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Chemotherapy

Haploidentical Bone Marrow Transplant for Sickle Cell Disease

Phase 2
Waitlist Available
Research Sponsored by Medical College of Wisconsin
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Umbilical cord blood or peripheral blood stem cell donors will not be accepted.
Hepatic function: Serum conjugated (direct) bilirubin < 2x upper limit of normal for age, ALT and AST < 5x upper limit of normal, and liver MRI for participants with specific transfusion history
Must not have
Had an Encephaloduroarteriosynangiosis (EDAS) procedure in the 6 months prior to enrollment
Uncontrolled autoimmune disease requiring active medical management
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 and 2 years
Awards & highlights

Summary

This trial tests a special type of bone marrow transplant for children and adults with severe sickle cell disease. It uses medications to prepare the body and a donor to replace defective bone marrow. The goal is to improve survival without major complications. Bone marrow transplantation is currently the only curative therapy for sickle cell disease, but it is limited by side effects and finding suitable donors.

Who is the study for?
This trial is for children (5-14.99 years) and adults (15-45.99 years) with severe sickle cell disease who have a family member willing to donate bone marrow that partially matches their HLA type. Participants must be in good physical condition, not pregnant or breastfeeding, and agree to use contraception if of childbearing potential.
What is being tested?
The study tests the effectiveness and safety of haploidentical bone marrow transplantation in treating severe sickle cell disease. It includes medications like Hydroxyurea, Rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide plus Total Body Irradiation and Mesna as part of the treatment process.
What are the potential side effects?
Possible side effects include immune reactions from the transplant leading to graft-versus-host disease, infections due to weakened immunity post-transplantation, organ damage from chemotherapy drugs or radiation therapy used before transplantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am not using umbilical cord or peripheral blood stem cells for my treatment.
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My liver tests are within the required range and I may need a liver MRI due to my transfusion history.
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I have a family member who matches my HLA type and is willing to donate bone marrow.
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My kidney function is within the normal range for my age.
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I can care for myself but may need occasional help.
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My heart's pumping ability is within the required range.
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My oxygen levels are good and my lungs work well enough to breathe properly.
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I am between 5 and 14 years old.
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I can do most activities, my heart and lungs work well enough for treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I had a surgery to improve brain blood flow within the last 6 months.
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I am actively being treated for an autoimmune disease that is not under control.
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I am not pregnant or breastfeeding.
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I have had a bone marrow or stem cell transplant.
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I have a sibling who matches my HLA type and can donate bone marrow.
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My body has antibodies against a donor organ.
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I agree to use two forms of birth control or practice true abstinence.
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I am a male and agree to use effective contraception or practice abstinence.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 and 2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Two-Year Post-Transplant Event Free Survival (EFS)
Secondary study objectives
Chimerism
Disease Recurrence
Graft Rejection
+3 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Haploidentical TransplantationExperimental Treatment8 Interventions
A conditioning regimen with Hydroxyurea, rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, and Mesna will be administered prior to Haploidentical Bone Marrow Transplantation.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Haploidentical Bone Marrow Transplantation
2008
Completed Phase 2
~60
Hydroxyurea
2006
Completed Phase 4
~3620
Thiotepa
2008
Completed Phase 3
~2120
Fludarabine
2012
Completed Phase 4
~1860
Cyclophosphamide
2010
Completed Phase 4
~2320
Total Body Irradiation
2006
Completed Phase 3
~820
Mesna
2003
Completed Phase 2
~1380

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and hematopoietic stem cell transplantation (HSCT). Hydroxyurea works by increasing fetal hemoglobin (HbF) levels, which reduces the sickling of red blood cells and decreases vaso-occlusive episodes. Red blood cell transfusions help by increasing the number of normal red blood cells, thereby reducing the proportion of sickled cells and preventing complications such as stroke. HSCT, including haploidentical bone marrow transplantation, involves replacing defective bone marrow with healthy donor marrow, potentially curing SCD by enabling the production of normal red blood cells. These treatments are crucial for SCD patients as they aim to alleviate pain, prevent severe complications, and improve overall quality of life and survival.
Hematopoietic stem cell transplantation for children with sickle cell disease.

Find a Location

Who is running the clinical trial?

Medical College of WisconsinLead Sponsor
623 Previous Clinical Trials
1,178,292 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,905 Previous Clinical Trials
47,837,577 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,868 Previous Clinical Trials
41,010,757 Total Patients Enrolled

Media Library

Cyclophosphamide (Chemotherapy) Clinical Trial Eligibility Overview. Trial Name: NCT03263559 — Phase 2
Sickle Cell Disease Research Study Groups: Haploidentical Transplantation
Sickle Cell Disease Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT03263559 — Phase 2
Cyclophosphamide (Chemotherapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03263559 — Phase 2
~10 spots leftby Oct 2025