Trials / Completed
CompletedNCT03625466
A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
An Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 51 (actual)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
- Sex
- All
- Age
- 2 Years – 5 Years
- Healthy volunteers
- Not accepted
Summary
This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | LUM/IVA | FDC tablets or granules for oral administration. |
| DRUG | LUM/IVA | FDC granules for oral administration. |
| DRUG | Placebo | Placebo matched to LUM/IVA for oral administration. |
Timeline
- Start date
- 2018-08-10
- Primary completion
- 2020-10-09
- Completion
- 2021-10-07
- First posted
- 2018-08-10
- Last updated
- 2022-11-02
- Results posted
- 2021-12-08
Locations
5 sites across 1 country: Germany
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03625466. Inclusion in this directory is not an endorsement.