Stem Cell Transplantation for Sickle Cell Anemia
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|ClinicalTrials.gov Identifier: NCT01877837|
Recruitment Status : Active, not recruiting
First Posted : June 14, 2013
Last Update Posted : August 6, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease||Drug: Alemtuzumab Drug: Fludarabine Drug: Melphalan Procedure: Stem Cells||Phase 3|
1) To determine disease free survival (DFS) at one year after matched sibling transplant using either bone marrow (BM), peripheral blood stem cells (PBSC), or umbilical cord blood (UCB) after a conditioning regimen consisting of Alemtuzumab, Fludarabine, and Melphalan in patients 2-30 y/o
- Overall survival
- Rate of neutrophil and platelet engraftment for BM vs. UCB
- Incidence of graft failure
- Incidence of grade II-IV and grade III-IV acute graft vs host disease (GVHD)
- Incidence of chronic GVHD
- Incidence of other transplant complications, such as veno-occlusive disease, central nervous system (CNS) toxicity, and idiopathic pneumonia syndrome (IPS)
- Incidence of reactivation of CMV, EBV, adenovirus, BK/JC virus
- Incidence of invasive fungal disease
- Time to immune reconstitution via monitoring of lymphocyte subpopulations and immunoglobulin levels
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||26 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Reduced Intensity Stem Cell Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old|
|Study Start Date :||June 2011|
|Estimated Primary Completion Date :||September 2019|
|Estimated Study Completion Date :||September 2020|
Experimental: Related donor
Matched sibling donors (9-10/10 marrow/PBSC or 5-6/6 UCB (single) with a total TNC dose of greater than 5 x 107/kg recipient weight), age 2-30 years after conditioning regimen Alemtuzumab , Fludarabine, and Melphalan.
1) Patients will receive a conditioning regimen composed of Alemtuzumab, Fludarabine, and Melphalan as detailed in the table below.
Adjusted Ideal Body Weight Formula: AIBW = IBW + [(0.4) x (ABW - IBW)]
i.) Alemtuzumab I. Hb S% must be < or = 45% within 7 days prior to initiation of Alemtuzumab II. Iron chelation and hydroxyurea must be discontinued >48 hours before initiating therapy III. Alemtuzumab will be diluted in 100mL of 0.9% NS and infused at a rate as below
Other Name: Alemtuzumab (Campath)
I. Fludarabine should be diluted in 100 ml 0.9%NS and given over 30 minutes. II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Fludarabine
Other Name: Fludarabine (Fludara)
I. Melphalan should be diluted in 0.9%NS to a concentration of 0.1 -0.45 mg/mL and given over 45 minutes. *Entire dose must be infused within 60 minutes of reconstitution in Pharmacy.
II. A daily dose of an antiemetic should be given 30 minutes prior to administration of the Melphalan III. Patients should be encouraged to suck on a popsicle or something similar during the Melphalan infusion.
Other Name: Melphalan (Alkeran)
Procedure: Stem Cells
Infusion of Hematopoietic Stem Cells
- Graft Failure [ Time Frame: 1 year ]
In each group, the DFS at the 1 year endpoint will be estimated using the Kaplan Meier product limit estimator. The frequencies of the events, ie graft failure, will be enumerated and presented.
- Overall survival [ Time Frame: 2 years ]
Overall survival: The distribution of time to death from any cause will be estimated by Kaplan- Meier product limit function and plotted. The overall survival will be measured from the time of transplant to any death and patients will be followed for 2 years.
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): NCT01877837
|United States, New Jersey|
|Hackensack University Medical Center|
|Hackensack, New Jersey, United States, 07601|
|Principal Investigator:||Jennifer Krajewski, MD||Hackensack Meridian Health|