI between 18.0 and 32.0 kg/m2 who are not of childbearing potential and have no recent febrile illness or conditions affecting drug absorption.

Not a fit: People with active illnesses, those of childbearing potential, those weighing 50 kg or less, or individuals with conditions that affect drug absorption — and patients with cystic fibrosis seeking immediate treatment benefit — are unlikely to receive direct therapeutic benefit from this healthy-volunteer Phase 1 study.

Why it matters

Potential benefit: If VX-272 is safe and has favorable pharmacokinetics, it could enable development of a new treatment option for people with cystic fibrosis.

How similar studies have performed: Dose-escalation safety and pharmacokinetic trials in healthy volunteers are a standard early-phase approach that has supported the development of other cystic fibrosis drugs, but VX-272 itself appears to be first-in-human with no prior published human results.

Eligibility criteria

Key Inclusion Criteria:

* Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m\^2)
* A total body weight of more than (\>) 50 kg
* Male and Female participants of non-childbearing potential

Key Exclusion Criteria:

* History of febrile illness or other acute illness that has not fully resolved within 14 days before the first dose of study drug
* Any condition possibly affecting drug absorption

Other protocol defined Inclusion/Exclusion criteria may apply.

Where this trial is running

Tempe, Arizona

• Celerion - Tempe — Tempe, Arizona, United States (Recruiting)

Study contacts

• Study coordinator: Medical Information

Conditions Cystic Fibrosis