A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation (VX15-770-124)

Details

Therapeutic approach: Restore CFTR Function
Trial status: Closed with results Participating Centres
Trials Tracker ID: TT001611
Last updated: 22/02/2019

Full title

A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who are Less than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

Study details

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation

Study results

This study evaluated the safety and effectiveness of Ivacaftor in patients aged 24 months or younger with at least one CFTR gating mutation.

This study took place in two parts. In Part A, 19 children received Ivacaftor treatment for 3 ½ days. In Part B, 43 children received the treatment for 24 weeks.

Ivacaftor was generally safe and well tolerated in children aged 4 to 24 months. Treatment for up to 24 weeks was associated with reductions in sweat chloride and improvements in the function of the pancreas. All adverse events reported were considered mild or moderate and likely not related to Ivacaftor treatment.

These results support Ivacaftor as a treatment for the underlying cause of cystic fibrosis in children ages 4 - 24 months with one or more gating mutation.

Link to full results https://clinicaltrials.gov/study/NCT02725567

Phase The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use: Phase 3
Recruitment target: 35
CF sponsor: Vertex Pharmaceuticals Incorporated
CF sponsor type: Commercial

Who can take part?

Age range

Between 0 and 24 months

Including people

Age 0 - 24 months

Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D

Excluding people

History of solid organ or hematological transplantation

History of abnormal liver function or abnormal liver function at screening

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A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation (VX15-770-124)

Details

A Phase 3, 2 Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who are Less than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

Who can take part?

Your donation will make a difference:

CF centres running this trial

Edinburgh - Royal Hospital for Children and Young People

Great Ormond Street Hospital for Children (London)

Royal Brompton Hospital (London)

Royal Manchester Children's Hospital (Paediatrics)