Bone Marrow Transplant for Non-Malignant Disorders

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

Recruiting Phase 1 Phase 2 Interventional Study
Metabolic Disorders Hematologic, Immune, or Bone Marrow Disorders Hemoglobinopathies Non-malignant Disorders
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
Up to 20
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
220 (estimated)
Sponsor
Washington University School of Medicine · Other
Who this trial is looking for

This trial is looking for young patients with certain non-cancer disorders who need a bone marrow transplant. Participants will receive a special conditioning treatment to prepare them for the transplant.

Are You a Good Fit for This Trial?

You may be able to join if

  • I am under 21 years old
  • I have a non-malignant disorder, excluding thalassemia
  • I am receiving an 8/8 HLA-matched bone marrow transplant
  • I have a hemoglobinopathy and am receiving a 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
  • I have adequate function of my lungs, kidneys, liver, and other organs
  • I have a Lansky/Karnofsky score of 40 or more
  • I have a negative pregnancy test
  • If I have sickle cell disease, my Hemoglobin S is less than 30%

You may not be able to join if

  • I am HIV positive
  • I have an invasive infection
  • I am pregnant or breastfeeding

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

Think this trial could be right for you?

Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.

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About This Trial

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.

Trial Locations
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Eligibility 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. Strat…
Contacts

Lissy Keller, CCRP

3142861168

kellerl@wustl.edu

CONTACT

Ian Snyder, CCRP

3142735953

ian.s@wustl.edu

CONTACT