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Monoclonal Antibodies

Stem Cell Transplant for Sickle Cell Disease

Phase 1 & 2

Waitlist Available

Led By Damiano Rondelli, MD

Research Sponsored by University of Illinois at Chicago

Eligibility Criteria Checklist

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

Must have

* Stroke or central nervous system event lasting longer than 24 hours

* Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year severe enough to interfere with the patient's normal daily function or require medical attention in the clinic, emergency room, acute care center, or hospital

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 365 days post-transplant.

Awards & highlights

No Placebo-Only Group

Summary

This trial will assess a non-chemo transplant protocol to reduce complications in Sickle Cell Disease patients not suitable for hydroxyurea. Donor is sibling for reducing risk.

Who is the study for?

This trial is for individuals aged 16-60 with aggressive sickle cell disease who haven't responded well to or can't take hydroxyurea. Candidates must have a fully matched sibling donor, good heart and lung function, no HIV or chronic hepatitis, and be willing to sign consent. They should have had complications like frequent pain episodes, stroke, vision problems due to retinopathy, kidney issues, or severe anemia requiring transfusions.

What is being tested?

The study tests a less harsh stem cell transplant from fully matched siblings using immune-suppressing drugs (Alemtuzumab and Sirolimus) and low-dose radiation instead of standard chemo. The goal is to see if this method helps patients accept the new cells with fewer complications.

What are the potential side effects?

Possible side effects include reactions related to the immune system being suppressed such as increased risk of infections, potential liver damage indicated by elevated ALT levels, organ inflammation due to Alemtuzumab and Sirolimus use; also risks associated with low-dose total body irradiation.

Eligibility Criteria

Inclusion Criteria

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 365 days post-transplant.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 365 days post-transplant.

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 365 days post-transplant. for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

To assess the frequency of acute and chronic complications of sickle cell disease

To determine the transplant related morbidity and mortality.

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: Allogeneic Non-Myeloablative Stem Cell TransplantationExperimental Treatment3 Interventions

The transplant regimen will consist of alemtuzumab 1mg/kg divided over five days, 300 cGy TBI, followed by sirolimus dosed for a target serum trough level of 10- 15 ng/mL.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Alemtuzumab

2004

Completed Phase 4

~1880

Sirolimus

2013

Completed Phase 4

~2750