Leucovorin for the Treatment of 5 q Minus Syndrome

March 3, 2008 updated by: National Institutes of Health Clinical Center (CC)

The Experimental Treatment of Transfusion Dependent 5q Minus Syndrome With Leucovorin

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment

14

Phase

  • Phase 2

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

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892
        • Warren G. Magnuson Clinical Center (CC)

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

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

Collaborators and Investigators

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

Sponsor

National Institutes of Health Clinical Center (CC)

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

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

April 1, 1998

Study Completion

July 1, 2002

Study Registration Dates

First Submitted

March 22, 2000

First Submitted That Met QC Criteria

March 22, 2000

First Posted (Estimate)

March 23, 2000

Study Record Updates

Last Update Posted (Estimate)

March 4, 2008

Last Update Submitted That Met QC Criteria

March 3, 2008

Last Verified

July 1, 2002

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 980101
  • 98-CC-0101

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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