Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
|ClinicalTrials.gov Identifier: NCT00003336|
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : June 11, 2010
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
|Condition or disease||Intervention/treatment||Phase|
|Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases||Biological: anti-thymocyte globulin Drug: busulfan Drug: cyclophosphamide Drug: melphalan Drug: methylprednisolone Procedure: bone marrow ablation with stem cell support Procedure: umbilical cord blood transplantation Radiation: radiation therapy||Phase 2|
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders|
|Study Start Date :||January 1998|
|Actual Primary Completion Date :||December 2005|
|Actual Study Completion Date :||February 2006|
Biological: anti-thymocyte globulin
- Event-free survival by disease assessment [ Time Frame: at 100 days and at 6, 9, 12, 18, and 24 months ]
- Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery. [ Time Frame: 100 days and at 6, 9, 12, 18, and 24 months ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00003336
|United States, Ohio|
|Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center|
|Cleveland, Ohio, United States, 44106-7284|
|Principal Investigator:||Mary J. Laughlin, MD||Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center|