← Back to Search

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)

Must not have

Clinically significant liver fibrosis or cirrhosis if on chronic transfusion therapy > 6 months

Women of childbearing potential: pregnant or breastfeeding

Timeline

Screening 3 weeks

Treatment Varies

Follow Up +2 years post-transplant

Awards & highlights

No Placebo-Only Group

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.

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.

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

Select...

I have had a stroke or neurological issues lasting more than a day.

Select...

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

Select...

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

Select...

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

Select...

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

Select...

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

Select...

My organs are functioning well enough for the trial.

Select...

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

Select...

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

Select...

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

Select...

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I have serious liver scarring and have been on blood transfusion therapy for over 6 months.

Select...

I am not pregnant or breastfeeding.

Select...

I have had a bone marrow transplant in the past.

Select...

I will be taking medication that strongly affects liver enzymes.

Select...

I am not currently receiving any experimental treatments or standard cancer therapies.

Select...

I have an active cancer other than non-melanoma skin cancer.

Select...

I have had a stem cell transplant before.

Select...

I currently have an infection.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Persistent immunosuppressant -dependent mixed chimerism

Persistent post-immunosuppressant mixed chimerism

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: 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

Tacrolimus

2019

Completed Phase 4

~5510

Mycophenolate mofetil

2014

Completed Phase 4

~3060