Anemia (Iron-Loading)Phase 2NCT00000595
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
A Phase 2 study testing deferoxamine in people with Anemia (Iron-Loading) and Beta-Thalassemia, run by National Heart, Lung, and Blood Institute (NHLBI).
Trial summary
To determine whether deferoxamine prevented the complications of transfusional iron overload. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases
- What is being tested?
- deferoxamine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Jan 1978to Sep 1994
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Males and females, 5 years or older, with transfusional hemochromatosis.
About this study
To determine whether deferoxamine prevented the complications of transfusional iron overload.
Read the 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.
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies, Thalassemia, Iron Overload
- Being tested
- deferoxamine
- Medical subject headings
- Anemia, beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies, Thalassemia, Iron Overload, Hemochromatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferoxamineDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 1999 (estimated)
- Study startJan 1978
- Study completionSep 1994
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Anemia (Iron-Loading)Studies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- Neal Young, Laboratory of Hematology, NHLBI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00000595
- Sponsor's protocol ID
- 401
- Status last verified
- May 2000
- First submitted
- 27 Oct 1999
- Last updated
- 26 Nov 2013
- Results posted
- No
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.
PubMed 6930878Brittenham 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.
PubMed 8047080
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
- ClinicalTrials.gov ID
- NCT00000595
- Acronym / sponsor ID
- 401
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies, Thalassemia, Iron Overload
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Jan 1978
- Study completion
- Sep 1994 (actual)
- First posted
- 28 Oct 1999 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| deferoxamine | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2013.
- Background: PubMed 6930878Nienhuis 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.
- Background: PubMed 8047080Brittenham 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.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Neal Young
Laboratory of Hematology, NHLBI
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing deferoxamine in people with Anemia (Iron-Loading) and Beta-Thalassemia, run by National Heart, Lung, and Blood Institute (NHLBI).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Nov 2013. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00000595, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2013.