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Alkylating agents

Stem Cell Transplant for Sickle Cell Disease

Phase 2

Recruiting

Led By Damiano Rondelli, MD

Research Sponsored by University of Illinois at Chicago

Eligibility Criteria Checklist

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

Must have

Patients with sickle cell disease who have the following complications: stroke or central nervous system event lasting longer than 24 hours, frequent vaso-occlusive pain episodes, recurrent episodes of priapism, acute chest syndrome with recurrent hospitalizations, red-cell alloimmunization, bilateral proliferative retinopathy with major visual impairment, osteonecrosis of 2 or more joints, sickle cell nephropathy, pulmonary hypertension

Karnofsky performance status of 60 or higher

Must not have

Donor relatives who are not HLA-haploidentical match (≥ 4/8)

Fully HLA-matched sibling donor willing to donate stem cells

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to day +60

Awards & highlights

Approved for 20 Other Conditions

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is looking at a new way to do a stem cell transplant for sickle cell disease. The old way is not working as well as they'd like, so they are trying something new. This study is to see if the new way is better.

Who is the study for?

This trial is for people aged 16-60 with severe sickle cell disease, who have complications like stroke, recurrent pain episodes, or organ damage. They must not be pregnant, HIV-negative, and without a fully HLA-matched sibling donor but have an HLA-haploidentical relative willing to donate stem cells.

What is being tested?

The study tests a new way of transplanting stem cells from half-matched relatives in patients with aggressive sickle cell disease. It involves low-dose body radiation and drugs like fludarabine and cyclophosphamide before the transplant, followed by post-transplant medication to help the body accept the new cells.

What are the potential side effects?

Possible side effects include reactions to medications such as mycophenolate mofetil and sirolimus (like nausea or diarrhea), risks from total body irradiation (like fatigue or skin changes), and complications related to stem cell infusion (such as infection risk).

Eligibility Criteria

Inclusion Criteria

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

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I have sickle cell disease with serious complications like stroke, frequent pain, or vision loss.

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I can care for myself but cannot do normal activities or work.

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My heart pumps well enough (ejection fraction is 40% or more).

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My lungs work well enough for treatment (DLCO ≥ 50% predicted).

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I have a family member who is a partial genetic match for me.

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I am between 16 and 60 years old.

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I do not have a sibling who is a complete HLA match for me.

Exclusion Criteria

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

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My donor relative is not a close enough genetic match for the transplant.

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I have a sibling who is a complete HLA match and willing to donate stem cells.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to day +60

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to day +60

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to day +60 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Estimate the number of patients who engraft by Day +60

Secondary study objectives

Adverse Effects

Disease free survival

Overall survival

Awards & Highlights

Approved for 20 Other Conditions

This treatment demonstrated efficacy for 20 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

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: Subject treatmentExperimental Treatment7 Interventions

Patients will receive the following conditioning regimen: ATG, fludarabine (6 days before stem cell infusion), cyclophosphamide, and total body irradiation. The stem cell product will be infused according to BMT unit policy. Patients will also receive GVHD prophylaxis which will consist of cyclophosphamide, sirolimus, and mycophenolate mofetil according to the protocol. Post-transplant evaluation will be done as per standard care with study data collected at days 30, 60, 100, 180, 365, and annually thereafter.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Sirolimus

FDA approved

Total body irradiation

2008

Completed Phase 3

~1080

Stem cell infusion

2002