A Phase 1, First-in-human Study of VX-634

Vertex Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: VX-634; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05579431

VX22-634-001

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.

Full description

This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.).

Enrollment: 127 patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants of age between 18 to 55 years (inclusive)
• 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
• Participants of non childbearing potential
• Non smoker or ex-smoker for at least 3 months before screening

Key Exclusion Criteria:

• Any condition possibly affecting drug absorption

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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127 participants in 4 patient groups, including a placebo group

Part A

Experimental group

Description:

Participants grouped in different cohorts will receive a single ascending dose of VX-634.

Treatment:

Drug: VX-634

Placebo Part A

Placebo Comparator group

Description:

Participants will be randomized to receive placebo matched to VX-634.

Treatment:

Drug: Placebo

Part B

Experimental group

Description:

Participants grouped in different cohorts will receive multiple doses of VX-634.The dose levels will be determined based on the data from Part A.

Treatment:

Drug: VX-634

Placebo Part B

Placebo Comparator group

Description:

Participants will be randomized to receive placebo matched to VX-634.

Treatment:

Drug: Placebo

Sponsor of this trial

Lead Sponsor

Vertex Pharmaceuticals