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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

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Intervention Drug: Ataluren
Enrollment 1
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ataluren Administration
Hide Arm/Group Description

dose of the drug administered (mg/kg body weight)

Ataluren: ataluren

Period Title: Overall Study
Started 1
Completed 1
Not Completed 0
Arm/Group Title Ataluren Administration
Hide Arm/Group Description

dose of the drug administered (mg/kg body weight)

Ataluren: ataluren

Overall Number of Baseline Participants 1
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
<=18 years
0
   0.0%
Between 18 and 65 years
1
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Inter-Quartile Range)
Unit of measure:  Years
Number Analyzed 1 participants
31
(31 to 31)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
Female
1
 100.0%
Male
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
1
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 1 participants
1
1.Primary Outcome
Title FEV1 as a Measure of Lung Function
Hide Description 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
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Ataluren Administration
Hide Arm/Group Description:

dose of the drug administered (mg/kg body weight)

Ataluren: ataluren

Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: percentage of liters
.35
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ataluren Administration
Hide Arm/Group Description

dose of the drug administered (mg/kg body weight)

Ataluren: ataluren

All-Cause Mortality
Ataluren Administration
Affected / at Risk (%)
Total   0/1 (0.00%)    
Hide Serious Adverse Events
Ataluren Administration
Affected / at Risk (%) # Events
Total   0/1 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ataluren Administration
Affected / at Risk (%) # Events
Total   1/1 (100.00%)    
Respiratory, thoracic and mediastinal disorders   
increased cough   1/1 (100.00%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Steven Rowe
Organization: Universtiy of Alabama at Birmingham
Phone: 205-975-9776
EMail: srowe@uabmc.edi
Layout table for additonal information
Responsible Party: Steven M Rowe, University of Alabama at Birmingham
ClinicalTrials.gov Identifier: NCT03256968    
Other Study ID Numbers: F160106006
First Submitted: August 18, 2017
First Posted: August 22, 2017
Results First Submitted: December 13, 2019
Results First Posted: March 26, 2020
Last Update Posted: March 26, 2020