Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

• Status: Recruiting
• Trial end date: 2031-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Washington University School of Medicine
• Collaborator: St. Louis Children's Hospital
• Treatments: Alemtuzumab; Fludarabine; Fludarabine phosphate; Hydroxyurea; Melphalan; Thiotepa; Vidarabine

Criteria

Inclusion Criteria:

Stratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8/8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

Stratum 3: Patient must have a hemoglobinopathy receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

All strata:

• Recipient age < 21 years

• Lansky/Karnofsky >/= 40

• Adequate pulmonary, renal, liver, and other organ function as defined in protocol

• Negative pregnancy test

• Adequate total nucleated cell or CD34+ dose of product as defined in protocol

• If sickle cell, Hemoglobin S <30%

Exclusion Criteria:

• HIV positive

• Invasive infection

• Pregnancy/lactating