Efficacy and Safety of Ambrisentan in Children 8-18yrs
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01332331 |
Recruitment Status
:
Suspended
(Ongoing juvenile rat toxicology studies.)
First Posted
: April 11, 2011
Last Update Posted
: April 20, 2018
|
Tracking Information | ||||
---|---|---|---|---|
First Submitted Date ICMJE | March 24, 2011 | |||
First Posted Date ICMJE | April 11, 2011 | |||
Last Update Posted Date | April 20, 2018 | |||
Actual Study Start Date ICMJE | January 4, 2011 | |||
Estimated Primary Completion Date | May 9, 2018 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
|
|||
Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | Complete list of historical versions of study NCT01332331 on ClinicalTrials.gov Archive Site | |||
Current Secondary Outcome Measures ICMJE |
|
|||
Original Secondary Outcome Measures ICMJE | Same as current | |||
Current Other Outcome Measures ICMJE | Not Provided | |||
Original Other Outcome Measures ICMJE | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Efficacy and Safety of Ambrisentan in Children 8-18yrs | |||
Official Title ICMJE | 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 | |||
Brief Summary | A 6-month (24-week), randomized, open label evaluation of the safety, tolerability, and efficacy of a high and low dose ambrisentan (adjusted for body weight) treatment group in subjects aged 8 years up to 18 years with pulmonary arterial hypertension (PAH). An additional objective is to determine the ambrisentan population pharmacokinetics in the paediatric population. The study will include a screening/baseline period and a treatment period. The treatment period will be 24 weeks or until the subject's clinical condition deteriorates to the point that alternative/additional treatment is necessary. Patients who participate in the study and in whom continued treatment with ambrisentan is desired will be eligible to enrol into a long term follow-up study. The primary comparison will be the safety and tolerability of the two ambrisentan dose groups (Low vs. High) in the paediatric PAH population The secondary comparison will be the change from baseline for the efficacy parameters between the two treatment groups. | |||
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 pediatric 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 paediatric study is to provide clinically relevant information on the safety and pharmacokinetic profile of ambrisentan in children with the most common causes of PAH in this age group. The design of the study is also intended to provide information to guide dose selection and supportive efficacy data in this age group. Despite the fact that none of the currently available adult treatments are licensed for use in children <12 yrs, (with the exception of bosentan which was recently approved for use in paediatric population from 2 years of age) they are widely used off label. This study will provide useful prescribing information to the medical community for treating this orphan disease in children in this environment of rapidly changing medical practice. This study is part of a Paediatric Investigational Plan (PIP; EMEA-000434-PIP01-08) agreed with the European Medicines Agency's Paediatric Committee (PDCO). |
|||
Study Type ICMJE | Interventional | |||
Study Phase | Phase 2 | |||
Study Design ICMJE | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Single (Outcomes Assessor) Primary Purpose: Treatment |
|||
Condition ICMJE | Hypertension, Pulmonary | |||
Intervention ICMJE |
|
|||
Study Arms |
|
|||
Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
||||
Recruitment Information | ||||
Recruitment Status ICMJE | Suspended | |||
Estimated Enrollment ICMJE |
66 | |||
Original Estimated Enrollment ICMJE |
60 | |||
Estimated Study Completion Date | May 9, 2018 | |||
Estimated Primary Completion Date | May 9, 2018 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
|
|||
Sex/Gender |
|
|||
Ages | 8 Years to 18 Years (Child, Adult) | |||
Accepts Healthy Volunteers | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | Argentina, Canada, France, Germany, Hungary, Italy, Japan, Netherlands, Russian Federation, Spain, United States | |||
Removed Location Countries | Brazil, Greece, Mexico | |||
Administrative Information | ||||
NCT Number ICMJE | NCT01332331 | |||
Other Study ID Numbers ICMJE | 112529 | |||
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 |
|
|||
PRS Account | GlaxoSmithKline | |||
Verification Date | April 2018 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |