CompletedNot Applicable

Stem Cell Transplantation (SCT) for Genetic Diseases

United StatesStarted 1995-01

Plain-language 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.

Who can participate

Age range0 Years – 17 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Hereditary enzymopathies, such as: Metachromatic leukodystrophy * Congenital Immunodeficiencies * Heritable hematologic disorders, such as: Thalassemia major Refractory Diamond-Blackfan anemia Fanconi anemia Amegakaryocytic thrombocytopenia --Patient Characteristics-- * Age: Under 18 * Other: SCT is performed using a histocompatible related donor, an unrelated donor, or an unrelated cord blood donor Haploidentical donors are accepted for patients with severe congenital immunodeficiency

Trial details

NCT IDNCT00004378

SponsorNational Center for Research Resources (NCRR)

Sponsor typeNIH

Study typeINTERVENTIONAL

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