Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Purpose
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).
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Under 10 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Part A: - Less than 10 years of age at the first study visit. - Documentation of a CF diagnosis. Part B: - Participated in Part A OR less than 7 years of age at the first study visit. - Documentation of a CF diagnosis. - CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor). - Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Exclusion Criteria
- Part A and Part B: - Use of an investigational drug within 28 days prior to and including the first study visit. - Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 28 days prior to and including the first study visit. - Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Part A | Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. |
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| 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. |
|
Recruiting Locations
Birmingham 4049979, Alabama 4829764 35233
Palo Alto 5380748, California 5332921 94304
Aurora 5412347, Colorado 5417618 80045
Jacksonville 4160021, Florida 4155751 32207
Miami 4164138, Florida 4155751 33136
Orlando 4167147, Florida 4155751 32827
Indianapolis 4259418, Indiana 4921868 46202
Kansas City 4273837, Kansas 4273857 66160
Boston 4930956, Massachusetts 6254926 02115
Ann Arbor 4984247, Michigan 5001836 48109
Grand Rapids 4994358, Michigan 5001836 49503
Minneapolis 5037649, Minnesota 5037779 55404
Minneapolis 5037649, Minnesota 5037779 55455
Kansas City 4393217, Missouri 4398678 64108
St Louis 4407066, Missouri 4398678 63110
Buffalo 5110629, New York 5128638 14203
New York 5128581, New York 5128638 10032
Syracuse 5140405, New York 5128638 13210
Valhalla 5142090, New York 5128638 10595
Cincinnati 4508722, Ohio 5165418 45229
Columbus 4509177, Ohio 5165418 43205
Oklahoma City 4544349, Oklahoma 4544379 73104
Portland 5746545, Oregon 5744337 97239
Hershey 5193342, Pennsylvania 6254927 17033
Philadelphia 4560349, Pennsylvania 6254927 19104
Pittsburgh 5206379, Pennsylvania 6254927 15224
Dallas 4684888, Texas 4736286 75207
Fort Worth 4691930, Texas 4736286 76104
Houston 4699066, Texas 4736286 77030
Salt Lake City 5780993, Utah 5549030 84113
Burlington 5234372, Vermont 5242283 05401
Charlottesville 4752031, Virginia 6254928 22903
Seattle 5809844, Washington 5815135 98105
Madison 5261457, Wisconsin 5279468 53792
More Details
- NCT ID
- NCT04509050
- Status
- Recruiting
- Sponsor
- Sonya Heltshe
Detailed Description
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health. Total duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years. Part B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.