Endothelin Blockade in Patients With Single Ventricle Physiology

• Sponsor: University of California, Los Angeles (Other)
• Overall Status: Completed
• CT.gov ID: NCT00989911
• Collaborator: Actelion (Industry)
• Enrollment: 10
• Location: 1
• Arm: 1
• Duration (Months): 34
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

Recent studies have shown beneficial effects of sildenafil in patients with single ventricle congenital heart disease. The purpose of this study is to determine whether Bosentan, a drug with similar effects, will have similar benefit in this patient population.

Condition or Disease	Intervention/Treatment	Phase
Congenital Heart Disease	Drug: Bosentan	N/A

Detailed Description

Patients enrolled in the study will take Bosentan. Baseline and follow up measurements will be collected then analyzed.

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Supportive Care
• Official Title: Endothelin Blockade in Patients With Single Ventricle Physiology
• Study Start Date: May 1, 2010
• Actual Primary Completion Date: Mar 1, 2013
• Actual Study Completion Date: Mar 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Bosentan Bosentan	Drug: Bosentan; Bosentan 62.5 mg tablet taken orally twice daily for one month, followed by Bosentan 125 mg tablet taken orally twice daily for three months.; Other Names: Tracleer

Outcome Measures

Primary Outcome Measures

1. Pulmonary Blood Flow as Determined by MRI Velocity Encoding at 3-6 Months [3-6 months]

   Magnetic resonance imaging-derived aortic flow

Eligibility Criteria

Criteria

• Ages Eligible for Study: 12 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• At least 12 years of age

• Single ventricle congenital heart disease

• Require cardiac magnetic resonance imaging (MRI) for clinical purposes.

Exclusion Criteria:

• < 12 years of age

• Pregnancy or women who may become pregnant (sexually active and unwilling to use birth control)

• Patients taking cyclosporin or glyburide

• Patients with baseline liver dysfunction (aspartate aminotransferase or alanine aminotransferase > 3 times upper limit of normal)

• Contraindications to MRI (including pacemakers)

• Use of Bosentan within one month prior to enrollment

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCLA Medical Center	Los Angeles	California	United States	90095

Sponsors and Collaborators

• University of California, Los Angeles
• Actelion

Investigators

• Principal Investigator: Jamil Aboulhosn, MD, University of California, Los Angeles

Study Documents (Full-Text)

None provided.

More Information

Publications

• Giardini A, Balducci A, Specchia S, Gargiulo G, Bonvicini M, Picchio FM. Effect of sildenafil on haemodynamic response to exercise and exercise capacity in Fontan patients. Eur Heart J. 2008 Jul;29(13):1681-7. doi: 10.1093/eurheartj/ehn215. Epub 2008 Jun 4.
• Votava-Smith JK, Perens GS, Alejos JC. Bosentan for increased pulmonary vascular resistance in a patient with single ventricle physiology and a bidirectional Glenn shunt. Pediatr Cardiol. 2007 Jul-Aug;28(4):314-6. Epub 2007 Jun 11.

• Responsible Party: Jamil Aboulhosn, Assistant Professor of Medicine, University of California, Los Angeles
• ClinicalTrials.gov Identifier: NCT00989911
• Other Study ID Numbers: Actelion - 1
• First Posted: Oct 6, 2009
• Last Update Posted: Feb 27, 2015
• Last Verified: Feb 1, 2015

• Keywords provided by Jamil Aboulhosn, Assistant Professor of Medicine, University of California, Los Angeles: Single ventricle congenital heart disease
• Additional relevant MeSH terms: Heart Diseases; Heart Defects, Congenital; Univentricular Heart; Bosentan

Study Results

Participant Flow

Arm/Group Title	Bosentan
Arm/Group Description	Bosentan: Bosentan 62.5 mg tablet taken orally twice daily for one month, followed by Bosentan 125 mg tablet taken orally twice daily for three months.
Period Title: Overall Study
STARTED	10
COMPLETED	7
NOT COMPLETED	3

Baseline Characteristics

Arm/Group Title	Bosentan
Arm/Group Description	Bosentan: Bosentan 62.5 mg tablet taken orally twice daily for one month, followed by Bosentan 125 mg tablet taken orally twice daily for three months.
Overall Participants	10
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	10 100%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	34 (10.9)
Sex: Female, Male (Count of Participants)
Female	7 70%
Male	3 30%
Region of Enrollment (participants) [Number]
United States	10 100%

Outcome Measures

1. Primary Outcome

Title	Pulmonary Blood Flow as Determined by MRI Velocity Encoding at 3-6 Months
Description	Magnetic resonance imaging-derived aortic flow
Time Frame	3-6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Bosentan
Arm/Group Description	Bosentan: Bosentan 62.5 mg tablet taken orally twice daily for one month, followed by Bosentan 125 mg tablet taken orally twice daily for three months.
Measure Participants	7
BaselineAortic Flow Volume	3.3 (1.27)
3-6 Month Aortic Flow Volume	4.4 (0.9)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Bosentan
	Comments
	Type of Statistical Test	Superiority or Other
	Comments
Statistical Test of Hypothesis	p-Value	0.03
	Comments
	Method	t-test, 2 sided
	Comments

Adverse Events

Arm/Group Title	Bosentan
Arm/Group Description	Bosentan: Bosentan 62.5 mg tablet taken orally twice daily for one month, followed by Bosentan 125 mg tablet taken orally twice daily for three months.
All Cause Mortality
	Bosentan
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Bosentan
	Affected / at Risk (%)	# Events
Total	0/10 (0%)
Other (Not Including Serious) Adverse Events
	Bosentan
	Affected / at Risk (%)	# Events
Total	3/10 (30%)
Cardiac disorders
fatigue	3/10 (30%)	3

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.