NCT01332331 is a Phase 2 clinical trial of Ambrisentan - low dose in Hypertension, Pulmonary, sponsored by GlaxoSmithKline.

Who is sponsoring NCT01332331?

NCT01332331 is sponsored by GlaxoSmithKline.

What drugs are being tested in NCT01332331?

Interventions in NCT01332331: Ambrisentan - low dose, Ambrisentan - high dose.

What condition does NCT01332331 study?

NCT01332331 studies Hypertension, Pulmonary.

Is NCT01332331 still recruiting?

NCT01332331 is currently terminated. Last updated 8 October 2019.

Are results published for NCT01332331?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2019-10-08 · How we verify

NCT01332331

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Efficacy and Safety of Ambrisentan in Children 8-18yrs

Terminated Phase 2 Results posted Last updated 8 October 2019

What this trial tests

Phase 2 trial testing Ambrisentan - low dose in Hypertension, Pulmonary in 41 participants. Terminated before completion.

Timeline

4 January 2011

Primary endpoint
12 November 2013

12 November 2013

Quick facts

Lead sponsor	GlaxoSmithKline
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	single
Primary purpose	treatment
Enrollment	41
Start date	4 January 2011
Primary completion	12 November 2013
Estimated completion	12 November 2013
Sites	24 locations across France, Italy, Japan, Russia, Germany, Hungary, Argentina, United States

Drugs / interventions tested

Ambrisentan - low dose — full drug profile →
Ambrisentan - high dose — full drug profile →

Conditions studied

Hypertension, Pulmonary — all drugs for Hypertension, Pulmonary →

Sponsor

GlaxoSmithKline — full company profile →

Who can join

Adults 8 to 18, any sex, with Hypertension, Pulmonary. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Treatment-emergent Adverse Events (SAEs) Primary · Up to 24 Weeks

AEs defined as any untoward medical occurrence in a patient or clinical investigation participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. SAEs defined as any untoward medical occurrence that, at any dose results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability or incapacity, or is a congenital anomaly or birth defect, medical events that may not immediately life threatening or result in death or hospitalization but may jeopardize the particip

Any AEs

Group	Value	95% CI
Low Dose Ambrisentan	16
High Dose Ambrisentan	16

Any SAEs

Group	Value	95% CI
Low Dose Ambrisentan	6
High Dose Ambrisentan	2

Number of Participants With Post Baseline Potential Clinical Importance (PCI) Value for Clinical Chemistry Parameters: Alanine Amino Transferase (ALT), Aspartate Aminotransferase (AST), Gamma Glutamyl Transferase (GGT), Total Bilirubin and Creatinine Primary · Up to 24 Weeks

Blood samples were collected from participants for analysis of following clinical chemistry parameters: ALT, AST, GGT, total bilirubin and creatinine. PCI ranges were \<3 times the upper limit of normal (ULN), \<34.2 Micromoles per liter (UMOL/L) for total bilirubin and \<176.8 (UMOL/L) for creatinine. Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

ALT

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

AST

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

GGT

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Total bilirubin

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Creatinine

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Number of Participants With Post Baseline PCI Value for Hematology Parameter: Hemoglobin Primary · Up to 24 Weeks

Blood samples were collected from participants for analysis of following hematology parameters: hemoglobin. PCI ranges were Males: 98 to180 grams per liter (G/L), Females: 91 to 161 (G/L) for hemoglobin. Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

Reference high range

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	2

Reference low range

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Number of Participants With Post Baseline PCI Value for Hematology Parameter: Hematocrit Primary · Up to 24 Weeks

Blood samples were collected from participants for analysis of following hematology parameter: hematocrit. PCI ranges were males: \<0.32 to \>0.54, females: \<0.29 to \>0.506 proportion of red blood cells in blood for hematocrit. Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

Reference high range

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	2

Reference low range

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	2

Number of Participants With Post Baseline PCI Value for Hematology Parameter: Platelet Count Primary · Up to 24 Weeks

Blood samples were collected from participants for analysis of following hematology parameter: platelet count. PCI ranges were 100 to 400 for Giga cells per liter platelet count. Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

Reference high range

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Reference low range

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	1

Number of Participants With Abnormal Value for Physical Examination Parameter: Liver Size Primary · Week 12 and 24

Physical examination included measurement of liver size. Any abnormal enlargement or reduction in the size of the liver is reported.

Week 12, Abnormal: Improved, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	1

Week 12, Abnormal: Worsened, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	1

Week 12, Abnormal: Unchanged, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Week 24, Abnormal: Improved, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	2
High Dose Ambrisentan	1

Week 24, Abnormal: Worsened, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 24, Abnormal: Unchanged, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Number of Participants With Abnormal Value for Physical Examination Parameter: Jugular Venous Pressure Primary · Week 12 and 24

Physical examination of participants jugular venous pressure is measured.

Week 12, Abnormal: Improved, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	1

Week 12, Abnormal: Worsened, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	1

Week 12, Abnormal: Unchanged, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	3

Week 24, Abnormal: Improved, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	1

Week 24, Abnormal: Worsened, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Week 24, Abnormal: Unchanged, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	4

Number of Participants With Abnormal Value for Physical Examination Parameter: Peripheral Edema Primary · Week 12 and 24

Physical examination of paricipants peripheral edema is measured. Day 1 was considered as Baseline.

Week 12, Abnormal: Improved, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 12, Abnormal: Worsened, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	1

Week 12, Abnormal: Unchanged, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	1

Week 24, Abnormal: Improved, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Week 24, Abnormal: Worsened, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Week 24, Abnormal: Unchanged, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Number of Participants With Abnormal Value for Physical Examination Parameter: Ascites Primary · Week 12 and 24

Physcial examination of paricipants ascites was measured. Day 1 was considered as Baseline.

Week 12, Abnormal: Improved, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 12, Abnormal: Worsened, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 12, Abnormal: Unchanged, n=20, 19

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 24, Abnormal: Improved, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 24, Abnormal: Worsened, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Week 24, Abnormal: Unchanged, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Percentage of Physical Examination Parameter: Saturated Oxygen Level Primary · Week 12 and 24

Physical examination of participants saturated oxygen level was measured. Day 1 was considered as Baseline.

Week 12, n=20, 18

Group	Value	95% CI
Low Dose Ambrisentan	96.9	± 2.59
High Dose Ambrisentan	96.9	± 6.93

Week 24, n=19, 18

Group	Value	95% CI
Low Dose Ambrisentan	97.3	± 1.85
High Dose Ambrisentan	97.4	± 1.92

Number of Participants With Post Baseline PCI Value for Vital Signs Parameter: Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) Primary · Up to 24 Weeks

SBP and DBP were measured in semi-supine position after 5 minutes rest for the participants at indicated time points. PCI ranges were \<80 to \>160 millimeters of mercury (mmHg) for SDP and \<40 to \>110 mmHg for DBP. Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

SBP, Reference range high

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

SBP, Reference range low

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	2

DBP, Reference range high

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

DBP, Reference range low

Group	Value	95% CI
Low Dose Ambrisentan	0
High Dose Ambrisentan	0

Number of Participants With Post Baseline PCI Value for Vital Signs Parameter: Heart Rate Primary · Up to 24 Weeks

Heart rate was measured in semi-supine position after 5 minutes rest for the participants at indicated time points. PCI ranges were \<50 to \>120 beats per minute (beats/min). Only those parameters having any time post-Baseline PCI values were presented. Day 1 was considered as Baseline.

Reference range high

Group	Value	95% CI
Low Dose Ambrisentan	2
High Dose Ambrisentan	2

Reference range low

Group	Value	95% CI
Low Dose Ambrisentan	1
High Dose Ambrisentan	0

Adverse events — posted to ClinicalTrials.gov

Time frame: On-treatment serious adverse events (SAEs) and non serious AEs were collected from the start of study treatment up to 24 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Low Dose Ambrisentan

Serious: 6/21 (29%)

Deaths: 1/21

High Dose Ambrisentan

Serious: 2/20 (10%)

Deaths: 0/20

Serious adverse events (9 terms)

Reaction	System	Low Dose Ambrisentan	High Dose Ambrisentan
Cardiac failure acute	Cardiac disorders	—	—
Right ventricular failure	Cardiac disorders	—	—
General physical health deterioration	General disorders	—	—
Device related infection	Infections and infestations	—	—
Pharyngitis	Infections and infestations	—	—
Pneumonia	Infections and infestations	—	—
Syncope	Nervous system disorders	—	—
Device breakage	Product Issues	—	—
Pulmonary hypertension	Respiratory, thoracic and mediastinal disorders	—	—

Other adverse events (45 terms — click to expand)

Reaction	System	Low Dose Ambrisentan	High Dose Ambrisentan
Headache	Nervous system disorders	—	—
Abdominal pain	Gastrointestinal disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Abdominal pain upper	Gastrointestinal disorders	—	—
Nasopharyngitis	Infections and infestations	—	—
Upper respiratory tract infection	Infections and infestations	—	—
Diarrhoea	Gastrointestinal disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Face oedema	General disorders	—	—
Pyrexia	General disorders	—	—
Oedema peripheral	General disorders	—	—
Gastroenteritis	Infections and infestations	—	—
Laryngitis	Infections and infestations	—	—
Pharyngitis	Infections and infestations	—	—
Pneumonia	Infections and infestations	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—
Nasal congestion	Respiratory, thoracic and mediastinal disorders	—	—
Erythema	Skin and subcutaneous tissue disorders	—	—
Heparin-induced thrombocytopenia	Blood and lymphatic system disorders	—	—
Lymphopenia	Blood and lymphatic system disorders	—	—
Neutropenia	Blood and lymphatic system disorders	—	—
Cyanosis	Cardiac disorders	—	—
Palpitations	Cardiac disorders	—	—
Dry mouth	Gastrointestinal disorders	—	—
Gastritis	Gastrointestinal disorders	—	—
Asthenia	General disorders	—	—
Fatigue	General disorders	—	—
Hepatomegaly	Hepatobiliary disorders	—	—
Ear infection	Infections and infestations	—	—
Gastroenteritis viral	Infections and infestations	—	—
Respiratory tract infection	Infections and infestations	—	—
Viral upper respiratory tract infection	Infections and infestations	—	—
Joint injury	Injury, poisoning and procedural complications	—	—
Limb injury	Injury, poisoning and procedural complications	—	—
Toxicity to various agents	Injury, poisoning and procedural complications	—	—
International normalised ratio increased	Investigations	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—
Neck pain	Musculoskeletal and connective tissue disorders	—	—

Most-reported serious reactions: Cardiac failure acute, Right ventricular failure, General physical health deterioration, Device related infection, Pharyngitis, Pneumonia, Syncope, Device breakage.

Data from ClinicalTrials.gov NCT01332331 adverse events section.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

A Randomized Study of Safety and Efficacy of Two Doses of Ambrisentan to Treat Pulmonary Arterial Hypertension in Pediatric Patients Aged 8 Years up to 18 Years.
Ivy D, Beghetti M, Juaneda-Simian E, Miller D, et al · · 2020 · cited 6× · PMID 37332660 · DOI 10.1016/j.ympdx.2020.100055
Long-term safety and tolerability of ambrisentan treatment for pediatric patients with pulmonary arterial hypertension: An open-label extension study.
Ivy D, Beghetti M, Juaneda-Simian E, Ravindranath R, et al · · 2024 · cited 2× · PMID 38366267 · DOI 10.1007/s00431-024-05446-1
Pediatric Population Pharmacokinetic Modeling and Exposure-Response Analysis of Ambrisentan in Pulmonary Arterial Hypertension and Comparison With Adult Data.
Okour M, Thapar MM, Farrell C, Lukas MA, et al · · 2023 · cited 2× · PMID 36579617 · DOI 10.1002/jcph.2199
Paediatric population pharmacokinetic and pharmacodynamic modelling of ambrisentan in pulmonary arterial hypertension and comparison with adult data
Okour M, Thapar M, Farrell C, Lukas MA, et al · · 2021 · DOI 10.22541/au.162191011.16279819/v1

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Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01332331 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by GlaxoSmithKline
Last refreshed: 8 October 2019

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01332331.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing

NCT01332331

Quick facts

Drugs / interventions tested

Conditions studied

Sponsor

Who can join

Results — posted to ClinicalTrials.gov

Adverse events — posted to ClinicalTrials.gov

Serious adverse events (9 terms)

Sponsor's own description

Publications & conference data

Related trials

Other recruiting trials for Hypertension, Pulmonary

Other GlaxoSmithKline trials

Verify against primary sources