Clinical trial NCT06506773: Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
Official title: A Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis Less Than 12 Months of Age
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2024-07-17
Enrollment: 79
Sex: ALL
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants with CF less than (\<) 6 months of age at the index date who are not eligible for commercial Kalydeco™ (according to local product labels). The day of enrollment into this study will be referred to as the "index date".

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.


Eligibility criteria: Key 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.

Primary outcomes:
measure: Proportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over Time
timeFrame: From Enrollment up to the Infant turning 12 Months of Age
Secondary outcomes:
measure: FE-1 level Over Time
timeFrame: From Enrollment up to the Infant turning 12 Months of Age
Other outcomes:
Locations:
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Hospital Outpatient Care Center
state: Florida
city: Boise
country: United States
facility: St. Luke's Cystic Fibrosis Center of Idaho
state: Idaho
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University Health
state: Indiana
city: Boston
country: United States
facility: Boston Childrens Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: Michigan Medicine
state: Michigan
city: Minneapolis
country: United States
facility: Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Billings
country: United States
facility: Billings Clinic
state: Montana
city: Omaha
country: United States
facility: Children's Nebraska
state: Nebraska
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
state: Ohio
city: Dayton
country: United States
facility: Dayton Children's Hospital
state: Ohio
city: Toledo
country: United States
facility: ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
state: Ohio
city: Columbia
country: United States
facility: Prisma Health Clinical Research Unit - Midlands
state: South Carolina
city: Sioux Falls
country: United States
facility: Sanford Children's Speciality Clinic
state: South Dakota
city: Tyler
country: United States
facility: The University of Texas Health Science Center at Tyler
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University Hospital Systems, Children's Pavilion
state: Virginia
city: Spokane
country: United States
facility: Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
state: Washington
city: Morgantown
country: United States
facility: West Virginia University
state: West Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Nedlands
country: Australia
facility: The Kids Research Institute Australia
city: North Adelaide
country: Australia
facility: Women's and Children's Hospital
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Montreal
country: Canada
facility: McGill University Health Centre, Glen Site, Montreal Children's Hospital
city: Quebec City
country: Canada
facility: Centre Hospitalier de Quebec - Universite Laval
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Essen
country: Germany
facility: Kinderklinik III, Abt. fur Pneumologie
city: Potsdam
country: Germany
facility: Klinikum Westbrandenburg (CF)
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
city: Christchurch
country: New Zealand
facility: Canterbury District Health Board
city: Grafton
country: New Zealand
facility: Starship Children's Hospital
city: Gdańsk
country: Poland
facility: Pediatric Hospital Polanki named of Maciej Płażyński
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Zurich
country: Switzerland
facility: Kinderspital Zurich
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
city: Glasgow
country: United Kingdom
facility: Royal Hospital for Children
city: Leeds
country: United Kingdom
facility: Leeds Regional Children's Cystic Fibrosis Centre
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary, University of Leicester
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: London
country: United Kingdom
facility: The Royal London Children's Hospital
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals NHS Trust, Children's Clinical Research Facility
city: Stoke on Trent
country: United Kingdom
facility: Royal Stoke University Hospital
Study officials:
References: