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Monoclonal Antibodies

Bone Marrow Transplant for Sickle Cell Disease

Phase 2

Recruiting

Led By Ann Haight, MD

Research Sponsored by Emory University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

- Previous clinical stroke, defined as a neurological deficit lasting longer than 24 hours plus new finding on head CT or brain MRI/MRA

Patients must meet criteria for symptomatic SCD as defined below

Must not have

- Patient's parent(s) or legal guardian is unable to understand the nature and the risks inherent in the HSCT process

- Donor who for psychological, physiologic, or medical reasons is unable to tolerate a bone marrow harvest or receive general anesthesia

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 2

Awards & highlights

Approved for 5 Other Conditions

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is testing a bone marrow transplant as a possible treatment for sickle cell disease in children. Eligible participants will undergo a pre-transplant evaluation, conditioning, and then the transplant itself, followed by routine care and checkups for at least 5 years.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects include weakened immune system leading to increased risk of infections; nausea; hair loss; mouth sores due to chemotherapy; and graft-versus-host disease where the new cells may attack the recipient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I have symptoms of sickle cell disease.

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I have had 2 or more episodes of acute chest syndrome in my life.

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I have had at least 3 acute chest syndrome events requiring blood transfusions.

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I am between 2 and 12 years old with a sickle cell disease.

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I've had 2 or more painful episodes needing strong painkillers at a hospital or clinic.

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I do not have symptoms of HbSS or HbSβ°thalassemia.

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I have had at least 3 episodes of acute chest syndrome or pain crises in my life.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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

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I have severe lung disease and may need oxygen during the day.

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My heart's pumping ability is reduced.

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I have severe liver scarring.

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I do not have severe neurological impairments, except possibly hemiplegia.

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My kidney function is below 50% of the expected level for my age.

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I do not have any active infections.

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My daily activities are significantly limited.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 2

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 2

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 2 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Immune Suppression-free, Rejection-free Survival

Secondary study objectives

CMV Invasive Disease

Chronic GVHD

Cytomegalovirus (CMV) Viremia

+12 more

Awards & Highlights

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

FDA approved

Alemtuzumab

FDA approved

Melphalan

FDA approved

0 / 17Eligibility criteria met