NCT01877837

Stem Cell Transplantation for Sickle Cell Anemia

Reduced Intensity Matched Sibling Bone Marrow Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Hackensack Meridian Health · Academic / Other
• Sex: All
• Age: 2 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of valine for glutamic acid at the sixth position in the B-chain of hemoglobin. This leads to sickling of the red blood cells under many conditions, such as hypoxia, dehydration, and hyperthermia. The sickling leads to vaso-occlusion, which causes irreversible damage in almost all systems in the body, including the central nervous system (CNS), lungs, heart, bones, eyes, liver, and kidneys.

Detailed description

Primary objective: 1\) To determine disease free survival (DFS) at two years after matched sibling transplant using bone marrow (BM) after a conditioning regimen consisting of distal timed Alemtuzumab, Fludarabine, and Melphalan for patients 2-30 y/o Secondary objectives: 1. Overall survival 2. Rate of neutrophil and platelet engraftment for BM 3. Incidence of graft failure 4. Incidence of grade II-IV and grade III-IV acute graft vs host disease (GVHD) 5. Incidence of chronic GVHD 6. Incidence of other transplant complications, such as veno-occlusive disease, central nervous system (CNS) toxicity, and idiopathic pneumonia syndrome (IPS) 7. Incidence of reactivation of CMV, EBV, adenovirus, BK/JC virus 8. Incidence of invasive fungal disease 9. Time to immune reconstitution via monitoring of lymphocyte subpopulations and immunoglobulin levels

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Alemtuzumab	Adjusted Ideal Body Weight Formula: AIBW = IBW + \[(0.4) x (ABW - IBW)\] b) Medications 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
DRUG	Fludarabine	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
DRUG	Melphalan	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.
PROCEDURE	Stem Cells	Infusion of Hematopoietic Stem Cells

Timeline

• Start date: 2011-06-01
• Primary completion: 2021-09-01
• Completion: 2021-09-01
• First posted: 2013-06-14
• Last updated: 2022-11-07
• Results posted: 2022-11-07

Locations

1 site across 1 country: United States