Leucovorin for the Treatment of 5 q Minus Syndrome

This study has been completed.
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00004997
First received: March 22, 2000
Last updated: March 3, 2008
Last verified: July 2002
  Purpose

The 5 q minus syndrome is a condition that occurs due to a missing segment of chromosome 5 in the normal genetic make-up of the cells responsible for forming blood cells. The condition causes patients to have the inability to make blood normally. Many patients with this syndrome need transfusions of red blood cells, platelets, and/or white blood cells. Low levels of platelets may cause the patient to bleed easily and low levels of whit blood cells make the patient susceptible to infections. A small number of patients with 5 q minus syndrome develop leukemia, which is often untreatable with chemotherapy.

Researchers believe that one of the genes missing in 5 q minus syndrome is the gene responsible for making folic acid active in the body. Folic acid is a vitamin required for normal blood production.

The purpose of this study to test the effectiveness of a drug called leucovorin for the treatment of 5 q minus syndrome. Leucovorin is an active form of the vitamin folic acid that does not require the missing genes to activate it.

Patients participating in this study may or may not improve with leucovorin treatment. However, the study will improve researchers understanding of the disease and may lead other potential therapies for the disease.


Condition Intervention Phase
5q Minus Syndrome
Myelodysplastic Syndrome
Drug: Leucovorin
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Primary Purpose: Treatment
Official Title: The Experimental Treatment of Transfusion Dependent 5q Minus Syndrome With Leucovorin

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 14
Study Start Date: April 1998
Estimated Study Completion Date: July 2002
Detailed Description:

The objective of this protocol is to determine whether leucovorin treatment can normalize hematopoietic cell growth and differentiation in patients with 5q- syndrome which may lack the gene for dihydrofolate reductase enzyme.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

All patients 18 years old and older with RA, RARS and RAEB who have a 5q deletion as their sole cytogenetic abnormality who require transfusion support with blood defined as needing at least one unit of packed red blood cells per month for greater than or equal to 2 months and/or thrombocytopenia defined as a platelet count less than 50,000, and/or severe neutropenia (absolute neutrophil count less than 500) are eligible, including patients that had been treated with chemotherapy or experimental agents such as retinoids, phenylbutyrate, amifostine, Vitamin D, ATG or hematopoietic growth factors.

Patients must not be transforming to acute leukemia (greater than 20% blasts in marrow aspirate).

Patients must not have had treatment with growth factors or any other experimental drug within 4 weeks prior to entry on protocol.

Patients must not have an ECOG performance status greater than 2.

Patients must not have an active uncontrolled infection.

No patients for whom bone marrow transplant is indicated as primary therapy.

Must be able to give informed consent.

Patients must not be HIV positive.

Patients must not be pregnant.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004997

Locations
United States, Maryland
Warren G. Magnuson Clinical Center (CC)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00004997     History of Changes
Other Study ID Numbers: 980101, 98-CC-0101
Study First Received: March 22, 2000
Last Updated: March 3, 2008
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Hematopoietic
DHFR Enzyme
Anemia
Folic Acid
MDS
Myelodysplasia
Myelodysplastic

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Leucovorin
Levoleucovorin
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs
Pharmacologic Actions
Antidotes
Protective Agents

ClinicalTrials.gov processed this record on August 28, 2014