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Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)

Active, not recruiting
Conditions
Cystic Fibrosis
Registration Number
NCT06506773
Lead Sponsor
Vertex Pharmaceuticals Incorporated
Brief Summary

The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
79
Inclusion Criteria
  • Participants with CF less than (<) 6 months of age at the index date
  • Participants not eligible to receive commercial Kalydeco™ (based on local product labels) and are not receiving Kalydeco or any other cystic fibrosis transmembrane conductance regulator gene (CFTR) modulator

Key

Exclusion Criteria
  • Participant whose mother took any CFTR modulator while pregnant with the participant, or who has any history of exposure to a CFTR modulator

Other protocol defined Inclusion/Exclusion criteria apply.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Proportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over TimeFrom Enrollment up to the Infant turning 12 Months of Age
Secondary Outcome Measures
NameTimeMethod
FE-1 level Over TimeFrom Enrollment up to the Infant turning 12 Months of Age

Trial Locations

Locations (50)

Johns Hopkins All Children's Hospital Outpatient Care Center

🇺🇸

Saint Petersburg, Florida, United States

St. Luke's Cystic Fibrosis Center of Idaho

🇺🇸

Boise, Idaho, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

🇺🇸

Chicago, Illinois, United States

Riley Hospital for Children at Indiana University Health

🇺🇸

Indianapolis, Indiana, United States

Boston Childrens Hospital

🇺🇸

Boston, Massachusetts, United States

Michigan Medicine

🇺🇸

Ann Arbor, Michigan, United States

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Billings Clinic

🇺🇸

Billings, Montana, United States

Children's Nebraska

🇺🇸

Omaha, Nebraska, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

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Johns Hopkins All Children's Hospital Outpatient Care Center
🇺🇸Saint Petersburg, Florida, United States
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