1010002807 a Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime with Campath-1H Fludarabine and Melphalan

PH

Paul Haut, MD

Principal Investigator

Status: Enrolling By Invitation Ages: 20 Years Gender: All Genders Phase: N/A 2 Locations

Brief Description

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.  The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY - Only eligible patients that have been referred by a physician or self-referred to and seen by section of Pediatric Hematology/Oncology staff will be invited to participate in the clinical trial.

Detailed Description

The study uses reduced intensity conditioning that is immune suppressive to achieve donor cell engraftment without exposure to radiation or high dose chemotherapy in children with non-malignant disorders. The intent is to minimize early and late regimen related toxicities in the context of a reduced intensity regimen.

In addition to maximizing opportunity for donor cell engraftment, the trial seeks to minimize toxicities associated with transplant such as graft versus host disease and employs GVHD prophylaxis that seeks to decrease rates of acute and chronic GVHD in the setting of matched and mismatched donor stem cell transplants from marrow and cord blood sources.

  • Drug: Treatment Plan 1: Stratum 1
  • Drug: Treatment Plan 2: Strata 2, 3, or 4
  • Drug: GVHD Regimen A: UCB Recipients
  • Drug: GVHD Regimen B: BM Recipients

Eligibility of study

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

Interested in participating?

Enrollment in IU School of Medicine clinical trials is managed through our All IN for Health program. Visit allinforhealth.info to see if you're eligible and join the study.