Stem Cell Transplant for Sickle Cell Disease

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Columbia University

Disqualifiers: Pregnant, Infections, Liver fibrosis, others

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: 30

53 Participants Needed

Trial Summary

What is the purpose of this trial?

The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease. Patients will receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab followed by an infusion of stem cells, either from a family-related or cord-blood matched donor.

Research Team

Monica Bhatia, MD

Principal Investigator

Columbia University

Eligibility Criteria

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.

Inclusion Criteria

Age ≤30

Diagnosis of Homozygous Hemoglobin S Disease or Heterozygous Hemoglobin Sickle Cell (SC) or S 0/+ thalassemia, or Sickle/variant resulting in Chronic Hemolytic Anemia with hemoglobin (HgB) ≤10 mg/dL

Matched sibling donor and asymptomatic, or 8/8 human leukocyte antigen (HLA) matched unrelated adult donor

Exclusion Criteria

Evidence of uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms) within 1 month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen.

Karnofsky/Lansky Performance Score <60%

Histologic Exam of Liver (liver biopsy) with bridging fibrosis or cirrhosis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Chemotherapy

Participants receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab

4-6 weeks

Multiple visits for chemotherapy administration

Stem Cell Transplant

Participants undergo allogeneic stem cell transplant

1 week

Inpatient stay for transplant procedure

Follow-up

Participants are monitored for safety and effectiveness after treatment

10 years

Regular follow-up visits at Day 30, 60, 100, 180, 1 year, 2 years, 3 years, 5 years, 10 years

Treatment Details

Interventions

• Alemtuzumab
• Allogeneic stem cell transplant
• Busulfan
• Fludarabine

Trial Overview 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.

Participant Groups

Group I: SCD group Experimental Treatment 4 Interventions

Sickle Cell Disease patients receiving chemotherapy (Busulfan, Fludarabine and Alemtuzumab) will undergo allogeneic stem cell transplant.

Group II: BT group Experimental Treatment 4 Interventions

Beta Thalassemia patients receiving chemotherapy (Busulfan, Fludarabine and Alemtuzumab) will undergo allogeneic stem cell transplant.

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?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+