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Cell Therapy

Stem Cell Transplant for Sickle Cell Disease (HaploSCD Trial)

Phase 2
Waitlist Available
Led By Mitchell S Cairo, MD
Research Sponsored by New York Medical College
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients must demonstrate one or more of the following Sickle Cell Disease Complications: Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
Recurrent painful events (at least 3 in the 2 years prior to enrollment).
Must not have
Clinically significant fibrosis or cirrhosis of the liver
Females who are pregnant or breast-feeding
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 4 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying a high-dose chemotherapy regimen followed by an infusion of stem cells from a partially matched adult family member donor in treating patients with sickle cell disease.

Who is the study for?
This trial is for high-risk sickle cell disease patients who've had multiple painful events, adequate organ function, and specific complications like stroke or acute chest syndrome. They need a partially matched family donor and can't be pregnant, breastfeeding, non-compliant with medical care, have significant liver fibrosis/cirrhosis or previous stem cell transplants.
What is being tested?
The study tests the safety and effectiveness of high-dose chemotherapy followed by an infusion of immune cells from a family member to manage sickle cell disease. It aims to see if this approach offers long-term control over the condition.
What are the potential side effects?
Potential side effects may include reactions to the transplant such as graft-versus-host disease (where donated cells attack your body), infection risks due to weakened immunity from chemotherapy, and typical chemo-related issues like nausea or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have had a stroke or brain injury related to my sickle cell disease.
Select...
I have had at least 3 painful episodes in the last 2 years.
Select...
My donor is a family member who does not have sickle cell disease.
Select...
I can do most activities but may need help.
Select...
I need regular blood transfusions due to an abnormal TCD test.
Select...
I have sickle cell disease or Hemoglobin S Beta thalassemia.
Select...
I have had at least two episodes of severe chest pain.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have severe liver scarring.
Select...
I am not pregnant or breastfeeding.
Select...
I have a family member who matches my HLA type and is willing to donate.
Select...
I have had a stem cell transplant before.
Select...
I have sickle cell disease with an untreated infection.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~4 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Treatment related events
Secondary study objectives
Health-related quality of life
Pulmonary/pulmonary vascular status
neurological/neurocognitive status

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: Haplo Stem Cell TransplantationExperimental Treatment1 Intervention
CD34 selected T-cell depleted allogeneic SCT

Find a Location

Who is running the clinical trial?

Miltenyi Biomedicine GmbHIndustry Sponsor
37 Previous Clinical Trials
1,665 Total Patients Enrolled
Ann & Robert H Lurie Children's Hospital of ChicagoOTHER
270 Previous Clinical Trials
5,182,079 Total Patients Enrolled
Washington University School of MedicineOTHER
1,997 Previous Clinical Trials
2,298,740 Total Patients Enrolled
New York Medical CollegeLead Sponsor
71 Previous Clinical Trials
6,133 Total Patients Enrolled
UCSF Benioff Children's Hospital OaklandOTHER
79 Previous Clinical Trials
19,030 Total Patients Enrolled
Medical College of WisconsinOTHER
633 Previous Clinical Trials
1,181,674 Total Patients Enrolled
Tufts Medical CenterOTHER
263 Previous Clinical Trials
264,317 Total Patients Enrolled
University of California, Los AngelesOTHER
1,567 Previous Clinical Trials
10,266,979 Total Patients Enrolled
University of California, San FranciscoOTHER
2,591 Previous Clinical Trials
14,901,582 Total Patients Enrolled
Mitchell S Cairo, MDPrincipal InvestigatorNew York Medical College
14 Previous Clinical Trials
1,147 Total Patients Enrolled

Media Library

CD34 selected T-cell depleted allogeneic SCT (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT01461837 — Phase 2
Sickle Cell Disease Research Study Groups: Haplo Stem Cell Transplantation
Sickle Cell Disease Clinical Trial 2023: CD34 selected T-cell depleted allogeneic SCT Highlights & Side Effects. Trial Name: NCT01461837 — Phase 2
CD34 selected T-cell depleted allogeneic SCT (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01461837 — Phase 2
~2 spots leftby Dec 2025
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