Haploidentical Bone Marrow Transplant for Sickle Cell Disease

Palo Alto (17 mi)

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Medical College of Wisconsin

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

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.

Inclusion Criteria

I am not using umbilical cord or peripheral blood stem cells for my treatment.

My liver tests are within the required range and I may need a liver MRI due to my transfusion history.

I have a family member who matches my HLA type and is willing to donate bone marrow.

My kidney function is within the normal range for my age.

I can care for myself but may need occasional help.

My heart's pumping ability is within the required range.

My oxygen levels are good and my lungs work well enough to breathe properly.

I am between 5 and 14 years old.

I can do most activities, my heart and lungs work well enough for treatment.

Exclusion Criteria

I had a surgery to improve brain blood flow within the last 6 months.

I am actively being treated for an autoimmune disease that is not under control.

I am not pregnant or breastfeeding.

I have had a bone marrow or stem cell transplant.

I have a sibling who matches my HLA type and can donate bone marrow.

My body has antibodies against a donor organ.

I agree to use two forms of birth control or practice true abstinence.

I am a male and agree to use effective contraception or practice abstinence.

Treatment Details

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.

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.

Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸 Approved in United States as Cytoxan for: