Bosentan in Children With Pulmonary Arterial Hypertension Extension Study

NCT00319020 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: AC-052-3672005-001967-70
• Study Type: interventional
• Target: 33
• Locations: 0 countries
• Sites: N/A

Brief Summary

The main objective of the FUTURE 2 study was to assess the long-term safety and tolerability of the pediatric formulation of bosentan in children with idiopathic pulmonary arterial hypertension or familial pulmonary arterial hypertension who completed FUTURE 1 study.

Conditions

Pulmonary Arterial Hypertension

Keywords

bosentan; pulmonary arterial hypertension; children; FUTURE 1

Outcome Measures

Primary Outcomes (8)

• Change From Baseline to End of Study (EOS) in Height for Age.

  In order to compare the growth data with those of healthy children, growth curves are calculated from height data collected throughout the follow-up period. For each patient, height measured at each study visit was converted to a z-score and expressed in standard deviations (SD) from WHO growth standards. The Z-score was calculated according to the following formula: Z-score = (observed value of the study participant - median value of the reference population) / SD value of the reference population

  From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

• Change From Baseline to End of Study (EOS) in Body Weight

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including growth as measured by changes from baseline in body weight and height.

  From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

• Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.

  From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

• Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.

  From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

• Change From Baseline to End of Study (EOS) in Pulse Rate

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in pulse rate.

  From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

• Proportion of Patients With Treatment-emergent Liver Function Abnormalities

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including laboratory abnormalities related to liver enzymes. Proportion of patients with increase in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) above 3 times upper limit of normal (ULN) is reported here.

  After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average

• Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities

  The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including hemoglobin abnormalities. Proportion of patients with marked hemoglobin decreases (i.e., decrease of or above 15% of the lower normal limit (LL)) is reported here.

  After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average

• Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment

  From the first study drug administration in FUTURE 1, for an average of 31 months

Study Arms (1)

Bosentan

EXPERIMENTAL

Bosentan was administered at 4 mg/kg twice daily (b.i.d.) until the end of the study. It could be down-titrated to 2 mg/kg b.i.d. if not well tolerated.

Drug: Bosentan

Interventions

Bosentan DRUG

32-mg dispersible and breakable tablet. The body weight-adjusted dose of the dispersible tablet was dispersed in a teaspoon of water (not mixed with food) before being administered orally

Also known as: Ro 47-0203, Tracleer

Bosentan

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Signed informed consent by the parents or the legal representatives.
• Patients who completed the FUTURE 1 study.
• Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial at the end of FUTURE 1.
• Males or females \>= 2 and \< 12 years of age at enrollment in FUTURE 2 (this study). Females who are menstruating must have a negative pregnancy test. A reliable method of contraception must be considered, if appropriate.

You may not qualify if:

• Intolerance to bosentan despite dose reductions.
• Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy.
• Pregnancy or breast-feeding.
• Known hypersensitivity to bosentan or any of the excipients.
• Premature and permanent study drug discontinuation during FUTURE 1.

Sponsors & Collaborators

• Actelion: lead

Related Publications (1)

• Berger RM, Haworth SG, Bonnet D, Dulac Y, Fraisse A, Galie N, Ivy DD, Jais X, Miera O, Rosenzweig EB, Efficace M, Kusic-Pajic A, Beghetti M. FUTURE-2: Results from an open-label, long-term safety and tolerability extension study using the pediatric FormUlation of bosenTan in pUlmonary arterial hypeRtEnsion. Int J Cardiol. 2016 Jan 1;202:52-8. doi: 10.1016/j.ijcard.2015.08.080. Epub 2015 Aug 9.

  PMID: 26386921 RESULT

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

Bosentan

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Benzenesulfonamides; Sulfonamides; Amides; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Sulfones; Sulfur Compounds; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: clinical trial disclosure desk
• Organization: Actelion Pharmaceuticals Ltd

clinical-trials-disclosure@actelion.com

Study Officials

• Andjela Kusic-Pajic, MD

  Actelion

  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: April 26, 2006
• First Posted: April 27, 2006
• Study Start: August 23, 2005
• Primary Completion: October 28, 2011
• Study Completion: October 28, 2011
• Last Updated: February 4, 2025
• Results First Posted: March 29, 2017

Record last verified: 2025-01