Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children

Conditions

• Cystic Fibrosis

Eligibility Criteria

Sex

ALL

Age

N/A - 10 Years

Healthy Volunteers

Not accepted

Interventions

• Ivacaftor or elexacaftor/tezacaftor/ivacaftor — DRUG
  In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators

Study Details

This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).

Key Dates

Start date

Nov 18, 2020

Status verified

Aug 2024

Primary completion

Dec 1, 2029

Completion

Dec 1, 2029

Study Design

Enrollment

210 participants (estimated)

Arms

• Arm: Part A
  Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.
• Arm: Part B
  Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them.

Primary Outcome Measure

Part A Primary Outcome Measure: Change in weight-for-age z-scores [ Time Frame: Baseline to 12 months ]

Central Contacts

• Rachael Buckingham
  206-884-7517
  [email protected]
• Anna Mead
  206-884-7531
  [email protected]

Locations (35)

Find similar trials in Birmingham, AL

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