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Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

N/A

Waitlist Available

Led By Michele Nassin, MD

Research Sponsored by University of Chicago

Eligibility Criteria Checklist

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

Must have

Partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait) phenotype

Hemoglobin SS, SC, S-β0 Thalassemia, or SO-Arab Sickle Cell Disease

Must not have

Severe renal impairment (as evidenced by creatinine clearance of <50ml/minute glomerular filtration rate (GFR) < 50% predicted normal)

Cardiac function that demonstrates shortening fraction less than 26% by cardiac echocardiogram or pulmonary hypertension.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial will test whether a stem cell transplant can be a safe and effective treatment for sickle cell disease.

Who is the study for?

This trial is for children and young adults aged 2-25 with severe forms of sickle cell disease who lack a fully matched stem cell donor. Eligible participants may have had strokes, frequent pain episodes, or acute chest syndrome despite treatment. They must not be pregnant, lactating, or have certain organ dysfunctions like severe heart or kidney problems.

What is being tested?

The study tests a new way to perform stem cell transplants in patients with severe sickle cell disease using αβ+ T-cell depletion from partially-matched family donors. It aims to establish if this method can safely cure the condition without requiring a perfect donor match.

What are the potential side effects?

Potential side effects are not explicitly listed but may include typical transplant-related complications such as immune reactions against the host's body (graft-versus-host disease), infections due to weakened immunity post-transplant, and other organ-specific issues.

Eligibility Criteria

Inclusion Criteria

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

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I have a family member who is a partial match and has normal or sickle trait blood.

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I have a specific type of sickle cell disease.

Exclusion Criteria

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

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My kidney function is severely impaired.

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My heart's pumping ability is reduced, or I have high blood pressure in the lungs.

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I have acute hepatitis or severe liver damage, confirmed by a biopsy.

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My oxygen levels are low or my lungs struggle to transfer oxygen into my blood.

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I need assistance with my daily activities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year post transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post transplant for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Safety, as Measured by Incidence of Graft Failure, Grade III/IV Irreversible End Organ Toxicity, Grade III/IV aGvHD, or Death Within 100 Days Post-Hap-HSCT

Secondary study objectives

Estimate 1-year Overall and Event-free Survival After Hap-HSCT

Observe Incidence of Severe Acute GvHD as Defined by Grades III Through IV

Observe Incidence of Severe Chronic GvHD as Defined by Grades III and IV

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