Extension Study of Ataluren (PTC124) in Cystic Fibrosis

NCT ID: NCT01140451

Last Updated: 2020-10-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 191 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-08-12
• Study Completion Date: 2013-12-02

Brief Summary

Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients with the disease. Ataluren is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. Funding source for this study is the FDA OOPD.

Detailed Description

This Phase 3, open-label, safety and efficacy study will be performed at sites in North America, Europe, and Israel. The study will enroll up to approximately 208 participants with nmCF who participated in a previous Phase 3 study of ataluren (PTC124-GD-009-CF [Study 009], NCT00803205). Participants will receive study drug 3 times per day (TID) (at breakfast, lunch, and dinner) for approximately 48 weeks (approximately 1 year). Study assessments will be performed at clinic visits every 8 weeks.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ataluren/Ataluren

Participants who received double-blind ataluren during Study 009 will continue to receive open-label ataluren 3 times per day TID: 10 milligram (mg)/kilogram (kg) of body weight with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner (total dose 40 mg/kg/day), for up to 96 weeks. Participants will be followed for 4 weeks after treatment.

Group Type: EXPERIMENTAL

Ataluren

Intervention Type: DRUG

Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Placebo/Ataluren

Participants who received double-blind placebo during Study 009 will receive open-label ataluren TID: 10 mg/kg of body weight with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner (total dose 40 mg/kg/day), for up to 96 weeks. Participants will be followed for 4 weeks after treatment.

Group Type: EXPERIMENTAL

Ataluren

Intervention Type: DRUG

Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Interventions

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Intervention Type: DRUG

Other Intervention Names

PTC124

Eligibility Criteria

Inclusion Criteria

• Completion of blinded study drug treatment in the previous Phase 3 study (PTC124-GD-009-CF).
• Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).
• In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 4-week follow up period.
• Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria

• Known hypersensitivity to any of the ingredients or excipients of the study drug (list provided at study sites).
• Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3 study.
• Ongoing participation in any other therapeutic clinical trial.
• Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.

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

Sponsors

Cystic Fibrosis Foundation

OTHER

Sponsor Role: collaborator

PTC Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Temitayo Ajayi, 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

Lucile Packard Children's Hospital

Palo Alto, California, United States

The Children's Hospital

Aurora, Colorado, United States

University of Miami

Miami, Florida, United States

Emory University Cystic Fibrosis Center

Atlanta, Georgia, United States

Children's Memorial Hospital

Chicago, Illinois, United States

Johns Hopkins Children's Center

Baltimore, Maryland, United States

Children's Hospital Boston

Boston, Massachusetts, United States

Washington University

St Louis, Missouri, United States

St. Vincent's Hospital

New York, New York, United States

New York Medical College

Valhalla, New York, United States

University of North Carolina

Chapel Hill, North Carolina, United States

Rainbow Babies & Children's Hospital

Cleveland, Ohio, United States

Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Hôpital Erasme

Brussels, , Belgium

Hôpital Universitaire des Enfants Reine Fabiola

Brussels, , Belgium

University Hospital Brussels

Brussels, , Belgium

University Hospital Leuven

Leuven, , Belgium

University of Toronto

Toronto, , Canada

Hôpital Cochin

Paris, , France

Hôpital Necker - Enfants Malades

Paris, , France

Hôpital des Enfants

Toulouse, , France

Klinikum der Universität Köln

Cologne, , Germany

Hadassah University Hospital - Mount Scopus

Jerusalem, , Israel

Università La Sapienza

Roma, , Italy

Azienda Ospedaliera di Verona

Verona, , Italy

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Hospital Universitario La Paz

Madrid, , Spain

Karolinska University Hospital, Huddinge

Stockholm, , Sweden

Belfast City Hospital

Belfast, , United Kingdom

Countries

United States; Belgium; Canada; France; Germany; Israel; Italy; Netherlands; Spain; Sweden; United Kingdom

Related Links

http://www.ptcbio.com

PTC Therapeutics, Inc. website

Other Identifiers

Orphan Product Grant #FD003715

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2010-019692-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PTC124-GD-009e-CF

Identifier Type: -

Identifier Source: org_study_id