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VX21-522-001

  • Status
    Accepting Candidates
  • Age
    18 Years - 64 Years
  • Sexes
    All
  • Healthy Volunteers
    No
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Objective

The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.

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.).

Details

Full study title A Phase 1 Single Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
Protocol number OCR43319
ClinicalTrials.gov ID NCT05668741
Phase Phase 1/Phase 2

Eligibility

Key Inclusion Criteria:

  • Body mass index is less than () 50 kg

  • Stable CF disease

  • CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy o Example mutations include but are not limited to, mutations that do not produce CFTR protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice mutations (e.g., 621+1G->T)

  • Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to (鈮�)40 percent (%), MAD: 鈮� 40% to less than or equal to (鈮�) 90%

Key Exclusion Criteria:

  • History of uncontrolled asthma within a year prior to screening

  • History of solid organ or hematological transplantation

  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)

  • Arterial oxygen saturation on room air less than (

Lead researcher

  • Cesar A Trillo-Alvarez, MD
    Critical Care Medicine Specialist, Pulmonologist (Lung Specialist)
    Languages: Spanish, French
    Cesar A Trillo-Alvarez

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  1. Step
    1

    Contact the research team

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    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

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  3. Step
    3

    Provide your consent to participate

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  4. Step
    4

    Participate

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