← Back to Search

Monoclonal Antibodies

Stem Cell Transplant for Sickle Cell Disease

Phase 1 & 2
Recruiting
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 10 years post-transplant.
Awards & highlights

Study 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.Check my eligibility
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.See study design
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 weeks
Treatment ~ Varies
Follow Up ~up to 10 years post-transplant.
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 10 years post-transplant. for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
To determine the engraftment after non-myeloablative HSC transplant
Secondary outcome measures
To assess the frequency of acute and chronic complications of sickle cell disease
To determine the long-term engraftment after non-myeloablative HSC transplant
To determine the transplant related morbidity and mortality.
+1 more
Other outcome measures
To determine whether ocular findings from sickle cell disease are reversible in patients undergoing stem cell transplantation to treat their sickle cell disease.

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
Sirolimus
2013
Completed Phase 4
~2750
Alemtuzumab
2004
Completed Phase 4
~1890

Find a Location

Who is running the clinical trial?

University of Illinois at ChicagoLead Sponsor
609 Previous Clinical Trials
1,559,294 Total Patients Enrolled
Damiano Rondelli, MDPrincipal Investigator - University of Illinois at Chicago
University of Illinois at Chicago Eye & Ear Infirmary
6 Previous Clinical Trials
270 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)
Damiano Rondelli, MD 2011. "Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01499888.
All > Sickle Cell Disease > Sickle Cell Anemia
~1 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top