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Chemotherapy

Stem Cell Transplant for Sickle Cell Disease

Phase 1 & 2

Waitlist Available

Led By Courtney D Fitzhugh, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

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

Must have

Glomerular filtration rate >60 mL/min/1.73m^2 by cystatin C-based or iothalamate-based or other equivalent GFR testing

Ejection fraction greater than or equal to 35%

Must not have

Available 6/6 HLA-matched sibling donor

ECOG performance status of 3 or more

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to year 5

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new transplant regime to see if it is effective, safe and well tolerated in people with sickle cell disease.

Who is the study for?

Adults over 18 with sickle cell disease and severe complications like stroke, high blood flow in the lungs, liver issues, serious lung problems or silent strokes on MRI. They need a half-matched relative donor, good heart and kidney function, and can't be pregnant or breastfeeding. People with uncontrolled infections or major illnesses that could interfere with stem cell transplant recovery are excluded.

What is being tested?

The trial is testing a new bone marrow transplant method using donors who are half tissue matches for adults with severe sickle cell disease. It includes taking stem cells from the donor's blood after drug stimulation, preparing the recipient's body with radiation and immune system drugs before infusing these cells through a central vein line.

What are the potential side effects?

Possible side effects include reactions to medications like cyclophosphamide and sirolimus (such as nausea or low blood counts), risks from low-dose radiation exposure, complications from having a central line (like infection), and graft-versus-host disease where donated cells attack the recipient's body.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function test shows a filtration rate above 60 mL/min.

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My heart pumps blood effectively.

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I am 18 years old or older.

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I have sickle cell disease and am at high risk for stroke or early death.

Exclusion Criteria

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

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I have a sibling donor who is a perfect match for me.

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I need help with my daily activities due to my health condition.

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I do not have any major illnesses that would make a stem cell transplant too risky.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to year 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to year 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to year 5 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Percentage of Participants Who Have Not Rejected Their Stem Cell Graft and Who Are Without Severe Graft-versus-host Disease Following Stem Cell Transplant

Secondary study objectives

Chimeric Value That is Required to Maintain Graft Survival and Hematologic Normalcy.

Incidence of Donor Type Hemoglobin Post-transplant in SCD Patients Who Have Not Been Transfused in the Previous 3 Months.

Incidence of Graft Failure Following Stem Cell Transplant

+7 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Participants with Sickle Cell Disease with Nonmyeloablative Haplo TransplantsExperimental Treatment6 Interventions

Participants will receive pentostatin on days -21, -17, -13, and -9 and oral cyclophosphamide from days -21 to -8. Alemtuzumab to be infused on days -7 to -3, followed by 400 cGy TBI on day -1. Donor-derived peripheral blood stem cells will be given on Day 0 then cyclophosphamide will be given at 50 mg/kg on day +3. Sirolimus loading dose of 5mg PO q4h x three doses at one day after the completion of cyclophosphamide (on day +4) and continued the following day at 5mg PO q24h to maintain trough levels between 5-15 ng/ml.

Group II: Human Leukocyte Antigens (HLA) Haploidentical Related Stem Cell DonorExperimental Treatment1 Intervention

A haploidentical relative donor will receive filgrastim (G-CSF) 10 to 16 µg/kg/d subcutaneously or intravenously for up to 6 days with apheresis collections of peripheral blood hematopoietic progenitor cells (PBPC) after the 5th day (and after the 6th day if required).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Filgrastim

2000

Completed Phase 3

~3690

Cyclophosphamide

2010

Completed Phase 4

~2310