Home › Metabolic Disorders › NCT00920972

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

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

Who can join

Up to age 20 · All sexes

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

About the 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.

What is being tested

• 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 (drug)

Sponsor: Washington University School of Medicine · Participants: 220 · Started: 2001-12

Contact the study team

• Lissy Keller, CCRP · Phone: 3142861168
• Ian Snyder, CCRP · Phone: 3142735953

Official record on ClinicalTrials.gov — NCT00920972

Locations in the U.S.

Conditions

• Metabolic Disorders
• Hemoglobinopathies

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.