Siplizumab for Sickle Cell Anemia

(CD2 SCD Trial)

Overseen By Markus Y. Mapara

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Markus Mapara

Disqualifiers: Pulmonary dysfunction, Severe cardiac dysfunction, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 5

18 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to find out whether siplizumab is safe and effective for patients with SCD undergoing an allogeneic transplant and to prevent development of Graft versus Host Disease (GVHD) and graft failure. The main goals of this study are : * To determine if acute GVHD occurs and how severe the acute GVHD is in subjects receiving the study drug * To determine if graft failure occurs in subjects receiving the study drugs In this study, participants will receive 5 infusions of the study drug, siplizumab, while getting a stem cell transplant for SCD. Before siplizumab infusion, participants will be given medications to reduce the risks of allergic reaction to the drug.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, since the study involves a stem cell transplant, it's possible that some medications might need to be adjusted. Please consult with the study team for specific guidance.

Research Team

Markus Mapara, MD

Principal Investigator

Columbia University

Eligibility Criteria

This trial is for people with Sickle Cell Disease (SCD) who've had a stroke, frequent pain crises or acute chest syndrome despite treatment, need regular blood transfusions, have heart issues like high blood flow velocity through the tricuspid valve or pulmonary hypertension, kidney disease including those on dialysis, recurrent priapism, and are not pregnant. They should be fairly active (ECOG status of 2 or better) and have a left ventricular ejection fraction of 40% or greater.

Inclusion Criteria

I have chronic kidney disease or am on dialysis.

Recipient cannot be pregnant or lactating.

My heart pumps well, with an ejection fraction of 40% or more.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning and Transplantation

Participants receive 5 infusions of siplizumab and undergo stem cell transplantation

3 weeks

5 visits (in-person)

Initial Follow-up

Participants are monitored for graft failure, GVHD, and other complications

100 days

Regular visits (in-person)

Extended Follow-up

Participants are monitored for long-term outcomes including immune reconstitution and GVHD

12 months

Treatment Details

Interventions

• Siplizumab

Trial Overview The study tests if siplizumab can safely prevent Graft versus Host Disease (GVHD) and graft failure in patients receiving stem cell transplants for SCD. Participants will get five infusions of siplizumab along with other treatments like cyclophosphamide and total body irradiation to support the transplant process.

Participant Groups

Group I: Siplizumab Experimental Treatment 8 Interventions

Participants will receive 5 infusions of siplizumab. The first dose is given 14 days prior to the infusion of stem cells; the second dose is given 6 days before infusion; and doses 3, 4, and 5 are given on the day before, day of, and day after stem cell infusion.

Who Is Running the Clinical Trial?

Markus Mapara

Lead Sponsor

Trials

1

Recruited

20+

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

ITB-Med LLC

Industry Sponsor

Trials

12

Recruited

390+