Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

NCT02456103 | Terminated Phase 3 Results DMC

• 1 other identifier: PTC124-GD-021e-CF
• Study Type: interventional
• Target: 246
• Locations: 16 countries
• Sites: 70

Brief Summary

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs)

  TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.

  Baseline up to Week 100

• Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter

  Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.

  Baseline up to Week 100

Secondary Outcomes (4)

• Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24

  Baseline, Week 24

• Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24

  Baseline, Week 24

• Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24

  Baseline, Week 24

• Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks

  Baseline up to Week 48

Study Arms (1)

Ataluren

EXPERIMENTAL

Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.

Drug: Ataluren

Interventions

Ataluren DRUG

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

Also known as: PTC124, Translarna

Ataluren

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:

• Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
• Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

You may not qualify if:

• Known hypersensitivity to any of the ingredients or excipients of the study drug.
• Ongoing participation in any other therapeutic clinical trial.

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (70)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

Miller Children's Hospital Long Beach

Long Beach, California, 90806, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Children's Hospital and Research Center at Oakland

Oakland, California, 94609, United States

Location

Stanford University-Children's Hospital

Palo Alto, California, 94304, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Miami Children's Hospital

Miami, Florida, 33155, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Morristown Medical Center

Morristown, New Jersey, 07960, United States

Location

Beth Israel Medical Center

New York, New York, 10011, United States

Location

New York University Langone Medical Center

New York, New York, 10016, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45221, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, 19129, United States

Location

Texas Children's Hospital

Houston, Texas, 77094, United States

Location

University of Texas Health Science Center

Tyler, Texas, 75708, United States

Location

Childrens Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Hospital de Ninos Ricardo Gutierrez

Buenos Aires, 1330, Argentina

Location

Hospital Universitario Austral

Buenos Aires, Argentina

Location

Royal Adelaide Hospital

Adelaide, 5000, Australia

Location

Prince Charles Hospital

Chermside, 4032, Australia

Location

Princess Margaret Hospital

Perth, 6840, Australia

Location

University Hospital Brussels

Brussels, 1090, Belgium

Location

Hopital Universitaire des Enfants Reine Fabiola

Brussels, 15, Belgium

Location

University Hospital Leuven

Leuven, 3000, Belgium

Location

Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul

Porto Alegre, Brazil

Location

University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD

Plovdiv, Bulgaria

Location

University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

Sofia, Bulgaria

Location

Clinical Research Institute of Montreal

Montreal, H2W 1R7, Canada

Location

University of Toronto Hospital for Sick Children

Toronto, M5G 1X8, Canada

Location

British Columbia Children's Hospital

Vancouver, V6H 3V4, Canada

Location

Hoptial Arnaud de Villeneuve

Montpellier, 34295, France

Location

Hopital Necker - Enfants Malades

Paris, 75015, France

Location

Centre de Perharidy

Roscoff, 29684, France

Location

Centre Hospitalier Regional Sud Reunion

Saint-Pierre, 97448, France

Location

Charite-Universitatsmedizin Berlin

Berlin, 10117, Germany

Location

St. Josef Hospital GmbH

Bochum, 44791, Germany

Location

University of Cologne Children's Hospital

Cologne, 50937, Germany

Location

Christiane Herzog CF-Zentrum

Frankfurt am Main, 60590, Germany

Location

Universitatsklinikum Jena

Jena, 1, Germany

Location

LMU Klinikum der Universitat Muchen

Munich, 80539, Germany

Location

Dr. Von Haunersches Kinderspital

München, 80337, Germany

Location

Ippokratio General Hospital Of Thessaloniki

Thessaloniki, 541, Greece

Location

Meyer Children's Hospital

Haifa, 31096, Israel

Location

Hadassah University Hospital

Jerusalem, 91240, Israel

Location

Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi

Ancona, 71, Italy

Location

Azienda Ospedaliera A Meyer

Florence, 50139, Italy

Location

Lombardia Cystic Fibrosis Center

Milan, 20122, Italy

Location

Azienda Policlinico Umberto I

Rome, 00161, Italy

Location

Ospedale Pediatrico Bambino Gesu

Rome, 4, Italy

Location

Azienda Ospedaliera di Verona

Verona, Italy

Location

Hagaziekenhuis

s-Gravenweg, South Holland, 2545 CH, Netherlands

Location

Radboud University

Nijmegen, 6525 GA, Netherlands

Location

Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku

Gdansk, Poland

Location

Institute of Mother and Child

Warsaw, 01-211, Poland

Location

Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital University

Barcelona, 08035, Spain

Location

Hospital San Juan

Esplugues de Llobregat, 08950, Spain

Location

Hospital Regional Universitario de Malaga

Málaga, 29001, Spain

Location

Hospital de Sabadell, Consorci Sanitari Parc Tauli

Sabadell, 08208, Spain

Location

Hospital Universitario Virgen del Rocio

Seville, 41013, Spain

Location

St James University Hospital

Leeds, LS9 7TF, United Kingdom

Location

Royal Brompton Hospital

London, SW3 6NP, United Kingdom

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

Limitations and Caveats

This study was terminated early because the CF data from the double-blind CF Study PTC124-GD-021-CF (NCT02139306) did not meet endpoints.

Results Point of Contact

• Title: Patient Advocacy
• Organization: PTC Therapeutics, Inc.

medinfo@ptcbio.com

1-866-562-4620

Study Officials

• Joseph McIntosh, MD

  PTC Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2015
• First Posted: May 28, 2015
• Study Start: August 31, 2015
• Primary Completion: June 2, 2017
• Study Completion: June 2, 2017
• Last Updated: April 27, 2020
• Results First Posted: April 15, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (6); BE (3); IT (6); BR (1); US (24); BU (2); GR (1); CA (3); AR (2); PL (2); DE (7); GB (2); IL (2); AU (3); FR (4); NL (2)