COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

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. Identifier: NCT03256968
Recruitment Status : Completed
First Posted : August 22, 2017
Results First Posted : March 26, 2020
Last Update Posted : March 26, 2020
Information provided by (Responsible Party):
Steven M Rowe, University of Alabama at Birmingham

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

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Ataluren Phase 4

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : January 27, 2017
Actual Primary Completion Date : December 1, 2018
Actual Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: ataluren administration
dose of the drug administered (mg/kg body weight)
Drug: Ataluren

Primary Outcome Measures :
  1. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03256968

Layout table for location information
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
Sponsors and Collaborators
University of Alabama at Birmingham
Layout table for investigator information
Principal Investigator: Steven M Rowe, MD University of Alabama at Birmingham
  Study Documents (Full-Text)

Documents provided by Steven M Rowe, University of Alabama at Birmingham:
Layout table for additonal information
Responsible Party: Steven M Rowe, Principal Investigator, University of Alabama at Birmingham Identifier: NCT03256968    
Other Study ID Numbers: F160106006
First Posted: August 22, 2017    Key Record Dates
Results First Posted: March 26, 2020
Last Update Posted: March 26, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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
Additional relevant MeSH terms:
Layout table for MeSH terms
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases