PTC Study to Evaluate Ataluren in Combination With Ivacaftor

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03256968
• Date of registration: 18/08/2017
• Prospective Registration: No
• Primary sponsor: University of Alabama at Birmingham
• Public title: PTC Study to Evaluate Ataluren in Combination With Ivacaftor
• Scientific 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
• Date of first enrolment: January 27, 2017
• Target sample size: 1
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03256968
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

United States

Contacts

• Name: Steven M Rowe, MD
• Affiliation: University of Alabama at Birmingham

Key inclusion & exclusion criteria

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.

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Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ataluren

Primary Outcome(s)

FEV1 as a Measure of Lung Function [Time Frame: 1 year]

Secondary ID(s)

F160106006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 26/03/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03256968