A randomized, open label study comparing safety and efficacy parameters for a high and a low dose of ambrisentan (adjusted for body weight) for the treatment of pulmonary arterial hypertension in paediatric patients aged 8 years up to 18 years (AMB112529)

Phase 2

Withdrawn

• Conditions: Hypertension; Pulmonary; Pulmonary Arterial Hypertension; 10037454

• Registration Number: NL-OMON34214
• Lead Sponsor: GlaxoSmithKline

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 5

Inclusion Criteria

• 8-17 (inclusive) years.
• Pulmonary arterial hypertension due to
o idiopathic or heritable PAH
o secondary to connective tissue disease
o persistent after surgical repair of congenital heart defects (greater than 6 months prior to screening).
• WHO class II or III.
• In case of HIV: stable disease (se protocol for details).
• mPAP at least 25 mmHg and PVR at least 240 dyne*sec/cm5 and PCWP or LVEDP of at most 15 mmHg.
• Treatment naïve, without PAH medication for at least 1 month or stable background medication.
• Females of childbearing potential: reliable method of contraception.

Exclusion Criteria

• ERA or cyclosporine A treatment.
• Weight less than 20 kgs.
• Previous safety or tolerability issues with PAH treatment (see protocol for details).
• Pregnancy or breastfeeding

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Adverse events.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>PK, exercise tolerance, time to clinical worsening, NT proBNP, NYHA class,<br /><br>SF-10 questionnaire.</p><br>