Study of Ataluren (PTC124) in Cystic Fibrosis

NCT ID: NCT02107859

Last Updated: 2020-03-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 61 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-05-23
• Study Completion Date: 2017-06-05

Brief Summary

The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ataluren

Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.

Group Type: EXPERIMENTAL

Ataluren

Intervention Type: DRUG

Ataluren will be administered per dose and schedule specified in the arm.

Interventions

Ataluren

Ataluren will be administered per dose and schedule specified in the arm.

Intervention Type: DRUG

Other Intervention Names

PTC124

Eligibility Criteria

Inclusion Criteria

• Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
• Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
• Body weight greater than or equal to (≥) 16 kilograms (kg).
• Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
• Confirmed laboratory values within the central laboratory ranges at screening.
• In male and female participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period.
• Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions.

Exclusion Criteria

• Chronic use of systemic tobramycin within 4 weeks prior to screening.
• Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
• 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 4 weeks prior to screening and randomization.
• Known hypersensitivity to any of the ingredients or excipients of the study drug.
• Exposure to another investigational drug within 4 weeks prior to screening.
• Treatment with intravenous antibiotics within 3 weeks prior to screening.
• History of solid organ or hematological transplantation.
• Ongoing immunosuppressive therapy (other than corticosteroids).
• Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
• Known portal hypertension.
• Pregnancy or breast-feeding.

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PTC Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Joseph McIntosh, MD

Role: STUDY_DIRECTOR

PTC Therapeutics, Inc.

Locations

University of Alabama-Birmingham

Birmingham, Alabama, United States

Miller Children's Hospital Long Beach

Long Beach, California, United States

Denver Children's Hospital

Aurora, Colorado, United States

Children's Hospital Chicago

Chicago, Illinois, United States

Children's Hospital Boston

Boston, Massachusetts, United States

Beth Israel Medical Center

New York, New York, United States

Rainbow Babies & Children's Hospital

Cleveland, Ohio, United States

Hôpital Universitaire des Enfants Reine Fabiola

Brussels, , Belgium

University Hospital Brussels

Brussels, , Belgium

University Hospital Leuven

Leuven, , Belgium

Hôpital Necker - Enfants Malades

Paris, , France

Hôpital des Enfants

Toulouse, , France

Hadassah University Hospital - Mount Scopus

Jerusalem, , Israel

Università La Sapienza

Roma, , Italy

Azienda Ospedaliera di Verona

Verona, , Italy

Hospital Universitario La Paz

Madrid, , Spain

Karolinska University Hospital, Huddinge

Stockholm, , Sweden

Countries

United States; Belgium; France; Israel; Italy; Spain; Sweden

References

Aslam AA, Sinha IP, Southern KW. Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2023 Mar 3;3(3):CD012040. doi: 10.1002/14651858.CD012040.pub3.

Reference Type: DERIVED

PMID: 36866921 (View on PubMed)

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Related Links

http://www.ptcbio.com

PTC Therapeutics, Inc. website

Other Identifiers

2013-005449-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PTC124-GD-023-CF

Identifier Type: -

Identifier Source: org_study_id