No registrations found.
ID
Source
Brief title
Health condition
Pulmonary arterial hypertension in children
Sponsors and support
Gewerbestrasse 16
CH-4123 Allschwil
Switzerland
Gewerbestrasse 16
CH-4123 Allschwil
Switzerland
Intervention
Outcome measures
Primary outcome
No primary endpoint was defined in this study.
Secondary outcome
Exploratory efficacy endpoints:
1. Change from baseline to end of study in WHO functional class;
2. Time to death, lung transplantation or hospitalization for PAH-Progression;
3. Time to death, lung transplantation, hospitalization for PAH-progression or initiation of new therapy for PAH or new/worsening right heart failure;
4. Changes from baseline to 3, 6, 9, 12, 15 and 18 months of treatment over AC 052 373 and AC-052-374 in Global Clinical Impression scale assessed by the physician and parents.
Safety and tolerability endpoints:
1. Treatment-emergent AEs and serious adverse events (SAEs) up to 7 days after permanent discontinuation of study drug;
2. Adverse events leading to premature discontinuation of study drug;
3. Serious adverse events from 7 up to 60 days after permanent discontinuation of study drug;
4. Changes from baseline to end of study in vital signs, body weight, and height/length;
5. Treatment-emergent marked laboratory abnormalities up to end of study.
Background summary
This is a prospective, multicenter, multinational, open-label, double-arm exploratory Phase 3 extension study enrolling those patients who completed the FUTURE 3 core study (AC 052-373). It is designed to evaluate the long-term tolerability and safety of bosentan using the pediatric formulation in children with idiopathic or heritable PAH or PAH persisting after complete repair of a congenital heart defect.
Patients will receive the bosentan pediatric formulation. The bosentan dosage will be adjusted to the patient’s body weight during the study to achieve a maintenance dose of 2 mg/kg either b.i.d. or t.i.d.
The maximum number of participants corresponds to the number of patients treated in the FUTURE 3 core study (AC-052-373).
The study will be conducted at expert pediatric PAH centers in Europe, US, Latin America, Australia and Asia.
The study will consist of a treatment period and a post-treatment follow-up period of 60 days. Patients will receive the maintenance dose (2 mg/kg either b.i.d. or t.i.d.) of bosentan using the pediatric formulation for the entire duration of the study.
The treatment period in FUTURE 3 Study Extension will last for 12 months or until:
1. The investigator or the patient decides to discontinue the study treatment permanently;
2. The sponsor decides not to pursue the development of the pediatric formulation of bosentan.
Study objective
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).
Study design
The study will consist of a treatment period and a post-treatment follow-up period of 60 days. Patients will receive the maintenance dose (2 mg/kg either b.i.d. or t.i.d.) of bosentan using the pediatric formulation for the entire duration of the study. The treatment period in FUTURE 3 Study Extension will last for 12 months or until:
1. The investigator or the patient decides to discontinue the study treatment permanently;
2. The sponsor decides not to pursue the development of the pediatric formulation of bosentan.
Intervention
Bosentan dispersible tablet (32 mg) in the dosage of 2 mg/kg b.i.d. or 2 mg/kg t.i.d.
Peter Westerveld
Woerden 3446 GR
The Netherlands
+31 (0)348 489181
peter.westerveld@actelion.com
Peter Westerveld
Woerden 3446 GR
The Netherlands
+31 (0)348 489181
peter.westerveld@actelion.com
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 by the investigator at the end of 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.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL2538 |
| NTR-old | NTR2656 |
| Other | : |
| ISRCTN | ISRCTN wordt niet meer aangevraagd. |