Long-term Ambrisentan Extension Study for Pediatric Patients Who Participated in AMB112529
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ClinicalTrials.gov Identifier: NCT01342952 |
Recruitment Status
:
Recruiting
First Posted
: April 27, 2011
Last Update Posted
: March 30, 2018
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Tracking Information | |||||
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First Submitted Date ICMJE | April 26, 2011 | ||||
First Posted Date ICMJE | April 27, 2011 | ||||
Last Update Posted Date | March 30, 2018 | ||||
Study Start Date ICMJE | June 1, 2011 | ||||
Estimated Primary Completion Date | October 27, 2022 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | Complete list of historical versions of study NCT01342952 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | Not Provided | ||||
Original Other Outcome Measures ICMJE | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Long-term Ambrisentan Extension Study for Pediatric Patients Who Participated in AMB112529 | ||||
Official Title ICMJE | An Open-label, Long Term Extension Study for Treatment of Pulmonary Arterial Hypertension in Paediatric Patients Aged 8 Years up to 18 Years Who Have Participated in AMB112529 and in Whom Continued Treatment With Ambrisentan is Desired | ||||
Brief Summary | An open label, long term extension to Study AMB112529. All subjects may remain in the extension study for a minimum of six months. Beyond the six month period, subjects may continue in the extension study until one of the following conditions is met: the subject turns 18 years of age (when the subject can receive marketed product) the product is approved and available for use in the subject's age group, development for use in the paediatric population is discontinued. the subject decides he/she no longer wants to participate in the study, the investigator considers it is in the best interest of the subject to discontinue ambrisentan (e.g. for safety reasons). The primary objective is the long-term safety and tolerability of ambrisentan in the paediatric PAH population. Secondary objectives are all cause mortality and change from baseline in Study AMB112529 on efficacy parameters. |
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Detailed Description | Pulmonary arterial hypertension (PAH) is a rare, progressive, highly debilitating disease characterized by vascular obstruction and the variable presence of vasoconstriction, leading to increased pulmonary vascular resistance and right-sided heart failure. If left untreated, PAH ultimately leads to right ventricular failure and death; adult subjects have a median survival of 2.8 years without treatment. Epidemiological estimates vary but prevalence in Europe is thought to be of the order of 15 cases per million. Large scale epidemiology studies of PAH in children have not been conducted and there is no or limited outcome data in paediatric PAH patients. A register in France (1995-1996) estimates the prevalence in children is as low as 3.7 cases per million. In a national, comprehensive country wide survey of the epidemiology of idiopathic PAH (IPAH) management and survival in the United Kingdom (UK) the incidence was 0.48 cases per million children per year and the prevalence was 2.1 cases per million children. Ambrisentan (VOLIBRIS™ tablets) is an endothelin receptor antagonist (ERA) marketed in the European Union (EU) and some other countries by GlaxoSmithKline (GSK) and in the United States as LETAIRIS® by Gilead Sciences Inc. Ambrisentan is indicated for the treatment of adult patients with PAH to improve exercise capacity, decrease the symptoms of PAH, and delay clinical worsening. The primary purpose of this long term paediatric study is to provide clinically relevant information on the long term safety of ambrisentan in children with the most common causes of PAH in this age group. This study is only open to patients who have participated in Study AMB112529, 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, and in whom continued treatment with ambrisentan is warranted. This study is part of a Paediatric Investigational Plan (PIP; EMEA-000434-PIP01-08) agreed with the European Medicines Agency's Paediatric Committee (PDCO). |
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Study Type ICMJE | Interventional | ||||
Study Phase | Phase 2 | ||||
Study Design ICMJE | Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Hypertension, Pulmonary | ||||
Intervention ICMJE | Drug: Ambrisentan
open label, flexible dosing from 2.5 to 10 mg (not to exceed 10 mg/kg) per day |
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Study Arms | Experimental: Ambrisentan
Open label, flexible dosing from 2.5 mg to 10 mg (not to exceed 0.25 mg/kg) per day
Intervention: Drug: Ambrisentan |
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Recruiting | ||||
Estimated Enrollment ICMJE |
66 | ||||
Original Estimated Enrollment ICMJE |
60 | ||||
Estimated Study Completion Date | October 24, 2024 | ||||
Estimated Primary Completion Date | October 27, 2022 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender |
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Ages | 8 Years to 18 Years (Child, Adult) | ||||
Accepts Healthy Volunteers | No | ||||
Contacts ICMJE |
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Listed Location Countries ICMJE | Argentina, Canada, France, Germany, Hungary, Italy, Japan, Russian Federation, Spain, United States | ||||
Removed Location Countries | Brazil, Mexico, Netherlands | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT01342952 | ||||
Other Study ID Numbers ICMJE | 114588 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product | Not Provided | ||||
IPD Sharing Statement | Not Provided | ||||
Responsible Party | GlaxoSmithKline | ||||
Study Sponsor ICMJE | GlaxoSmithKline | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | GlaxoSmithKline | ||||
Verification Date | March 2018 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |