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

February 11, 2010 updated by: Actelion

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)

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment

236

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Amsterdam, Netherlands
        • Amsterdam Medical Center
  • United Kingdom
      • London, United Kingdom
        • Royal Free Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama
    • California
      • Los Angeles, California, United States, 90095
        • University of California, Los Angeles
    • Colorado
      • Denver, Colorado, United States, 80220-3706
        • University of Colorado
    • Illinois
      • Chicago, Illinois, United States, 60612
        • University of Illinois
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • National Heart, Lung and Blood Institute (NHLBI), 9000 Rockville Pike
    • Massachusetts
      • Boston, Massachusetts, United States, 02118-2354
        • Boston University School of Medicine
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health Systems
      • Detroit, Michigan, United States, 48201
        • Wayne State University Hutzel Hospital
    • Missouri
      • St. Louis, Missouri, United States, 63104
        • St. Louis University
    • New York
      • Bronx, New York, United States, 10461
        • Albert Einstein College Of Medicine
      • New York, New York, United States, 10032-3784
        • Columbia University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599-7030
        • University of North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • University Hospitals of Ohio
      • Columbus, Ohio, United States, 43210-1240
        • Ohio State University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University
      • Philadelphia, Pennsylvania, United States, 19107-6541
        • Thomas Jefferson University
    • Tennessee
      • Memphis, Tennessee, United States, 38163
        • University of Tennessee
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
      • Houston, Texas, United States, 77030
        • University of Texas, Houston
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Actelion

Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2006

Primary Completion (Actual)

August 1, 2007

Study Completion (Actual)

December 1, 2007

Study Registration Dates

First Submitted

August 2, 2006

First Submitted That Met QC Criteria

August 2, 2006

First Posted (Estimate)

August 3, 2006

Study Record Updates

Last Update Posted (Estimate)

February 12, 2010

Last Update Submitted That Met QC Criteria

February 11, 2010

Last Verified

February 1, 2010

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AC-052-371

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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