PTC Study to Evaluate Ataluren in Combination With Ivacaftor

NCT03256968 | Completed Phase 4 Results FDA Drug

• 1 other identifier: F160106006
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• FEV1 as a Measure of Lung Function

  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

  1 year

Study Arms (1)

ataluren administration

EXPERIMENTAL

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

Drug: Ataluren

Interventions

Ataluren DRUG

ataluren

ataluren administration

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Age ≥6 years
• Body weight ≥16 kg
• Diagnosis of cystic fibrosis and documentation of the presence of a nonsense mutations of the CFTR gene, as determined by historical genotyping
• 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.
• 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
• Willingness and ability to comply with all study procedures and assessments.
• Currently being administered ivacaftor, either alone (Kalydeco) or in combination with lumacaftor (Orkambi)

You may not qualify if:

• 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.
• Ongoing participation in any other therapeutic clinical trial.
• Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 2 weeks prior to screening.
• Ongoing inhaled tobramycin therapy.
• Ongoing immunosuppressive therapy (other than corticosteroids up to 10mg/d equivalent of prednisone)
• Ongoing warfarin, phenytoin, or tolbutamide therapy.
• History of solid organ or hematological transplantation.
• A history of positive hepatitis B surface antigen test, hepatitis C antibody test, or human immunodeficiency
• Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 4 weeks prior to screening.
• Pregnancy or breast-feeding.
• Current smoker or a smoking history of ≥10 pack-years (number of cigarette packs/day × number of years smoked).
• 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.

Sponsors & Collaborators

• University of Alabama at Birmingham: lead

Study Sites (1)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ataluren

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Steven Rowe
• Organization: Universtiy of Alabama at Birmingham

srowe@uabmc.edi

205-975-9776

Study Officials

• Steven M Rowe, MD

  University of Alabama at Birmingham

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: August 18, 2017
• First Posted: August 22, 2017
• Study Start: January 27, 2017
• Primary Completion: December 1, 2018
• Study Completion: December 31, 2018
• Last Updated: March 26, 2020
• Results First Posted: March 26, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)