Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
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|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|
|Primary Completion Date :||December 2005|
|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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|