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NCT03795363 · ClinicalTrials.gov registry record
Orkambi Treatment in 2 to 5 Year Old Children With CF
A clinical trial, sponsored by Children's Hospital of Philadelphia.
- Completed
- Registry status
- 28
- Enrollment target
NCT03795363: Completed study, sponsored by Children's Hospital of Philadelphia.
NCT03795363 is a clinical trial that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 28 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03795363 has completed, sponsored by Children's Hospital of Philadelphia.
- COMPLETED
- Registry status
- 28 participants
- Enrollment target
Study Summary
The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy expenditure, growth status and gut health and function.
Primary Outcome
Investigators will examine the effects of 24 weeks of orkambi treatment on subject's SEE (sleeping energy expenditure) or REE (resting energy expenditure). Using indirect calorimetry, SEE/REE will be assessed using a computerized metabolic cart Vmax ENCORE at each protocol visit while the child is asleep or resting quietly. SEE/REE will be assessed in the morning if possible and careful note of previous feeding of the child, including the time of day, amount of food, and feeding interval prior t
Interventions
- DRUG Orkambi
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 28 participants |
| Start Date | 2019-04-10 |
| Est. Completion | 2021-06-16 |
What the finished NCT03795363 record still lists
NCT03795363 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 28 participants, a relatively small participant target.
The record links to 0 conditions, and to 1 intervention - of which Orkambi is the first listed.
NCT03795363 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03795363 about?
NCT03795363 is a clinical study titled "Orkambi Treatment in 2 to 5 Year Old Children With CF". The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic fibrosis transmembrane conductance regulator (CFTR) gene on sleeping energy expenditure, growth statu...
What is the current status of trial NCT03795363?
This trial is currently completed. The enrollment target is 28 participants. The study started on 2019-04-10. Estimated completion is 2021-06-16.
What interventions are being tested in trial NCT03795363?
The interventions under investigation include: Orkambi (DRUG).
Who is sponsoring clinical trial NCT03795363?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
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