Ignite Creation Date:
2024-05-05 @ 11:27 PM
Last Modification Date:
2024-10-26 @ 10:34 AM
Study NCT ID:
NCT01332331
Status:
TERMINATED
Last Update Posted:
2019-10-08
First Post:
2011-03-24
Brief Title:
Efficacy and Safety of Ambrisentan in Children 8-18yrs
Sponsor:
GlaxoSmithKline
Organization:
GlaxoSmithKline
Study Overview
Official Title:
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
Status:
TERMINATED
Status Verified Date:
2019-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Ongoing juvenile rat toxicology studies
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 screeningbaseline period and a treatment period The treatment period will be 24 weeks or until the subjects clinical condition deteriorates to the point that alternativeadditional 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 28 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 37 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 048 cases per million children per year and the prevalence was 21 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 Agencys Paediatric Committee PDCO
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 Agencys Paediatric Committee PDCO
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2010-019547-19 | EUDRACT_NUMBER | None | None |