Alemtuzumab intravenously, low dose total body irradiation, Sirolimus for Sickle Cell Disease

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Sickle Cell DiseaseAlemtuzumab intravenously, low dose total body irradiation, Sirolimus - Drug
Eligibility
2 - 25
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new transplant method for children with sickle cell disease that may be less toxic.

Eligible Conditions
  • Sickle Cell Disease

Treatment Effectiveness

Study Objectives

1 Primary · 1 Secondary · Reporting Duration: Day +30, and day +100 post transplant

100 days post transplant
Acute GVHD
Platelet transfusion
Day +30, and day +100 post transplant
Patient-Reported Outcomes Measurement Information System (PROMIS)
PedsQL 4.0 Measurement model for the Pediatric Quality of Life Inventory

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

SUN regimen
1 of 1

Experimental Treatment

30 Total Participants · 1 Treatment Group

Primary Treatment: Alemtuzumab intravenously, low dose total body irradiation, Sirolimus · No Placebo Group · Phase 2

SUN regimen
Drug
Experimental Group · 1 Intervention: Alemtuzumab intravenously, low dose total body irradiation, Sirolimus · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: day +30, and day +100 post transplant

Who is running the clinical trial?

Allistair Abraham, MDLead Sponsor
Alberta Children's HospitalOTHER
50 Previous Clinical Trials
38,914 Total Patients Enrolled
The Hospital for Sick ChildrenOTHER
642 Previous Clinical Trials
6,816,489 Total Patients Enrolled
Levine Children's HospitalOTHER
2 Previous Clinical Trials
451 Total Patients Enrolled
Ann & Robert H Lurie Children's Hospital of ChicagoOTHER
232 Previous Clinical Trials
5,178,228 Total Patients Enrolled

Eligibility Criteria

Age 2 - 25 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a history of cerebral infarction on brain MRI.
You have had two or more episodes of acute chest syndrome (ACS) in lifetime.
You have a history of hospitalization for SCD pain or ACS while receiving hydroxyurea treatment.