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PTC Study to Evaluate Ataluren in Combination With Ivacaftor

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03256968
Recruitment Status : Completed
First Posted : August 22, 2017
Results First Posted : March 26, 2020
Last Update Posted : March 26, 2020
Sponsor:
Information provided by (Responsible Party):
Steven M Rowe, University of Alabama at Birmingham

Tracking Information
First Submitted Date  ICMJE August 18, 2017
First Posted Date  ICMJE August 22, 2017
Results First Submitted Date  ICMJE December 13, 2019
Results First Posted Date  ICMJE March 26, 2020
Last Update Posted Date March 26, 2020
Actual Study Start Date  ICMJE January 27, 2017
Actual Primary Completion Date December 1, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 25, 2020)
FEV1 as a Measure of Lung Function [ Time Frame: 1 year ]
effect of ataluren on lung function as assessed by spirometry and measured by percentage of Liters (minimum value would be .0% Liters and maximum value of liters is dependent from person to person). The higher the value the better the outcome. The measure will include the change from baseline to one year to report a change between the two measurement points
Original Primary Outcome Measures  ICMJE
 (submitted: August 18, 2017)
  • lung function [ Time Frame: 1 year ]
    effect of ataluren on lung function as assessed by spirometry
  • health related quality of life [ Time Frame: 1 year ]
    effect of ataluren as assessed by the cystic fibrosis queastionaire revised
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE PTC Study to Evaluate Ataluren in Combination With Ivacaftor
Official Title  ICMJE An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor in Combination With Ataluren (PTC124) in Subjects With Nonsense Mutation Cystic Fibrosis
Brief Summary The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with nonsense mutation cystic fibrosis
Detailed Description Cystic Fibrosis (CF) is a life threatening genetic disorder resulting from mutations found in the CF gene known as the cystic fibrosis transmembrane conductance regulator or CFTR. This defect prevents correct chloride absorption in and out of the cells The purpose of this study is to explore the combination of ataluren and ivacaftor as a treatment for patients with a specific cystic fibrosis mutation
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: Ataluren
ataluren
Study Arms  ICMJE Experimental: ataluren administration
dose of the drug administered (mg/kg body weight)
Intervention: Drug: Ataluren
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 18, 2017)
1
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 31, 2018
Actual Primary Completion Date December 1, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial.
  2. Age ≥6 years
  3. Body weight ≥16 kg
  4. Diagnosis of cystic fibrosis and documentation of the presence of a nonsense mutations of the CFTR gene, as determined by historical genotyping
  5. Ability to perform a valid, reproducible spirometry with demonstration of a forced expiratory volume in 1second (FEV1) ≥30% and ≤90% of predicted for age, gender, and height.
  6. If the subject is sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration
  7. Willingness and ability to comply with all study procedures and assessments.
  8. Currently being administered ivacaftor, either alone (Kalydeco) or in combination with lumacaftor (Orkambi)

Exclusion Criteria:

  1. Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 2 weeks prior to screening.
  2. Ongoing participation in any other therapeutic clinical trial.
  3. Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 2 weeks prior to screening.
  4. Ongoing inhaled tobramycin therapy.
  5. Ongoing immunosuppressive therapy (other than corticosteroids up to 10mg/d equivalent of prednisone)
  6. Ongoing warfarin, phenytoin, or tolbutamide therapy.
  7. History of solid organ or hematological transplantation.
  8. A history of positive hepatitis B surface antigen test, hepatitis C antibody test, or human immunodeficiency
  9. Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 4 weeks prior to screening.
  10. Pregnancy or breast-feeding.
  11. Current smoker or a smoking history of ≥10 pack-years (number of cigarette packs/day × number of years smoked).
  12. Prior or ongoing medical condition (eg, renal failure, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.

    -

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03256968
Other Study ID Numbers  ICMJE F160106006
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Steven M Rowe, University of Alabama at Birmingham
Study Sponsor  ICMJE University of Alabama at Birmingham
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Steven M Rowe, MD University of Alabama at Birmingham
PRS Account University of Alabama at Birmingham
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP