Clinical Trials Logo
  1. Home
  2. Cystic Fibrosis
  3. NCT00237380

Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:
A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis

Clinical Trial Summary

In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase functional CFTR protein in the cells of participants with CF due to a nonsense mutation.

Clinical Trial Description

In this study, participants with CF due to a nonsense mutation will be treated with a new investigational drug called ataluren. Evaluation procedures to determine if a participant qualifies for the study will be performed within 21 days prior to the start of treatment. Eligible participants who elect to enroll in the study will then participate in two 28-day treatment and follow-up periods (56 days total). There will be a 2-night stay at the clinical research center at the beginning and at the end of each 14 days of ataluren treatment, which means that there will be four 2-night stays at the clinical research center during the study.

One of the measurements for the study is transepithelial potential difference (TEPD), which is also known as nasal potential difference and provides a sensitive evaluation of sodium and chloride transport directly in secretory epithelial cells. TEPD assessments are made on the nasal epithelium cells lining the inferior turbinate because these cells are easier to access than the respiratory epithelial cells lining the lower airways and have been shown to have the same ion transport characteristics. As an endpoint, TEPD has the advantage that it can detect chloride transport changes that are a quantitative integration of the presence, functional activity, and apical location of the CFTR in airway cells. Furthermore, it is a direct measure of CFTR activity that is not likely to be affected by supportive or palliative treatments for CF (with the possible exception of systemically administered aminoglycoside antibiotics). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00237380
Study type Interventional
Source PTC Therapeutics
Contact
Status Completed
Phase Phase 2
Start date November 30, 2005
Completion date May 31, 2006
View Details
See also
  Status Clinical Trial Phase
Completed NCT04696198 - Thoracic Mobility in Cystic Fibrosis Care N/A
Completed NCT00803205 - Study of Ataluren (PTC124™) in Cystic Fibrosis Phase 3
Terminated NCT04921332 - Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD N/A
Completed NCT03601637 - Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del Phase 3
Terminated NCT02769637 - Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
Recruiting NCT06030206 - Lung Transplant READY CF 2: A Multi-site RCT N/A
Recruiting NCT06012084 - The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis N/A
Recruiting NCT06032273 - Lung Transplant READY CF 2: CARING CF Ancillary RCT N/A
Recruiting NCT06088485 - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting NCT05392855 - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF) N/A
Recruiting NCT04039087 - Sildenafil Exercise: Role of PDE5 Inhibition Phase 2/Phase 3
Recruiting NCT04056702 - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Completed NCT04038710 - Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
Completed NCT04058548 - Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation N/A
Completed NCT03637504 - Feasibility of a Mobile Medication Plan Application in CF Patient Care N/A
Recruiting NCT03506061 - Trikafta in Cystic Fibrosis Patients Phase 2
Completed NCT03566550 - Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
Recruiting NCT04828382 - Prospective Study of Pregnancy in Women With Cystic Fibrosis
Completed NCT04568980 - Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
Recruiting NCT04010253 - Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis N/A