SNPMiner Trials by Shray Alag


SNPMiner SNPMiner Trials (Home Page)


Report for Clinical Trial NCT01784419

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. However, it is highly likely that CF patients with many other mutations can benefit similarly from this drug, some of whom can be identified by phenotype or genotype. We will enroll up to 30 CF subjects with clinical presentations in which there is one or more signs of residual CF channel function. The signs of residual function include: normal digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF disease in a CF patient with severe gene mutations. The primary outcome measure will be the difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor. Secondary outcome measured will be lung function.

NCT01784419 Cystic Fibrosis
MeSH: Cystic Fibrosis Fibrosis

1 Interventions

Name: ivacaftor

Description: Prospective randomized double-blinded placebo controlled crossover study of the effects of ivacaftor in selected cystic fibrosis patients

Type: Drug

ivacaftor-placebo placebo-ivacaftor


Primary Outcomes

Description: Sweat chloride concentration measured by pilocarpine iontophoresis, a standard clinical laboratory technique. Sweat collection accomplished with the Wescor Macroduct system. Sweat chloride is measured at the start and end of each study period. There are two study periods during which subjects take either ivacaftor or placebo.

Measure: Sweat chloride concentration

Time: 14 +/- 2 days

Secondary Outcomes

Description: Standard spirometry will be performed at the start and end of each 2 week study period. Subjects will take study drug (ivacaftor or placebo during each study period.

Measure: Spirometry

Time: 14 +/- 2 days

Description: Subjects will perform multibreath washout testing using standard techniques to measure functional residual capacity and lung clearance index at the beginning and end of each study period.

Measure: Multibreath washout testing

Time: 14 +/- 2 days

Purpose: Screening

Allocation: Randomized

Crossover Assignment


There is one SNP

SNPs


1 G551D

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients. --- G551D ---

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. --- G551D ---

This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. --- G551D ---

It is currently FDA approved for use only in CF patients with the G551D gating mutation. --- G551D ---

Based on results from previous studies, we hypothesize that non-G551D patients with signs of residual CF channel activity might respond favorably to treatment with ivacaftor. --- G551D ---

This includes patients with any of the non-G551D gating mutations, that are pancreatic sufficient, that have a sweat chloride concentration between 55 and 85 mmol, or that are much healthier than expected. --- G551D ---



HPO Nodes