Search / Trial NCT00001749

Medical Treatment for Diamond Blackfan Anemia

Launched by NATIONAL HEART, LUNG, AND BLOOD INSTITUTE (NHLBI) · Nov 3, 1999

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Trial Information

Current as of April 22, 2024

Completed

Keywords

Immune Suppression Erythroid Hypoplasia Congenital Anemia Reticulocytopenia Diamond Blackfan Anemia

Description

Diamond Blackfan anemia (DBA) is a constitutional pure red cell aplasia of unknown etiology. There is laboratory evidence for an immune mechanism and most patients respond to corticosteroids. However the relapse and failure rate are high, and corticosteroids are associated with many short and long term side effects. Patients who do not respond or who do not tolerate corticosteriods require lifelong red blood cell transfusion and iron chelation therapy. Allogeneic bone marrow transplantation is an option for those with a related histocompatible donor, but this procedure is associated with hi...

Gender

All

Eligibility criteria

  • INCLUSION CRITERIA:
  • Diagnosis of DBA as characterized by a hyporegenerative anemia presenting in early childhood with reticulocytopenia, and low or absent erythroid precursors in the bone marrow.
  • Transfusion-dependence due to steroid failure or intolerance of steroid side effects.
  • Ineligible for or declining an allogeneic transplant.
  • Ages 3 to 75.
  • EXCLUSION CRITERIA:
  • Serum creatinine greater than 2 times normal or a creatinine clearance less than 50% normal.
  • SGPT or SGOT greater than 5 times normal.
  • History of epilepsy (any seizures besides childhood febrile seizures).
  • Current pregnancy or unwillingness to take oral contraceptives if menstruating.
  • Positive diepoxybutane (DEB) test for Fanconi anemia.
  • HIV positivity.
  • Inability or unwillingness to sign an informed consent, either by the patient, or in the case of a minor, by the parent or guardian responsible for the patient.
  • Underlying organ failure and/or those with a Karnofsky performance status of less than 1.
  • Treatment with androgens, prednisone greater than 10 mg/day, growth factors, or other immunosuppressive therapies within one month of protocol entry.
  • Ongoing treatment with Beta-adrenergic blocking drugs.
  • Previous treatment with ATG and concurrent CSA. Previous treatment with either drug alone is acceptable if greater than one year prior to study entry.

Attachments

readout_NCT00001749_2024-04-22.pdf

4.5 MB

NCT00001749_study_protocol.pdf

4.5 MB

About company

The National Institute of Allergy and Infectious Diseases (NIAID, /ˈnaɪ.æd/) is one of the 27 institutes and centers that make up the National Institutes of Health (NIH), an agency of the United States Department of Health and Human Services (HHS). NIAID's mission is to conduct basic and applied research to better understand, treat, and prevent infectious, immunologic, and allergic diseases.

Contacts

JC

Jennifer Cobb

Immunology at National Institute of Allergy and Infectious Diseases (NIAID)

Locations

Bethesda, Maryland, United States

People applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Reviews (48)

4.6

All reviews come from applied patients

5 stars
41
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Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Michael Foster
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Dries Vincent
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

Leslie Alexander
20 September 2023

Duis aute irure dolor in reprehenderit in voluptate velit esse cillum dolore eu fugiat nulla pariatur. Excepteur sint occaecat cupidatat non proident, sunt in culpa qui officia deserunt mollit anim id est laborum?

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