Haploidentical Bone Marrow Transplant for Sickle Cell Disease

Recruiting in Palo Alto (17 mi)

+32 other locations

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.

Research Team

Mary Horowitz, MD

Principal Investigator

Center for International Blood and Marrow Transplant Research

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

My direct bilirubin levels are within twice the normal limit, not due to blood transfusion issues.

I need a liver MRI because I've had many blood transfusions.

Your liver enzymes ALT and AST must be within the normal range.

Exclusion Criteria

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

You have participated in another research study that involved testing a new or unapproved drug or medical device within the past three months.

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

Treatment Details

Interventions

• Cyclophosphamide (Chemotherapy)
• Fludarabine (Chemotherapy)
• Haploidentical Bone Marrow Transplantation (Bone Marrow Transplantation)
• Hydroxyurea (Chemotherapy)
• Mesna (Chemotherapy)
• Thiotepa (Chemotherapy)
• Total Body Irradiation (Radiation Therapy)

Trial Overview 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.

Participant Groups

Group I: Haploidentical Transplantation Experimental Treatment 8 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:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma
• Rheumatoid arthritis

🇪🇺 Approved in European Union as Endoxan for:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma
• Rheumatoid arthritis

🇨🇦 Approved in Canada as Neosar for:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma
• Rheumatoid arthritis

🇯🇵 Approved in Japan as Endoxan for:

• Breast cancer
• Ovarian cancer
• Multiple myeloma
• Leukemia
• Lymphoma

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Levine Children's Hospital Charlotte, NC

Duke University Medical Center Durham, NC

University Hospitals of Cleveland/Case Western Cleveland, OH

Nationwide Children's Hospital Columbus, OH

More Trial Locations

Who Is Running the Clinical Trial?

Medical College of Wisconsin

Lead Sponsor

Trials

645

Patients Recruited

1,180,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3987

Patients Recruited

47,860,000+

National Cancer Institute (NCI)

Collaborator

Trials

14080

Patients Recruited

41,180,000+

Blood and Marrow Transplant Clinical Trials Network

Collaborator

Trials

51

Patients Recruited

14,600+

National Marrow Donor Program

Collaborator

Trials

63

Patients Recruited

202,000+