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NCT01807949 · ClinicalTrials.gov registry record · Phase 3
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
A Phase 3 study of Cystic Fibrosis, Homozygous for the F508del CFTR Mutation, sponsored by Vertex Pharmaceuticals Incorporated.
- Completed
- Registry status
- Phase 3
- Development phase
- 563
- Enrollment target
- 20
- Study locations
NCT01807949: Completed Phase 3 study of Cystic Fibrosis, Homozygous for the F508del CFTR Mutation, sponsored by Vertex Pharmaceuticals Incorporated.
NCT01807949 is a Phase 3 study of Cystic Fibrosis, Homozygous for the F508del CFTR Mutation that has completed, run by Vertex Pharmaceuticals Incorporated. The registered enrollment target is 563 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (27% lower). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01807949, a Phase 3 study of Cystic Fibrosis, Homozygous for the F508del CFTR Mutation, has completed, sponsored by Vertex Pharmaceuticals Incorporated.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 563 participants
- Enrollment target
- 20
- Study locations
Study Summary
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Primary Outcome
Absolute change from baseline at week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Ivacaftor
- DRUG Lumacaftor Plus Ivacaftor Combination
Study Locations (20)
California
- - Oakland
- - Sacramento
Connecticut
- - Hartford
- - New Haven
Florida
- - Jacksonville
- - Miami
Michigan
- - Detroit
- - Grand Rapids
Colorado
- - Aurora
Indiana
- - Indianapolis
Iowa
- - Iowa City
Kansas
- - Kansas City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 563 participants |
| Start Date | 2013-04 |
| Est. Completion | 2014-04 |
| Phase | Phase 3 |
What the finished NCT01807949 record still lists
NCT01807949 is an interventional study that assigns participants to a tested intervention. The registered 563 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (27% lower).
The record links to 1 condition, with Cystic Fibrosis, Homozygous for the F508del CFTR Mutation appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT01807949 names 20 study sites across 16 states, led by California, Connecticut, Florida.
Frequently Asked Questions
What is clinical trial NCT01807949 about?
NCT01807949 is a clinical study titled "A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation". The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
What is the current status of trial NCT01807949?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 563 participants. The study started on 2013-04. Estimated completion is 2014-04.
What conditions does trial NCT01807949 study?
This clinical trial studies the following conditions: Cystic Fibrosis, Homozygous for the F508del CFTR Mutation.
What interventions are being tested in trial NCT01807949?
The interventions under investigation include: Placebo (DRUG), Ivacaftor (DRUG), Lumacaftor Plus Ivacaftor Combination (DRUG).
Who is sponsoring clinical trial NCT01807949?
This trial is sponsored by Vertex Pharmaceuticals Incorporated, which has 191 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01807949 being conducted?
This trial has 20 study locations across California, Colorado, Connecticut, Florida, Indiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01807949's enrollment target sits among peer trials
563 668th of 2000 higher than 1,332 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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