Clinical trial NCT02456103: Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
Official title: Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: PTC Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-05-28
Enrollment: 246
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed description: The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

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

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: Baseline up to Week 100
measure: Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter
timeFrame: Baseline up to Week 100
Secondary outcomes:
measure: Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
timeFrame: Baseline, Week 24
measure: Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24
timeFrame: Baseline, Week 24
measure: Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
timeFrame: Baseline up to Week 48
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Long Beach
country: United States
facility: Miller Children's Hospital Long Beach
state: California
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Oakland
country: United States
facility: Children's Hospital and Research Center at Oakland
state: California
city: Palo Alto
country: United States
facility: Stanford University-Children's Hospital
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Miami
country: United States
facility: Miami Children's Hospital
state: Florida
city: Saint Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Boston
country: United States
facility: Children's Hospital Boston
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Morristown
country: United States
facility: Morristown Medical Center
state: New Jersey
city: New York
country: United States
facility: Beth Israel Medical Center
state: New York
city: New York
country: United States
facility: New York University Langone Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel University College of Medicine
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Tyler
country: United States
facility: University of Texas Health Science Center
state: Texas
city: Milwaukee
country: United States
facility: Childrens Hospital of Wisconsin
state: Wisconsin
city: Buenos Aires
country: Argentina
facility: Hospital de Ninos Ricardo Gutierrez
city: Buenos Aires
country: Argentina
facility: Hospital Universitario Austral
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Chermside
country: Australia
facility: Prince Charles Hospital
city: Perth
country: Australia
facility: Princess Margaret Hospital
city: Brussels
country: Belgium
facility: University Hospital Brussels
city: Brussels
country: Belgium
facility: Hopital Universitaire des Enfants Reine Fabiola
city: Leuven
country: Belgium
facility: University Hospital Leuven
city: Porto Alegre
country: Brazil
facility: Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul
city: Plovdiv
country: Bulgaria
facility: University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
city: Montreal
country: Canada
facility: Clinical Research Institute of Montreal
city: Toronto
country: Canada
facility: University of Toronto Hospital for Sick Children
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Montpellier
country: France
facility: Hoptial Arnaud de Villeneuve
city: Paris
country: France
facility: Hopital Necker - Enfants Malades
city: Roscoff
country: France
facility: Centre de Perharidy
city: Saint-Pierre
country: France
facility: Centre Hospitalier Regional Sud Reunion
city: Berlin
country: Germany
facility: Charite-Universitatsmedizin Berlin
city: Bochum
country: Germany
facility: St. Josef Hospital GmbH
city: Cologne
country: Germany
facility: University of Cologne Children's Hospital
city: Frankfurt am Main
country: Germany
facility: Christiane Herzog CF-Zentrum
city: Jena
country: Germany
facility: Universitatsklinikum Jena
city: Munchen
country: Germany
facility: Dr. Von Haunersches Kinderspital
city: Munich
country: Germany
facility: LMU Klinikum der Universitat Muchen
city: Thessaloniki
country: Greece
facility: Ippokratio General Hospital Of Thessaloniki
city: Haifa
country: Israel
facility: Meyer Children's Hospital
city: Jerusalem
country: Israel
facility: Hadassah University Hospital
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi
city: Firenze
country: Italy
facility: Azienda Ospedaliera A Meyer
city: Milan
country: Italy
facility: Lombardia Cystic Fibrosis Center
city: Rome
country: Italy
facility: Azienda Policlinico Umberto I
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Verona
country: Italy
facility: Azienda Ospedaliera di Verona
city: s-Gravenweg
country: Netherlands
facility: Hagaziekenhuis
state: Zuid-Holland
city: Nijmegen
country: Netherlands
facility: Radboud University
city: Gdansk
country: Poland
facility: Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku
city: Warsaw
country: Poland
facility: Institute of Mother and Child
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital University
city: Esplugues De Llobregat
country: Spain
facility: Hospital San Juan
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga
city: Sabadell
country: Spain
facility: Hospital de Sabadell, Consorci Sanitari Parc Tauli
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Leeds
country: United Kingdom
facility: St James University Hospital
city: London
country: United Kingdom
facility: Royal Brompton Hospital
Study officials:
affiliation: PTC Therapeutics
name: Joseph McIntosh, MD
role: STUDY_DIRECTOR
References: