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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).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 years

Awards & highlights

HaploSCD Trial 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.Check my eligibility

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.See study design

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.

HaploSCD Trial 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.

HaploSCD Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 years

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Treatment related events

Secondary outcome measures

Health-related quality of life

Pulmonary/pulmonary vascular status

neurological/neurocognitive status

HaploSCD Trial Design

1Treatment groups

Experimental Treatment

Group I: Haplo Stem Cell TransplantationExperimental Treatment1 Intervention

CD34 selected T-cell depleted allogeneic SCT

0 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Miltenyi Biomedicine GmbHIndustry Sponsor

33 Previous Clinical Trials

1,602 Total Patients Enrolled

Ann & Robert H Lurie Children's Hospital of ChicagoOTHER

256 Previous Clinical Trials

5,187,859 Total Patients Enrolled

Washington University School of MedicineOTHER

1,931 Previous Clinical Trials

2,299,688 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

In how many different areas is this study offered?

"This study has six active sites, which are New york Medical College in Valhalla, Children's Hospital and Research Center Oakland in Oakland, and Medical College of Wisconsin/Children's Hospital of Wisconsin in Milwaukee. Other locations include _____, _____, and _____."

Answered by AI

Does this clinical trial have an age limit? If so, what is the maximum age of participants?

"According to the age requirements specified in this study's inclusion criteria, would-be participants must be between 2 and 20 years old."

Answered by AI

Could I possibly qualify to participate in this test?

"Eligible participants for this study must be between 2 and 20 years old, have a diagnosis of sickle cell anemia, and demonstrate one or more of the following Sickle Cell Disease Complications: Recurrent painful events (at least 3 in the 2 years prior to enrollment), minimum of two episodes of acute chest syndrome, Karnofsky or Lansky (age appropriate) Performance Score ≥50%."

Answered by AI

Are there any opportunities for people to participate in this research?

"The clinical trial in question is no longer actively recruiting patients. Originally posted on 2012-01-01, the study was last updated on 27 September 2022. There are currently 202 other studies that may be of interest to you."

Answered by AI

What is the mortality rate for CD34 selected T-cell depleted allogeneic SCT?

"We have given CD34 selected T-cell depleted allogeneic SCT a score of 2. This is based on it being a phase 2 trial, where while there is some evidence suggesting its safety, there is currently no data affirming its efficacy."

Answered by AI

Recent research and studies

Bone Marrow TransplantationJournal

Significant Improvement of Child Physical and Emotional Functioning After Familial Haploidentical Stem Cell Transplant

Parsons, Susan K., Angie Mae Rodday, Ruth Ann Weidner, Erin Morris, Suzanne Braniecki, Shalini Shenoy, Julie-An Talano, et al.. 2022. “Significant Improvement of Child Physical and Emotional Functioning After Familial Haploidentical Stem Cell Transplant”. Bone Marrow Transplantation. Springer Science and Business Media LLC. doi:10.1038/s41409-022-01584-y.

Bone Marrow TransplantationJournal

Significant Improvement of Child Physical and Emotional Functioning After Familial Haploidentical Stem Cell Transplant

clinicaltrials.govStudy

Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

Mitchell Cairo 2012. "Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01461837.

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