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Hematopoietic Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

Phase 1

Recruiting

Led By Anna B. Pawlowska, MD

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

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

Must have

Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%

Significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; or increased transcranial Doppler velocity (>200 m/s)

Must not have

Patients with any active malignancy are ineligible for this study, other than non-melanoma skin cancers

Prior autologous or allogeneic transplant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying a new way to treat sickle cell disease using haploidentical donors (a donor with partial matching of the patient's HLA) and a new myeloablative preparative regimen.

Who is the study for?

This trial is for patients aged 1-30 with sickle cell anemia who have had severe symptoms like stroke, acute chest syndrome, or frequent pain crises despite treatment. They must not be pregnant, agree to use contraception, and have a half-matched (haploidentical) family donor because no fully matched donor is available.

What is being tested?

The study tests a new approach using two cycles of drugs to suppress the immune system before receiving stem cells from a half-matched family donor. The goal is to safely expand the pool of donors and achieve consistent results without causing harm.

What are the potential side effects?

Potential side effects include reactions related to immune suppression such as increased infection risk, possible damage to bone marrow function (myeloablation), and other complications that can arise from transplant procedures.

Eligibility Criteria

Inclusion Criteria

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

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I have sickle cell anemia with more than 60% Hgb S.

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I have had a stroke or a lasting neurological issue, or my brain blood flow speed is high.

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I've had one or more acute chest syndrome episodes in the last 2 years despite treatment.

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I have bone damage in two or more joints despite receiving care.

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I've had 8 or more blood transfusions yearly for over a year to prevent complications.

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I have a family donor who matches half of my genetic markers for a transplant.

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I've had severe pain crises yearly despite treatment.

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I have had repeated priapism treated with medication.

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I don't have a fully matched donor for a transplant.

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I am between 1 and 30 years old.

Exclusion Criteria

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

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I do not have any active cancer except for non-melanoma skin cancers.

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I have had a stem cell transplant from myself or a donor.

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I do not have any uncontrolled illnesses or infections.

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I am not currently on any experimental treatments or other cancer therapies.

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I have a donor who is a complete HLA match for me.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT

Secondary study objectives

Disease free survival rate

Event-free survival rate

Immune reconstitution at day 100, 180 and 365

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