Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00295971
• Date of registration: 23/02/2006
• Prospective Registration: No
• Primary sponsor: University of California, San Francisco
• Public title: Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease
• Scientific title: Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases
• Date of first enrolment: April 2005
• Target sample size: 21
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00295971
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Morton J. Cowan, MD
• Affiliation: University of California, San Francisco

Key inclusion & exclusion criteria

DISEASE CHARACTERISTICS:

- Diagnosis of one of the following:

- Acute lymphoblastic leukemia in = 2nd remission or delayed remission induction

- High-risk myelodysplastic syndromes

- Refractory anemia with excess blasts (RAEB)

- RAEB in transformation

- Chronic myelogenous leukemia in second chronic phase

- No accelerated phase (> 5% blasts in marrow)

- Juvenile myelomonocytic leukemia

- Acute nonlymphoblastic leukemia in > 1st remission or induction failure and <
30% blasts in marrow

- Severe aplastic anemia, defined as absolute neutrophil count < 500/mm^3 and
platelet and/or red blood cell transfusion dependent

- Unresponsive to immunosuppressive therapy

- No Fanconi's anemia

- Congenital marrow aplasias unresponsive to cytokines and transfusion dependent

- Inherited immunodeficiency disease involving neutrophils or lymphocytes,
including any of the following:

- Chediak-Higashi disease

- Wiskott-Aldrich syndrome

- Combined immunodeficiency disease (Nezelof's)

- Hyper IgM syndrome

- No relapsed disease

- Haplocompatible related donor, including parent, cousin, aunt, uncle, grandparent,
half-sibling, or sibling (= 12 years of age), available

- 2 or 3 HLA antigen mismatch

- At least a 3 HLA antigen genotypic match

- No closely matched related or unrelated donor available in sufficient time to do
the transplant

PATIENT CHARACTERISTICS:

- No active hepatitis or cytomegalovirus infection

- Cardiac ejection fraction = 30%

- Creatinine clearance = 70 mL/min

- DLCO = 70% of predicted

- No active infection

- No HIV positivity

PRIOR CONCURRENT THERAPY:

- See Disease Characteristics



Age minimum: 1 Year
Age maximum: 17 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Myelodysplastic Syndromes

Congenital Amegakaryocytic Thrombocytopenia

Severe Congenital Neutropenia

Leukemia

Intervention(s)

Drug: thiotepa

Biological: anti-thymocyte globulin

Drug: fludarabine phosphate

Procedure: allogeneic hematopoietic stem cell transplantation

Procedure: in vitro-treated peripheral blood stem cell transplantation

Radiation: total-body irradiation

Procedure: allogeneic bone marrow transplantation

Biological: therapeutic allogeneic lymphocytes

Primary Outcome(s)

Engraftment at 4 weeks post bone marrow transplantation through 100 days [Time Frame: 100 days]

Secondary Outcome(s)

Disease-free survival and infection assessed monthly for 6 months, every 3 months for 2 years, every 6 months for 1 year, and then yearly for 5 years post transplantation [Time Frame: 1 year]

CD4 count in blood < 100/mm³ at 12 weeks [Time Frame: 12 weeks]

Graft-versus-host disease assessed monthly for 6 months, every 3 months for 2 years, every 6 months for 1 year, and then yearly for 5 years post transplantation [Time Frame: 2 years]

Survival assessed monthly for 6 months, every 3 months for 2 years, every 6 months for 1 year, and then yearly for 5 years post transplantation [Time Frame: 1 year]

Secondary ID(s)

UCSF-H411-17122-07

UCSF-01151

CDR0000462168

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Cancer Institute (NCI)