Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis

• ClinicalTrials.gov Identifier: NCT00000595
• Recruitment Status: Completed
• First Posted: October 28, 1999
• Last Update Posted: November 26, 2013
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Information provided by: National Heart, Lung, and Blood Institute (NHLBI)

Tracking Information

• First Submitted Date: October 27, 1999
• First Posted Date: October 28, 1999
• Last Update Posted Date: November 26, 2013
• Study Start Date: January 1978
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures: Not Provided
• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
• Official Title: Not Provided
• Brief Summary: To determine whether deferoxamine prevented the complications of transfusional iron overload.

Detailed Description

BACKGROUND:

The prognosis of congenital or long-term anemia was formerly limited by the complications of blood transfusion, splenectomy, or infection, problems now largely overcome by sophisticated clinical care. Lifespan is now determined by the rate of myocardial iron deposition, with death occurring from cardiac failure or arrhythmia, usually between the ages of 15 and 25. Endocrine complications and hepatic enlargement are also evident by this age. Deferoxamine increases urinary iron excretion and is the only chelator currently available for chronic administration. Daily administration of deferoxamine results in negative iron balance in most patients by the age of 10; this study was designed to determine whether the onset of cardiac complications was delayed and life prolonged by iron removal.

This trial began in 1978. Its forerunner was a study involving both deferoxamine and ascorbic acid. Although ascorbic acid promotes iron removal, its administration was followed by cardiac deterioration in several patients. In this study, patients receiving subcutaneous deferoxamine were randomized to receive either ascorbic acid or placebo, thereby providing a controlled test of this agent in treatment of iron overload. Sixty-five patients with homozygous beta-thalassemia participated in the long-term chelation trial. Of these, 49 were randomized to the ascorbic acid trial.

Several noninvasive techniques have been developed to evaluate organ function in iron-overloaded patients, thereby facilitating the assessment of chelation therapy. These techniques included chest x-rays, electrocardiograms, echocardiograms, and 24-hour Holter monitoring to assess cardiac function. Liver function was evaluated by standard liver function tests, CAT scan, and live biopsy. During the last six years of the study, hepatic iron stores were measured magnetically with a dual channel superconducting quantum-interference susceptomer. Endocrine function was also assessed by standard tests.

DESIGN NARRATIVE:

All patients received subcutaneous deferoxamine and iron removal was determined by measurement of serum ferritin and periodic non-invasive measurements of liver iron concentration. Clinical status was evaluated by non-invasive testing of cardiac and endocrine function.

The study completion date listed in this record was inferred from the last publication listed in the Citations section of this study record.

• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Primary Purpose: Treatment

Condition

• Anemia (Iron-Loading)
• Beta-Thalassemia
• Hematologic Diseases
• Hemoglobinopathies
• Thalassemia
• Iron Overload
• Hemochromatosis

• Intervention: Drug: deferoxamine
• Study Arms: Not Provided

Publications *

• Nienhuis AW, Griffith P, Strawczynski H, Henry W, Borer J, Leon M, Anderson WF. Evaluation of cardiac function in patients with thalassemia major. Ann N Y Acad Sci. 1980;344:384-96. doi: 10.1111/j.1749-6632.1980.tb33677.x.
• Brittenham GM, Griffith PM, Nienhuis AW, McLaren CE, Young NS, Tucker EE, Allen CJ, Farrell DE, Harris JW. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. N Engl J Med. 1994 Sep 1;331(9):567-73. doi: 10.1056/NEJM199409013310902.

Recruitment Information

• Recruitment Status: Completed
• Enrollment: Not Provided
• Original Enrollment: Not Provided
• Actual Study Completion Date: September 1994
• Primary Completion Date: Not Provided
• Eligibility Criteria: Males and females, 5 years or older, with transfusional hemochromatosis.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 5 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT00000595
• Other Study ID Numbers: 401
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Not Provided
• Original Responsible Party: Same as current
• Current Study Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Investigator: Neal Young; Laboratory of Hematology, NHLBI

• PRS Account: National Heart, Lung, and Blood Institute (NHLBI)
• Verification Date: May 2000