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Hematopoietic Stem Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

Phase 1

Waitlist Available

Led By Joseph Rosenthal, MD

Research Sponsored by City of Hope Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; or increased transcranial Doppler velocity (>200 m/s). A stroke is defined as a sudden neurologic change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes.

Prior treatment with regular RBC transfusion therapy, defined as receiving ≥ 8 transfusions per year for > 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up +2 years post-transplant

Awards & highlights

Study Summary

This trial is testing a new way to do a half-matched blood stem cell transplant to treat severe sickle cell disease in adults.

Who is the study for?

This trial is for patients with severe sickle cell disease who have had multiple joint issues, strokes, or vaso-occlusive crises despite treatment. They need a related half-matched donor (like a parent or sibling) and can't have had prior transplants. Women must not be pregnant and agree to use contraception.Check my eligibility

What is being tested?

The study tests a blood stem cell transplant from a half-matched relative using new chemotherapy that's less harsh on bone marrow. It aims to create mixed chimerism where the patient's body accepts both their own and the donor's blood cells, potentially reversing sickle cell disease.See study design

What are the potential side effects?

Potential side effects include reactions to immune-suppressing drugs like Tacrolimus and anti-thymocyte globulin, which may cause infections, liver problems, or allergic reactions. The chemotherapy agents used could also lead to nausea, hair loss, mouth sores, or low blood counts.

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 neurological issues lasting more than a day.

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I've had regular blood transfusions (8 or more a year) for over a year to prevent complications like pain, stroke, or chest issues.

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I have been diagnosed with sickle cell disease (SS or S-βº Thalassemia).

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I have had at least one episode of severe chest pain in the last 2 years despite treatment.

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I don't have a fully matched donor for a transplant.

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I've had 2 or more severe pain crises a year needing hospital care despite treatment.

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My organs are functioning well enough for the trial.

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I have tried hydroxyurea for my condition and it didn't work or caused side effects.

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I agree to use effective birth control during and for 6 months after the study.

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I have bone damage in two or more joints despite receiving care.

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My donor is a half-match for me based on specific genetic markers.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ +2 years post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~+2 years post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and +2 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

Feasibility of producing an infusion ready CD4+ T-cell-depleted hematopoietic product

Mixed Chimerism defined as 30-90% donor cells

Toxicity per NCI-Common Terminology Criteria for Adverse Events version 4.0

+1 more

Secondary outcome measures

Acute Graft versus Host Disease per 1994 Keystone Consensus Criteria

Adverse events of Grade 3 or higher

Graft-vs-Host Disease

+17 more

Other outcome measures

Ratio donor: recipient FoxP3+ regulatory T cells

Ratio donor: recipient de novo thymic T cells

Tolerance status of donor: recipient type T cells

Trial Design

1Treatment groups

Experimental Treatment

Group I: COH-MC-17 and immunosuppressantsExperimental Treatment6 Interventions

Participants receive COH-MC-17: a 21-day nonmyeloablative conditioning regimen (cyclophosphamide, pentostatin and rabbit anti-thymocyte globulin), followed by CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant on Day 0. Immunosuppressants (tacrolimus and mycophenolate mofetil) given on Day -1 onwards until discontinuation post-transplant. The minimally manipulated transplant product is manufactured using the CliniMACS device.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cyclophosphamide

1995

Completed Phase 3

~3770

Mycophenolate mofetil

2014

Completed Phase 4

~2350

Pentostatin

2000

Completed Phase 3

~1300

Rabbit anti-thymocyte globulin

2009

Completed Phase 2

~40

Tacrolimus

2011

Completed Phase 4

~4740

0 / 11Eligibility criteria met

Find a Location

Who is running the clinical trial?

City of Hope Medical CenterLead Sponsor

569 Previous Clinical Trials

1,924,685 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

65 Previous Clinical Trials

3,180 Total Patients Enrolled

Joseph Rosenthal, MDPrincipal InvestigatorCity of Hope Medical Center

4 Previous Clinical Trials

103 Total Patients Enrolled

Media Library

CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT03249831 — Phase 1

Sickle Cell Disease Research Study Groups: COH-MC-17 and immunosuppressants

Sickle Cell Disease Clinical Trial 2023: CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant Highlights & Side Effects. Trial Name: NCT03249831 — Phase 1

CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03249831 — Phase 1

Frequently Asked Questions

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

What is the current quota of participating individuals for this experiment?

"Affirmative. According to clinicaltrials.gov, this medical research program is still looking for volunteers. It was initially posted on April 1st 2019 and recently revised on July 6th 2022; the trial requires the recruitment of six patients from a single site."

Answered by AI

For what health conditions is CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant typically prescribed?

"Immunosuppressive treatment is normally treated with CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant, which has also been demonstrated to relieve a range of medical conditions such as dermatitis, atopic diseases, multiple sclerosis and acute leukemia myelocytic."

Answered by AI

Is this research currently seeking participants?

"Affirmative. The information archived on clinicaltrials.gov shows that this research project, initially posted in the early days of April 2019, is still recruiting participants from 1 site to complete their sample size of 6 patients."

Answered by AI

Does the protocol of this medical study include adults beyond 25 years old?

"In accordance with the parameters of this trial, the minimum age for enrolment is 18 years old and the maximum age cap is 45."

Answered by AI

How much peril do patients encounter with CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant?

"The safety of CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant is rated at a 1 since it's only in Phase 1. Limited information exists to support its efficacy and the proposed security profile."

Answered by AI

Is there a certain set of individuals eligible to participate in this clinical experiment?

"This clinical trial seeks to recruit 6 participants aged between 18 and 45 that possess hemoglobinopathies. Applicants should meet a range of criteria, such as having undergone hydroxyurea therapy without success or exhibiting intolerance to the treatment; displaying osteonecrosis in at least two joints despite supportive care measures being taken; enduring recurrent episodes of severe vaso-occlusive pain crises lasting more than 2 hours over the past two years with said pains requiring medical attention; previous experience receiving regular RBC transfusion therapy consisting of 8+ transfusions per year for more than one year so as to prevent vaso-occlusive complications like strokes and"

Answered by AI

Has there been previous research on the effects of CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant?

"As of now, 961 clinical trials related to CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant are taking place. Of those, 179 have already entered Phase 3. While the majority are based in Philadelphia, Pennsylvania, there is a global reach with nearly 30 thousand research sites currently running these investigations."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Blood Stem Cell Transplant for Sickle Cell Disease

2019. "A Blood Stem Cell Transplant for Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03249831.

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