A study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease using a Reduced-Intensity preparatory regime with Campath-1H Fludarabine and Melphalan

Investigating Hematopoietic Stem Cell Transplantation in Non-Malignant Diseases

J
Jodi Skiles

Primary Investigator

Enrolling By Invitation
20 years or below
All
Phase N/A
1 Location

Brief description of study

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 of study

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

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Metabolic Disorders, Non-malignant Disorders, Hematopoietic Stem Cell Transplantation (HSCT), Riley
  • Age: 20 years or below
  • Gender: All

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

This study investigates the use of Hematopoietic Stem Cell Transplantation (HSCT) in children with non-cancerous diseases. The purpose is to use a preparatory regimen that suppresses the immune system without destroying bone marrow to allow donor cells to engraft. The study aims to understand the main side effects of this treatment within the first 100 days after the transplant.

Participants will undergo a reduced intensity conditioning that avoids radiation and high-dose chemotherapy. This approach aims to lower the risk of transplant-related complications, including graft versus host disease (GVHD). The study employs specific GVHD prevention strategies for those receiving stem cells from bone marrow or cord blood.

  • Who can participate: Children under 21 with non-malignant disorders, excluding thalassemia in Stratum 1, may participate. They must have adequate organ function and not be pregnant or HIV positive.
  • Study details: Participants will receive reduced intensity conditioning to prepare for the transplant. The study includes specific treatments to prevent GVHD, a condition where donated cells attack the recipient's body.
Updated on 09 Dec 2025. Study ID: PHO-WASHU-DELG-1010002807, 1010002807
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only