Bone Marrow Transplant for Sickle Cell Disease

Recruiting at 22 trial locations

Overseen By Study Information

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Emory University

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

43 Participants Needed

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

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-3 months

Conditioning

Participants undergo conditioning with chemotherapy and other medicines to prepare for the transplant

3 weeks

Inpatient stay

Transplantation

Participants receive the bone marrow transplant

1 day

Inpatient stay

Post-Transplant Hospitalization

Participants remain in the hospital for recovery and monitoring after the transplant

4-6 weeks

Inpatient stay

Outpatient Follow-up

Participants are followed closely in the clinic with frequent visits for 6 to 12 months

6-12 months

Frequent clinic visits

Long-term Follow-up

Routine medical care and yearly examinations for many years after transplant

Up to 2 years

Treatment Details

Interventions

• Alemtuzumab
• Fludarabine
• Melphalan

Trial Overview The 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

Group I: Reduced Intensity Conditioning with FAM Experimental Treatment 3 Interventions

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

Alemtuzumab is already approved in United States, European Union for the following indications:

Approved in United States as Campath for:

• Chronic lymphocytic leukemia
• Multiple sclerosis

Approved in European Union as Lemtrada for:

• Multiple sclerosis

Approved in European Union as Campath for:

• Chronic lymphocytic leukemia

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials

1,735

Recruited

2,605,000+