Last updated: 06/03/2024 05:50:29
Long-term ambrisentan extension study for pediatric patients who participated in AMB112529
EudraCT ID
EU CT Number
Not applicable
Trial status
Completed
Completed
Trial overview
Official title: 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
Trial description: 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.
Primary purpose:
Treatment
Trial design:
Single Group Assignment
Masking:
None (Open Label)
Allocation:
Not applicable
Primary outcomes:
Serious Adverse Events
Timeframe: up to 18 years of age
Adverse Events
Timeframe: up to 18 years of age
Liver Function Tests
Timeframe: up to 18 years of age
Clinical Chemistries
Timeframe: up to 18 years of age
Haematology
Timeframe: up to 18 years of age
Physical examination
Timeframe: up to 18 years of age
Vital signs
Timeframe: up to 18 years of age
12-lead ECG
Timeframe: up to 18 years of age
Endocrinology assessments
Timeframe: up to 20 years of age
Pubertal Development
Timeframe: up to 20 years of age
Secondary outcomes:
6 minute walk distance
Timeframe: up to 18 years of age
WHO functional class
Timeframe: up to 18 years of age
Health outcomes assessments
Timeframe: up to 18 years of age
Echocardiogram
Timeframe: up to 18 years of age
Plasma NT-Pro-BNP
Timeframe: up to 18 years of age
Time to clinical worsening
Timeframe: up to 18 years of age
Interventions:
Drug: Ambrisentan
Enrollment:
38
Observational study model:
Not applicable
Primary completion date:
2022-09-06
Time perspective:
Not applicable
Clinical publications:
Dunbar Ivy, Maurice Beghetti, Ernesto Juaneda-Simian, Ramiya Ravindranath, Mary Ann Lukas, Sandra Machlitt-Northen, Nicola Scott, Jun Narita, Rolf M F Berger. Long-term safety and tolerability of ambrisentan treatment for pediatric patients with pulmonary arterial hypertension: An open-label extension study.. European journal of pediatrics. 2024-May;183(5): 2141-2153.
DOI : 10.1007/s00431-024-05446-1
PMID: 38366267
Medical condition
Hypertension, Pulmonary
Product
ambrisentan
Collaborators
Not applicable
Study date(s)
July 2011 to June 2022
Type
Interventional
Phase
2
Participation criteria
Sex
Female & Male
Age
8 - 18 years
Accepts healthy volunteers
No
- Have participated in and complied, to the best of their ability, with the protocol for AMB112529 and have met one of the following:
- a. Completed the Week 24 visit in AMB112529;
- Subjects who were withdrawn from ambrisentan in Study AMB112529;
- Subjects who did not comply with the protocol in Study AMB112529;
Inclusion and exclusion criteria
Inclusion criteria:
- Have participated in and complied, to the best of their ability, with the protocol for AMB112529 and have met one of the following: a. Completed the Week 24 visit in AMB112529; b. Required additional targeted treatment for PAH due to inadequate response to the current treatment or worsening of their clinical condition prior to week 24 in AMB112529; c. Required reduction in dose of baseline targeted treatment for PAH after ambrisentan was added to the treatment regimen; d. In the opinion of the investigator, continued treatment with ambrisentan is warranted.
- A female is eligible to participate in this study, as assessed by the investigator, if she is of: a. Non-childbearing potential (i.e., physiologically incapable of becoming pregnant); or, b. Child-bearing potential
- has a negative pregnancy test and is not lactating and, if sexually active, agrees to continue to use 2 reliable methods of contraception until study completion and for at least 30 days following the last dose of study drug (reliable methods of contraception are listed in Appendix 2).
- Subject or subject’s legal guardian is able and willing to give written informed consent. As part of the consent, female subjects of childbearing potential will be informed of the risk of teratogenicity and will need to be counselled in a developmentally appropriate manner on the importance of pregnancy prevention; and male subjects will need to be informed of potential risk of testicular tubular atrophy and aspermia.
Exclusion criteria:
- Subjects who were withdrawn from ambrisentan in Study AMB112529;
- Subjects who did not comply with the protocol in Study AMB112529;
- Female subjects who are pregnant or breastfeeding;
- Subjects with severe renal impairment (estimated creatinine clearance <30 mL/min assessed within the previous 45 days) at the point of transition from Study AMB112529 into this study;
- Subject with clinically significant fluid retention in the opinion of the investigator;
- Subject with clinically significant anaemia in the opinion of the investigator;
- Subjects who are to enter another clinical trial or be treated with another investigational product after exiting Study AMB112529.
Trial location(s)
Location
GSK Investigational Site
Ann Arbor, Michigan, United States, 48109-4204
Status
Study Complete
Location
GSK Investigational Site
Aurora, Colorado, United States, 80045
Status
Study Complete
Location
GSK Investigational Site
Ciudad de Buenos Aires, Argentina, 1118
Status
Study Complete
Location
GSK Investigational Site
Boston, Massachusetts, United States, 02115
Status
Study Complete
Location
GSK Investigational Site
New York, New York, United States, 10032
Status
Study Complete
Location
GSK Investigational Site
San Donato Milanese (MI), Lombardia, Italy, 20097
Status
Study Complete
Study documents
Protocol
Available language(s): English
Statistical analysis plan
Available language(s): English
If you wish to request for full study report, please contact - [email protected]
Results overview
Results posted on ClinicalTrials.gov
Recruitment status
Completed
Actual primary completion date
2022-09-06
Actual study completion date
2022-09-06
Plain language summaries
Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.
Additional information about the trial
Additional information
Not applicable
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