A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes

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 120 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

  Participants 6 to less than \<12 year of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg /IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. Doses were adjusted upward with subsequent changes in weight. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks.

  Drug: ELX/TEZ/IVA · Drug: IVA

Interventions

• DrugELX/TEZ/IVA

  Fixed dose combination (FDC) tablets 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. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

   Time frame: From Baseline up to Week 100

Secondary outcomes

1. Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)

   Sweat samples were collected using an approved collection device.

   Time frame: From Parent Study Baseline to Week 96

2. Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5)

   The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.

   Time frame: From Parent Study Baseline to 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 · Mar 22, 2023

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