Stem Cell Transplantation for Sickle Cell Anemia
|ClinicalTrials.gov Identifier: NCT01877837|
Recruitment Status : Active, not recruiting
First Posted : June 14, 2013
Last Update Posted : April 27, 2020
|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 :||June 2020|
|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.
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|