Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT04183790
Collaborator
(none)
64
21
1
49.4
3
0.1
Study Details
Study Description
Brief Summary
This study will evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
64 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
Actual Study Start Date
:
Feb 17, 2020
Anticipated Primary Completion Date
:
Apr 1, 2024
Anticipated Study Completion Date
:
Apr 1, 2024
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Triple Combination Arm Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening. |
Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
Other Names:
Drug: IVA
Mono tablet for oral administration.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Safety and tolerability as assessed by adverse events (AEs) and serious adverse events (SAEs) [From Baseline up to Week 196]
Secondary Outcome Measures
- Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [From Baseline up to Week 192]
- Absolute change in sweat chloride (SwCl) [From Baseline up to Week 192]
- Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [From Baseline up to Week 192]
- Absolute change in body mass index (BMI) [From Baseline up to Week 192]
- Absolute change in BMI-for-age z-score [From Baseline up to Week 192]
- Number of pulmonary exacerbations (PEx) [From Baseline up to Week 192]
- Number of CF-related hospitalizations [From Baseline up to Week 192]
- Absolute change in lung clearance index 2.5 (LCI 2.5) [From Baseline up to Week 192]
- Absolute change in weight [From Baseline up to Week 192]
- Absolute change in weight-for-age z-score [From Baseline up to Week 192]
- Absolute change in height [From Baseline up to Week 192]
- Absolute change in height-for-age z-score [From Baseline up to Week 192]
Eligibility Criteria
Criteria
Ages Eligible for Study:
6 Years
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
- Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
Key Exclusion Criteria:
- History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Hospital of Orange County | Orange | California | United States | 92868 |
| 2 | Children's Hospital Colorado | Aurora | Colorado | United States | 80045 |
| 3 | Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | United States | 60611 |
| 4 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
| 5 | Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | United States | 55404 |
| 6 | The Children's Mercy Hospital | Kansas City | Missouri | United States | 64108 |
| 7 | Northwell Health- Long Island Jewish Medical Center | New Hyde Park | New York | United States | 11040 |
| 8 | Clinical Research of Charlotte | Charlotte | North Carolina | United States | 28277 |
| 9 | Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | United States | 44106 |
| 10 | Nationwide Children's Hospital | Columbus | Ohio | United States | 43205 |
| 11 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
| 12 | Texas Children's Hospital | Houston | Texas | United States | 77030 |
| 13 | Seattle Children's Hospital | Seattle | Washington | United States | 98105 |
| 14 | Queensland Children's Hospital | South Brisbane | Australia | ||
| 15 | The Children's Hospital at Westmead | Westmead | Australia | ||
| 16 | The Hospital for Sick Children | Toronto | Canada | ||
| 17 | British Columbia's Children's Hospital | Vancouver | Canada | ||
| 18 | Children's Health Ireland at Crumlin | Dublin | Ireland | ||
| 19 | Children's Health Ireland at Temple Street | Dublin | Ireland | ||
| 20 | Birmingham Children's Hospital - NHS Foundation Trust | Birmingham | United Kingdom | ||
| 21 | Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT04183790
Other Study ID Numbers:
- VX19-445-107
- 2019-001827-11
First Posted:
Dec 3, 2019
Last Update Posted:
Feb 7, 2022
Last Verified:
Feb 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms: