Stem Cell Transplant for Sickle Cell Disease

Phase 2

Waitlist Available

Led By Monica Bhatia, MD

Research Sponsored by Columbia University

Eligibility Criteria Checklist

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

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day +30, 60, 100, 180, 365, 730, and 1005

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Approved for 5 Other Conditions

Summary

This trial is testing whether lower doses of chemo, followed by a stem cell transplant, will be successful in treating a patient's disease with fewer side effects.

Who is the study for?

This trial is for individuals under 30 years old with severe forms of blood disorders like Sickle Cell Disease or Thalassemia, who haven't responded well to other treatments. They need a matched stem cell donor, good kidney and liver function, heart and lung health, and can't be pregnant or have uncontrolled infections.

What is being tested?

The study tests if lower doses of chemotherapy (busulfan, fludarabine) followed by alemtuzumab and a stem cell transplant from a related or unrelated donor can effectively treat the disease with fewer harsh side effects.

What are the potential side effects?

Possible side effects include reactions to chemotherapy such as nausea, fatigue, hair loss; immune reactions from alemtuzumab; complications from the transplant like graft-versus-host disease where donated cells attack your body.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 30, day 60, day 100, day 180, 1 year, 2 years, 3 years, 5 years, 10 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Prevalence of toxicity associated with moderately ablative therapy (busulfan/fludarabine/alemtuzumab) and allogeneic stem cell transplantation in selected patients with Sickle Cell Disease (SCD) and Beta Thalassemia (BT)

Secondary study objectives

Incidence of acute and chronic graft versus host disease (GVHD) following moderately ablative therapy and allogeneic stem cell transplantation in selected patients with SCD and BT

Incidence of primary and secondary graft failure

Percent of mixed donor chimerism

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