Clinical trial NCT01338415: FUTURE 3 Study Extension
Official title: A Prospective, Multicenter, Open-label Extension of FUTURE 3 to Assess the Safety, Tolerability and Efficacy of the Pediatric Formulation of Bosentan Two Versus Three Times a Day in Children With Pulmonary Arterial Hypertension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pulmonary Arterial Hypertension
Keywords: pediatric
pulmonary arterial hypertension
bosentan
Tracleer
Lead sponsor: Actelion
Sponsor class: INDUSTRY
First posted: 2011-04-19
Enrollment: 58
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objectives of the FUTURE 3 Study Extension are to evaluate the long-term safety, tolerability and efficacy of the pediatric formulation of bosentan two versus three times a day in children with Pulmonary Arterial Hypertension (PAH).


Eligibility criteria: Inclusion Criteria:

1. Patients who completed the FUTURE 3 core study (AC-052-373) or prematurely discontinued due to PAH-progression, if bosentan was not permanently discontinued
2. Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial at the end of the FUTURE 3 core study (AC-052-373)
3. Signed informed consent by the parents or the legal representatives prior to any study-mandated procedure.

Exclusion Criteria:

1. Known intolerance or hypersensitivity to bosentan or any of the excipients of the dispersible bosentan tablet
2. Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy
3. Pregnancy
4. AST and/or ALT values \> 3 times the upper limit of normal range (ULN)
5. Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C
6. Premature and permanent study drug discontinuation during the FUTURE 3 core study (AC-052-373)
7. Any major violation of the FUTURE 3 core study (AC-052-373) protocol.

Primary outcomes:
measure: Treatment Emergent Adverse Events (AEs) up to 7 Days After Permanent Study Drug Discontinuation
timeFrame: Up to 62 weeks in average
Secondary outcomes:
Other outcomes:
measure: Change From Baseline up to 12 Months of Study Treatment in the World Health Organization Functional Classification (WHO FC)
timeFrame: At Month 12
measure: Change From Baseline up to 18 Months of Study Treatment in the World Health Organization Functional Classification (WHO FC)
timeFrame: At Month 18
measure: Change From Baseline up to 12 Months of Study Treatment in the Global Clinical Impression Scale (GCIS)
timeFrame: At Month 12
measure: Change From Baseline up to 18 Months of Study Treatment in the Global Clinical Impression Scale (GCIS)
timeFrame: At Month 18
measure: Number of Patients With Pulmonary Arterial Hypertension (PAH) Worsening Components up to the Last Day of Treatment + 7 Days
timeFrame: Up to 62 weeks in average
measure: Pulmonary Arterial Hypertension (PAH) Progression up to End of Treatment + 7 Days
timeFrame: From baseline to Month 18
measure: Overall Survival
timeFrame: From baseline to month 18
measure: Exceptional Use Treatment Period (EUTP): Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) up to 7 Days After Permanent Discontinuation of Study Drug
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Treatment-emergent Serious Adverse Events (SAEs) up to 7 Days After Permanent Discontinuation of Study Drug
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With AEs Leading to Premature Discontinuation of Study Drug
timeFrame: Up to 3 years and 4 months
measure: EUTP: Percentage of Participants With SAEs From 7 up to 60 Days After Permanent Discontinuation of Study Drug
timeFrame: From 7 up to 60 days after permanent discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Deaths
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Adverse Events of Special Interest (AESI)
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Treatment-emergent Marked Laboratory Abnormalities up to 7 Days After Permanent Discontinuation of Study Drug
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Liver Function Abnormalities
timeFrame: Up to 7 days after discontinuation of study drug (up to 3 years and 4 months)
measure: EUTP: Percentage of Participants With Any Time Occurrence of Hemoglobin <=10 Gram Per Deciliter (g/dL) and <=8g/dL Between Baseline and up to 7 Days After End of Treatment (EOT)
timeFrame: Baseline, up to 7 days after end of treatment (up to 3 years and 4 months)
Locations:
city: Aurora
country: United States
facility: The Children's Hospital - Site 9102
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center - Site 9104
state: District of Columbia
city: New York
country: United States
facility: Columbia University Medical Center Children's Hospital of New York Presbyterian - Site 9101
state: New York
city: Parkville
country: Australia
facility: Royal Children's Hospital Melbourne, Cardiology - Site 5001
city: Minsk
country: Belarus
facility: The Republican Scientific-Practical Center "Cardiology" - Site 3001
city: Beijing
country: China
facility: Cardiovascular Institute and Fuwai Hospital
city: Shanghai
country: China
facility: Shanghai Children's Medical Center - Site 5102
city: Prague
country: Czechia
facility: Fakultní nemocnice v Motole, dětské kardiocentrum - Site 3301
city: Paris
country: France
facility: Hopital Necker-Enfants Malades, Service de Cardiologie Pédiatrique - Site 2201
city: Toulouse
country: France
facility: CHU de Toulouse - Hôpital des Enfants, Service de Cardiologie Pédiatrique - Site 2202
city: Berlin
country: Germany
facility: Deutsches Herzzentrum Kinderkardiologie - Site 1401
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn Abteilung für Kinderkardiologie - Site 1404
city: Giessen
country: Germany
facility: Justus-Liebig-Universität Giessen, Kinderherzzentrum - Site 1403
city: Budapest
country: Hungary
facility: Gottsegen György Országos Kardiológiai Intézet, Gyermekszív Központ, Gyermek Kardiológiai osztály - Site 3401
city: Szeged
country: Hungary
facility: Szegedi Tudományegyetem ÁOK Szent-Györgyi Albert Klinikai Központ, Gyermekgyógyászati Klinika és Gyermekegészségügyi Központ - Site 3402
city: Hyderabad
country: India
facility: CARE Hospitals, Cardiology Dep. Hyderabad - Site 5302
city: Petach Tikvah
country: Israel
facility: Schneider Children's Medical Center- Institute of pediatric cardiology - Site 7101
city: Padova
country: Italy
facility: Università Degli Studi di Padova - Dipartimento di Pediatria - Servizio di Cardiologia Pediatrica - Site 1501
city: Rome
country: Italy
facility: Ospedale Pediatrico "Bambino Gesù" - Dipartimento Medico Chirurgico di Cardiologia Pediatrica - Site 1502
city: Mexico City
country: Mexico
facility: Instituto Nacional de Cardiologia (INC) Ignacio Chavez - Site 8401
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca - Site 3604
city: Wroclaw
country: Poland
facility: Wojewódzki Szpital Specjalistyczny we Wrocławiu Oddział Kardiologii Dziecięcej z pododdziałem Intensywnego Nadzoru Kardiologicznego - Site 3605
city: Kemerovo
country: Russian Federation
facility: RAMS Institution, Research Institute for complex issues of cardiovascular diseases, Siberian branch of the Russian Academy of Medical Sciences - Site 3805
city: Moscow
country: Russian Federation
facility: Scientific Center of Cardiovascular Surgery named after A.N.Bakulev of the RAMS - Site 3803
city: Moscow
country: Russian Federation
facility: Moscow Scientific Research Institute for Pediatrics and Childrens Surgery of Rosmedtechnologies - Site 3804
city: St. Petersberg
country: Russian Federation
facility: Federal State Institution "Federal center of Heart, Blood and Endocrinology named after V.A.Almazov Rosmedtekhnologies" - Site 3802
city: St. Petersburg
country: Russian Federation
facility: State Educational Institution of Higher Professional Education "Saint Petersburg State Pediatric Medical Academy of Roszdrav" - Site 3801
city: Belgrade
country: Serbia
facility: Univerzitetska dečja klinika, Služba za kardiologiju - Site 3901
city: Belgrade
country: Serbia
facility: Institut za zdravstvenu zaštitu majke i deteta Srbije "Dr Vukan Čupić", Služba za ispitivanje i lečenje bolesti srca i krvnih sudova - Site 3902
city: Bloemfontein
country: South Africa
facility: Department of Paediatric Cardiology University of the Free State - Site 6001
city: Pretoria
country: South Africa
facility: Division of Paediatric Cardiology, Steve Biko Academic Hospital - Site 6002
city: Barcelona
country: Spain
facility: Hospital Universitatario Vall d'Hebron, Neumologia - Site 1907
city: Madrid
country: Spain
facility: Hospital Universitario La Paz - Paediatric Cardiology Department - Site 1906
city: Dnepropetrovsk
country: Ukraine
facility: Clinical Diagnostic Center - Pediatric Cardiovascular and ANES and Intensive Care Department - Site 4103
city: Donetsk
country: Ukraine
facility: Gusak Ins Urgent and Recovery SUR AMS - Cardiovascular Rehabilitation Pediatric Department - Site 4101
city: Kiev
country: Ukraine
facility: Gover INS - Scientific Practical Cardiovascular Pediatric Center - MOH Ukraine - Site 4102
Study officials:
References: