A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

NCT03068312 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: VX16-770-1272017-000457-39
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the efficacy of ivacaftor treatment in subjects with CF 6 years of age and older who have a 3849 + 10KB C→T or D1152H CFTR mutation.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Change in Lung Clearance Index 2.5 (LCI2.5)

  LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

  From baseline through 8 weeks

Study Arms (2)

Sequence 1: First Ivacaftor (IVA) Then Placebo

EXPERIMENTAL

Participants received IVA 150 milligram (mg) every 12 hours (q12h) for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

Drug: Ivacaftor; Drug: Placebo

Sequence 2: First Placebo Then IVA

EXPERIMENTAL

Participants received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA 150 mg q12h for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

Drug: Ivacaftor; Drug: Placebo

Interventions

Ivacaftor DRUG

IVA 150 mg tablet.

Also known as: VX-770, IVA

Sequence 1: First Ivacaftor (IVA) Then Placebo; Sequence 2: First Placebo Then IVA

Placebo DRUG

Placebo matched to IVA tablet.

Sequence 1: First Ivacaftor (IVA) Then Placebo; Sequence 2: First Placebo Then IVA

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of CF based on protocol-specified clinical features and at least one of the following: increased sweat chloride level, identification of 2 CF causing mutations, or demonstration of abnormal nasal epithelial ion transport.
• A 3849 + 10KB C→T or D1152H mutation on at least 1 CFTR allele.
• FEV1 ≥40% of predicted and ≤105% of predicted at screening.

You may not qualify if:

• A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation.
• History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
• Ongoing or prior participation in an investigational drug study within 30 days before the Screening Visit.
• Protocol-specified abnormal laboratory values at the Screening Visit
• For subjects \<18 years of age at the Screening Visit, evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination (OE) at the Screening Visit.
• Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1.
• Pregnant, breastfeeding, or planning to become pregnant during the study.
• Sexually active subjects of reproductive potential must be willing to use appropriate contraception.

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (1)

Hadassah Medical Organization

Jerusalem, Israel

Location

Related Publications (1)

• Kerem E, Cohen-Cymberknoh M, Tsabari R, Wilschanski M, Reiter J, Shoseyov D, Gileles-Hillel A, Pugatsch T, Davies JC, Short C, Saunders C, DeSouza C, Sullivan JC, Doyle JR, Chandarana K, Kinnman N. Ivacaftor in People with Cystic Fibrosis and a 3849+10kb C-->T or D1152H Residual Function Mutation. Ann Am Thorac Soc. 2021 Mar;18(3):433-441. doi: 10.1513/AnnalsATS.202006-659OC.

  PMID: 33095038 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

+1 617 341 6777

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 24, 2017
• First Posted: March 1, 2017
• Study Start: July 18, 2017
• Primary Completion: December 18, 2018
• Study Completion: December 18, 2018
• Last Updated: February 26, 2020
• Results First Posted: January 6, 2020

Record last verified: 2020-02

Locations

IL (1)