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Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL

Last updated 2005-06-24

No results posted yet for this study

Summary

OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism.

II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.

Conditions

  • Thrombocytopenia
  • Metachromatic Leukodystrophy
  • Fanconi's Anemia
  • Thalassemia Major
  • Pure Red-Cell Aplasia
  • Inborn Errors of Metabolism

Interventions

PROCEDURE

Stem Cell Transplantation

Sponsors & Collaborators

Principal Investigators

  • Stephen A. Feig · University of California, Los Angeles

Study Design

Purpose
TREATMENT

Eligibility

Min Age
0 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1995-01-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00004378 on ClinicalTrials.gov