Trial Outcomes & Findings for The Effects of Ambrisentan on Exercise Capacity in Fontan Patients (NCT NCT01971580)

NCT ID: NCT01971580

Last Updated: 2018-05-07

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 28 participants
• Primary outcome timeframe: Baseline compared to 12 weeks therapy with either ambrisentan or placebo
• Results posted on: 2018-05-07

Participant Flow

Patients were recruited from Washington University School of Medicine Adult Congenital Heart Disease clinic between 10/2012 and 11/2014

Participant milestones

Measure	Ambrisentan First, Placebo Second These patients will be randomized to have ambrisentan during the first study period, and placebo during the second study period. Ambrisentan	Placebo First, Ambrisentan Second These patients will be randomized to have placebo during the first study period, and ambrisentan during the second study period. Ambrisentan
Overall Study STARTED	14	14
Overall Study COMPLETED	10	9
Overall Study NOT COMPLETED	4	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

The Effects of Ambrisentan on Exercise Capacity in Fontan Patients

Baseline characteristics by cohort

Measure	Ambrisentan First, Placebo Second n=14 Participants These patients will be randomized to have ambrisentan during the first study period, and placebo during the second study period. Ambrisentan	Placebo First, Ambrisentan Second n=14 Participants These patients will be randomized to have placebo during the first study period, and ambrisentan during the second study period. Ambrisentan	Total n=28 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants	14 Participants n=7 Participants	28 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	4 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline compared to 12 weeks therapy with either ambrisentan or placebo

Population: In this crossover study, all patients received both ambrisentan and placebo. Data was analyzed comparing baseline measures to those after the period on ambrisentan and baseline measures to those after the period on placebo.

Outcome measures

Measure	On Ambrisentan Therapy n=19 Participants After Ambrisentan compared to baseline	On Placebo n=19 Participants After Placebo compared to baseline
Change in VO2 Max	1.33 Change in ml/kg min Interval -0.92 to 3.57	0.745 Change in ml/kg min Interval -2.06 to 2.42

SECONDARY outcome

Timeframe: 12 weeks

Population: In this crossover study, all patients received both therapies. Analysis was performed comparing baseline to after indicated therapy

Change in Rand 36-item Short Form (SF-36) physical function score (0-100). On the Rand SF-36 questionnaire, a score closer to 100 (higher) suggests lesser disability, while a score closer to 0 (lower) suggests more disability.

Outcome measures

Measure	On Ambrisentan Therapy n=19 Participants After Ambrisentan compared to baseline	On Placebo n=19 Participants After Placebo compared to baseline
Quality of Life	3.75 Change in score from baseline Interval -5.78 to 13.2	0.195 Change in score from baseline Interval -9.34 to 9.73

Adverse Events

On Ambrisentan Therapy

Serious events: 1 serious events

Other events: 13 other events

Deaths: 0 deaths

On Placebo

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Measure	On Ambrisentan Therapy n=19 participants at risk Events that occurred while on ambrisentan therapy	On Placebo n=19 participants at risk Events that occurred while on placebo therapy
Gastrointestinal disorders Gastroenteritis and acidosis	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	0.00% 0/19 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.

Other adverse events

Measure	On Ambrisentan Therapy n=19 participants at risk Events that occurred while on ambrisentan therapy	On Placebo n=19 participants at risk Events that occurred while on placebo therapy
Respiratory, thoracic and mediastinal disorders Rhinitis or Pharyngitis	31.6% 6/19 • Number of events 6 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	42.1% 8/19 • Number of events 8 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Skin and subcutaneous tissue disorders edema	15.8% 3/19 • Number of events 3 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	10.5% 2/19 • Number of events 2 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Skin and subcutaneous tissue disorders rash	10.5% 2/19 • Number of events 2 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	15.8% 3/19 • Number of events 3 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Musculoskeletal and connective tissue disorders arthralgia	0.00% 0/19 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Respiratory, thoracic and mediastinal disorders cough/dyspnea	21.1% 4/19 • Number of events 4 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	15.8% 3/19 • Number of events 3 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Gastrointestinal disorders nausea	21.1% 4/19 • Number of events 4 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	10.5% 2/19 • Number of events 2 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Cardiac disorders chest pain	0.00% 0/19 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	10.5% 2/19 • Number of events 2 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
General disorders fatigue	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Gastrointestinal disorders abdominal pain	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	0.00% 0/19 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.
Gastrointestinal disorders diarrhea	5.3% 1/19 • Number of events 1 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.	10.5% 2/19 • Number of events 2 • During the study period and for one month after the completion of the study protocol, an average of 30 weeks.

Additional Information

Ari Cedars

UT Southwestern

Phone: 3149224788

Email: acedars97@gmail.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place