Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Browse trials for

• Cystic Fibrosis 1,581
• Fibrosis 3,255

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 251 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Lead sponsor

Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.

Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.

Eligibility criteria

Study arms

• Experimental
  ELX/TEZ/IVA

  Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.

  Drug: ELX/TEZ/IVA · Drug: IVA

Interventions

• DrugELX/TEZ/IVA

  Fixed-dose combination (FDC) tablet for oral administration.

  Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

• DrugIVA

  Tablet for oral administration.

  Also known as: VX-770, ivacaftor

Primary outcomes

1. Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

   Time frame: From Baseline up to Week 100

Secondary outcomes

1. Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

   FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

   Time frame: From Baseline at Week 96

2. Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)

   Sweat samples were collected using an approved collection device.

   Time frame: From Baseline at Week 96

3. Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI)

   BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).

   Time frame: From Baseline at Week 96

4. Part A: Absolute Change From Parent Study Baseline in BMI Z-score

   BMI was defined as weight in kilogram (kg) divided by squared height in meters (m\^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard.

   Time frame: From Baseline at Week 96

5. Part A: Absolute Change From Parent Study Baseline in Body Weight

   Time frame: From Baseline at Week 96

6. Part A: Absolute Change From Parent Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

   The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

   Time frame: From Baseline at Week 96

Baseline characteristics

Publications

• Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. PubMed 33331662 ↗

Study documents

• Study protocol · Jul 29, 2020
• Statistical analysis plan · Feb 2, 2022

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.