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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
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I have had a stroke or brain injury related to my sickle cell disease.
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I have had at least 3 painful episodes in the last 2 years.
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My donor is a family member who does not have sickle cell disease.
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I can do most activities but may need help.
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I need regular blood transfusions due to an abnormal TCD test.
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I have sickle cell disease or Hemoglobin S Beta thalassemia.
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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:
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I have severe liver scarring.
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I am not pregnant or breastfeeding.
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I have a family member who matches my HLA type and is willing to donate.
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I have had a stem cell transplant before.
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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
36 Previous Clinical Trials
1,628 Total Patients Enrolled
Ann & Robert H Lurie Children's Hospital of ChicagoOTHER
264 Previous Clinical Trials
5,182,601 Total Patients Enrolled
Washington University School of MedicineOTHER
1,989 Previous Clinical Trials
2,295,797 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
~0 spots leftby Dec 2024
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