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Monoclonal Antibodies

Siplizumab for Sickle Cell Anemia (CD2 SCD Trial)

Phase 1 & 2

Recruiting

Led By Markus Y Mapara, MD, PhD

Research Sponsored by Markus Mapara

Eligibility Criteria Checklist

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

Must have

Chronic kidney disease including patients on hemo-dialysis

Clinically significant neurologic event (stroke) or any neurological deficit lasting at least 24 hours. Stroke will be defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI or cerebral arteriopathy requiring chronic transfusion therapy.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months post stem cell transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial will assess the safety and effectiveness of siplizumab for SCD patients undergoing allogeneic stem cell transplants to reduce GVHD and graft failure.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects include allergic reactions to siplizumab which are mitigated by pre-medications. Other risks may come from associated treatments such as infections due to immune suppression by drugs like cyclophosphamide and complications related to stem cell infusion.

Eligibility Criteria

Inclusion Criteria

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

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I have chronic kidney disease or am on dialysis.

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I have had a stroke or lasting neurological issues confirmed by an MRI.

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I've had 3 or more severe pain crises a year for the last 2 years despite treatment.

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I can do most of my daily activities by myself.

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

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I have had at least 2 long-lasting erections involving all parts of the penis.

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I receive regular blood transfusions to prevent complications from my condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months post stem cell transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months post stem cell transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months post stem cell 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

Failure Rate

Secondary study objectives

Incidence of GVHD of Any Grade

Incidence of Significant transplant-related infections

Incidence of other transplant related toxicities

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: SiplizumabExperimental Treatment8 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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Siplizumab

2009

Completed Phase 1

~20

Total Body Irradiation

2006

Completed Phase 3

~820