Immunoablative Mini Transplant (Hematopoietic Peripheral Blood Stem Cell Transplant [HPBSC])
Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC)
1 other identifier
interventional
68
1 country
1
Brief Summary
The purpose of this research study is to evaluate the effectiveness of transplantation of high doses of peripheral blood stem cells (stem cells are special cells found in the blood and bone marrow that produce new blood cells) after treatment with non-myeloablative chemotherapy (not toxic to the bone marrow). In addition, this study will assess the side effects of the transplant.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for not_applicable
Started Mar 2000
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 10, 2000
CompletedFirst Submitted
Initial submission to the registry
September 10, 2005
CompletedFirst Posted
Study publicly available on registry
September 16, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 24, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
January 24, 2014
CompletedAugust 30, 2019
August 1, 2019
13.9 years
September 10, 2005
August 28, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Evaluate the morbidity and mortality of matched related and unrelated hematopoietic progenitor cell (HPC) transplantation at Children's Memorial Hospital using high dose CD34+ HPCs after a reduced intensity conditioning regimen.
To study end
Determine the toxicity of a reduced intensity conditioning regimen consisting of Fludarabine and Busulfan.
To study end
Secondary Outcomes (4)
Validate the pharmacokinetics of once-a-day dosing of intravenous Busulfan given as a 3-hour infusion, using a limited number of samples.
To study end
Assess chimeric engraftment utilizing this regimen in malignant and non-malignant disorders.
To study end
Assess the relapse rate of patients transplanted with this reduced intensity regimen.
To study end
Determine the incidence of acute and chronic Graft vs. Host Disease (GVHD) using prophylaxis with Cyclosporine A and mycophenolate mofetil following this reduced intensity regimen.
To study end
Study Arms (1)
Reduced Intensity Conditioning Regimen
OTHERInterventions
Immunoablative conditioning chemotherapy regimen, followed by transplantation of peripheral blood stem cells on Day 0 of the conditioning regimen.
Pharmacokinetics of once-a-day dosing of intravenous busulfan as a 3-hour infusion
* Patients diagnosed with ALL over 1 year of age and without prior CNS disease will receive CNS prophylaxis radiation to the whole brain prior to transplant. * Patients diagnosed with ALL with prior CNS disease over the age of 1 year will be treated with prophylaxis radiation to the whole brain and spine prior to transplant.
Eligibility Criteria
You may qualify if:
- Patients with recurrent solid tumors
- Patients with malignant melanoma
- Patients with hematological malignancies.
- Chronic myelogenous leukemia in chronic or accelerated phase, to include chronic myelomonocytic leukemia (juvenile chronic myelogenous leukemia (JCML) or CMML).
- Acute lymphoblastic leukemia (ALL)
- First remission high-risk ALL (Ph+ with initial high white blood cell (WBC)t (4-11) in infants less than 1 year and CALLA negative)
- Second or subsequent remission ALL or isolated extramedullary disease on or off therapy.
- Acute non-lymphocytic leukemia (ANLL)
- Patients with ANLL in first remission who have a matched sibling donor.
- ANLL in second remission, or patients who only achieve an initial partial remission \< 15% blasts, or early relapse.
- Myelodysplastic syndromes (MDS): refractory anemia (RA), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T) and CMML/JCML.
- Selected immunodeficiencies:
- Wiskott-Aldrich syndrome.
- Severe combined immunodeficiency variants that require ablation.
- Hyper-Immunoglobulin M (IgM) syndrome.
- +10 more criteria
You may not qualify if:
- Patients who are pregnant
- Inability to find a suitable donor for the patient
- Patient is HIV-positive
- Patient has active Hepatitis B
- Disease progression or relapse prior to HPC infusion
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Lurie Children's Hospital
Chicago, Illinois, 60611, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Morris Kletzel, M.D.
Ann & Robert H Lurie Children's Hospital of Chicago
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- MD
Study Record Dates
First Submitted
September 10, 2005
First Posted
September 16, 2005
Study Start
March 10, 2000
Primary Completion
January 24, 2014
Study Completion
January 24, 2014
Last Updated
August 30, 2019
Record last verified: 2019-08