Bone Marrow Transplant for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+22 other locations

Overseen byAnn Haight, MD

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Emory University

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This study aims to enroll 58 pre-adolescent (\<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant. It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant. Once they are found to be eligible for transplant, participants will be admitted to the hospital and will start transplant conditioning. Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells. It prevents the immune system from rejecting donor cells. Conditioning will start 21 days before transplant. Once they complete conditioning, participants will receive the bone marrow transplant. After the transplant, participants will stay in the hospital for 4-6 weeks. After they leave the hospital, participants will be followed closely in the clinic. Outpatient treatment and frequent clinic visits usually last 6 to 12 months. Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant. The researchers will collect and study information about participants for 2 years after transplant.

Research Team

Ann Haight, MD

Principal Investigator

Emory University

Eligibility Criteria

This trial is for pre-adolescent children under 13 with sickle cell disease who've had multiple episodes of acute chest syndrome or vaso-occlusive pain, and have a healthy sibling donor also under 13. Candidates must not have severe neurological impairments, organ dysfunction, HIV infection, or uncontrolled infections.

Inclusion Criteria

I have a brain condition without symptoms, confirmed by specific brain scans.

I have had a stroke with symptoms lasting over 24 hours and confirmed by brain scans.

- Co-enrollment on STAR Project Sickle Cure (PSC) study is required for sites that are activated and participating in the study

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Exclusion Criteria

My parent or guardian does not understand the risks of the HSCT process.

I cannot undergo a bone marrow harvest or general anesthesia due to health or personal reasons.

- Donor is HIV infected

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Treatment Details

Interventions

Alemtuzumab (Monoclonal Antibodies)
Fludarabine (Anti-metabolites)
Melphalan (Alkylating agents)

Trial OverviewThe study tests the effectiveness of a bone marrow transplant from an HLA-matched sibling in treating sickle cell disease. Participants will undergo chemotherapy (Fludarabine, Alemtuzumab, Melphalan) to prepare their body for the transplant.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Reduced Intensity Conditioning with FAMExperimental Treatment3 Interventions

Children with SCD will received reduced intensity conditioning with fludarabine, alemtuzumab and melphalan (FAM) during HSCT with a HLA matched sibling donor