Overview

This trial is active, not recruiting.

Condition sickle cell disease
Treatment biological: bone marrow transplant
Phase phase 2
Sponsor Emory University
Collaborator National Institutes of Health (NIH)
Start date March 2012
End date December 2017
Trial size 15 participants
Trial identifier NCT01565616, 1R34HL108761-01, IRB00068287

Summary

The primary goal of this multicenter study is to determine the safety and feasibility of a Bone Marrow Transplantation (BMT) in young adults with severe sickle cell disease (SCD) using a reduced toxicity conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/ anti-thymocyte globulin (ATG). A two-component design will be used for this study. The first component will be restricted to patients who have an HLA-identical sibling donor. Five patients will be enrolled during the first component of the study. The second component of enrollment will include patients who have a related or an unrelated HLA-matched donor. Up to 10 additional patients will be enrolled in this component of the study.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Other)
Patients undergoing bone marrow transplant for sickle cell disease
biological: bone marrow transplant
The bone marrow transplant regimen is below. Day 0 is the day of the transplant. The - sign is the number of days before and the + sign is the number of days after the transplant. SCHEMA OF CONDITIONING REGIMEN Day Treatment (BU- Busulfan, FLU-Fludarabine, ATG-Rabbit Anti-thymocyte globulin) Day -8 BU 3.2 mg/ kg/dose IV Day -7 BU 3.2 mg/kg/dose IV, FLU 35mg/m2 IV Day -6 BU 3.2 mg/kg/dose IV, FLU 35mg/m2 IV, ATG 0.5mg/kg IV Day -5 BU 3.2 mg/kg/dose IV, FLU 35mg/m2 IV, ATG 1.0mg/kg IV Day -4 FLU 35mg/m2 IV, ATG 1.5mg/kg IV Day -3 FLU 35mg/m2 IV, ATG 1.5mg/kg IV Day -2 ATG 1.5mg/kg IV Day -1 Rest Day 0 Stem cell infusion GVHD Regimen Day -3 Calcineurin Inhibitor (Cyclosporine or Tacrolimus) therapeutic doses through day 180, then taper Day 0 Stem cell infusion Day +1 Methotrexate 7.5 mg/m2 IV Day +3 Methotrexate 7.5 mg/m2 IV Day +6 Methotrexate 7.5 mg/m2 IV Day+11 Methotrexate 7.5 mg/m2 IV

Primary Outcomes

Measure
Event-free survival (EFS)
time frame: 1 year

Secondary Outcomes

Measure
Transplant-related outcomes
time frame: 1 year

Eligibility Criteria

Male or female participants from 16 years up to 40 years old.

Inclusion Criteria: - Diagnosis of severe sickle cell disease is denoted by one of the following: - Clinically significant neurologic event or any neurological deficit lasting greater than 24 hours - 2 or more episodes of acute chest syndrome in the last 2 years despite the use supportive care measures - 3 or more severe vaso-occlusive pain episodes per year in the last 2 years despite supportive care measures - Receives regular RBC transfusion therapy, defined as 8 or more transfusions per year for 1 year or longer - Echocardiograph finding of Tricuspid Regurgitation Jet (TRJ) velocity of 2.7 m/sec or greater - Adequate physical function - Must have an 8 of 8 HLA-A, B, C, and DRB1 allele matched related or unrelated bone marrow donor Exclusion Criteria: - Patients with cirrhosis of the liver, uncontrolled bacterial, viral or fungal infection in the past month, or seropositivity for HIV - Patients who have received prior HCT - Females who are pregnant or breast feeding

Additional Information

Official title A Phase II Study of Hematopoietic Stem Cell Therapy for Young Adults With Severe Sickle Cell Disease
Principal investigator Lakshmanan Krishnamurti, MD
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Emory University.