A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

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 87 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

• Placebo comparator
  Part 1: Placebo

  Participants received placebo matched to VX-121/TEZ/VX-561 triple combination (TC) for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.

  Drug: Placebo

• Experimental
  Part 1: VX-121/TEZ/VX-561 TC - Low Dose

  Participants received VX-121 5 milligram (mg) once daily (qd)/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period

  Drug: VX-121 · Drug: TEZ · Drug: VX-561

• Experimental
  Part 1: VX-121/TEZ/VX-561 TC - Medium Dose

  Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.

  Drug: VX-121 · Drug: TEZ · Drug: VX-561

• Experimental
  Part 1: VX-121/TEZ/VX-561 TC - High Dose

  Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.

  Drug: VX-121 · Drug: TEZ · Drug: VX-561

• Active comparator
  Part 2: TEZ/IVA

  Following run-in period with TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg/IVA 150 mg q12h for 4 weeks in the washout period.

  Drug: TEZ/IVA · Drug: IVA

• Experimental
  Part 2: VX-121/TEZ/VX-561 TC - High Dose

  Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.

  Drug: VX-121 · Drug: TEZ · Drug: VX-561

Interventions

• DrugVX-121

  Tablets for oral administration.

• DrugTEZ

  TEZ tablet for oral administration.

  Also known as: VX-661, Tezacaftor

• DrugVX-561

  Tablets for oral administration.

  Also known as: CTP-656, Deutivacaftor (D-IVA)

• DrugTEZ/IVA

  Fixed-dose combination tablets for oral administration.

  Also known as: VX-661/VX-770, Tezacaftor/Ivacaftor

• DrugIVA

  Tablets for oral administration.

  Also known as: VX-770, Ivacaftor

• DrugPlacebo

  Placebos matched to VX-121, TEZ, and VX-561 for oral administration.

Primary outcomes

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

   Time frame: From Day 1 Through Safety Follow-up (up to Day 75 for Part 1 and up to Day 85 for Part 2)

2. Absolute Change 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 Through Day 29

Secondary outcomes

1. Absolute Change in Sweat Chloride (SwCl) Concentrations

   Sweat samples were collected using an approved collection device.

   Time frame: From Baseline Through Day 29

2. Absolute Change 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 Day 29

3. Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolite (M1-TEZ) and, VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561)

   Time frame: Pre-dose at Day 15 and Day 29

Baseline characteristics

Study documents

• Study protocol · May 9, 2019
• Statistical analysis plan · Aug 16, 2019

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