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A Study of Patients Having Pulmonary Hypertension Associated With Sickle Cell Disease and Completing an ASSET Study (ASSET-3)

This study has been terminated.
(Slow enrollment)
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:
Actelion
ClinicalTrials.gov Identifier:
NCT00360087
First received: August 2, 2006
Last updated: February 11, 2010
Last verified: February 2010
History of Changes
  Purpose

This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease. That form of hypertension places people at risk for complications, including shortness of breath, pain, pneumonia, and death. Previous studies have shown that bosentan can be helpful in reducing pulmonary hypertension.

Patients ages 16 and older who have completed the 16-week treatment in the ASSET 1 or ASSET 2 study and who are not pregnant or breastfeeding may be eligible for this study. The research will be conducted in about 25 hospitals in the United States and Europe. Up to 30 participants will be enrolled. The screening visit will involve a physical examination, blood sample of about 3 teaspoons for laboratory tests, and a pregnancy test. Patients' doctors will give them bosentan tablets (62.5 mg each), to take one in the morning and one in the evening. After 1 month, patients will be told whether the dose should be increased to 125 mg tablets to take twice a day. Two weeks after the increase in dose, a blood test will be done to analyze the drug's effects on the liver. After the start of treatment, patients will return for visits every 6 months, when there will be a 6-minute walking test to measure exercise capacity and evaluate shortness of breath. There will be follow-up for patients up to the end of the study and for 28 days after the last dose of bosentan is taken, to collect information about side effects.

Some patients on bosentan have had changes in liver function and red blood cell count. Side effects commonly reported are headache, flushed appearance, inflammation of the throat and nasal passages, and gastrointestinal symptoms. If patients have sudden worsening in breathing in the first few weeks after taking bosentan, they should immediately tell their doctors, because it may be necessary to change the treatment.


Condition Intervention Phase
Pulmonary Hypertension
Sickle Cell Anemia
 Drug: Bosentan
 Phase 3

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Long-Term, Open-Label, Multicenter, Extension Study of Bosentan in Patients With Pulmonary Hypertension Associated With Sickle Cell Disease Completing a Double-Blind ASSET Study (AC-052-368 or AC 052-369)

Resource links provided by NLM:

Drug Information available for: Bosentan
U.S. FDA Resources

Further study details as provided by Actelion:

Primary Outcome Measures:
  • Change from baseline to all assessed time points in 6MWT, in Borg dyspnea index, and in modified NYHA functional class.

Estimated Enrollment: 236
Study Start Date: March 2006
Study Completion Date: December 2007
Primary Completion Date: August 2007 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Bosentan
    N/A
Detailed Description:

The object of this study is to assess long-term safety, tolerability and efficacy of bosentan in patients with pulmonary hypertension (PH) associated with sickle cell disease (SCD). The study population will include male and female patients with sickle cell disease (SS,S-beta-Thalassemia) who have previously completed the 16-week treatment period of the double-blind study of bosentan (ASSET 1 or ASSET 2). Patients who meet all the inclusion criteria and none of the exclusion criteria will be started on 62.5 mg bid for 4 weeks and then start the maintenance dose of 125 mg bid (or stay on 62.5 mg if their weight is less than 40kg/90lbs). Patients will be divided into two groups. Group A will consist of patients who begin this study within 4 weeks of completing ASSET 1 or ASSET2. Group B will consist of patients who begin this study longer than 4 weeks after completing ASSET I or ASSET 2. Patients will remain on drug until the FDA approves the drug for use in patients with pulmonary hypertension or until the sponsor decides to stop the study.

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA
  • Completion of the 16-week treatment period in the double-blind ASSET study
  • Women of childbearing potential must have a negative result on their serum pregnancy test and use reliable methods of contraception during study treatment and for 3 months after study treatment termination.

Reliable methods of contraception are:

  1. Barrier type devices (e.g., female condom, diaphragm, contraceptive sponge) only in combination with a spermicide.
  2. Intra-uterine devices.
  3. Oral, injectable, transdermal or implantable contraceptives only in combination with a barrier method.

Hormone-based contraceptives alone, regardless of the route of administration, are not considered to be reliable methods of contraception.

Abstention, rhythm method, and contraception by the partner alone are not acceptable methods of contraception.

Women not of childbearing potential are defined as prepubescent, postmenopausal (i.e., amenorrhea for at least 1 year), or surgically or naturally sterile.

Signed written informed consent is obtained from the patient or patient's parent/ legal representative prior to initiation of any study-related procedure.

EXCLUSION CRITERIA

All patients (Groups A and B):

  1. Any major protocol violation in the preceding double-blind ASSET study*.
  2. Hemoglobin concentration less than 6.0 g/dL.

  3. Pregnancy or breast-feeding.

    * Protocol violations will be reviewed by the monitor during site visits and discussed with the study staff on an ongoing basis and at the patient's completion of the double-blind study.

    Group B only:

  4. Acute liver disease.
  5. Newly diagnosed cirrhosis or portal hypertension.
  6. ALT greater than or equal to 3 times ULN and/or albumin greater than 20% below LLN.
  7. Newly diagnosed psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00360087

  Show 24 Study Locations
Sponsors and Collaborators
Actelion
National Heart, Lung, and Blood Institute (NHLBI)
  More Information

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Sponsor, Actelion
ClinicalTrials.gov Identifier: NCT00360087     History of Changes
Other Study ID Numbers: AC-052-371
Study First Received: August 2, 2006
Last Updated: February 11, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by Actelion:
Treatment Study
Sickle Cell Anemia
6-Minute Walk
Right Heart Catheterization
 Tracleer
Sickle Cell
SCD

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Hypertension
Hypertension, Pulmonary
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
 Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Bosentan
Antihypertensive Agents
Cardiovascular Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013