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

  • End date
    Dec 18, 2031
  • participants needed
  • sponsor
    Washington University School of Medicine
Updated on 24 January 2022
graft versus host disease
mycophenolate mofetil
hemoglobin s
transplant conditioning
preparative regimen
graft-versus-host disease


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 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.

Condition Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders
Treatment Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan, Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan, Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients, GVHD Regimen B: BM Recipients
Clinical Study IdentifierNCT00920972
SponsorWashington University School of Medicine
Last Modified on24 January 2022


Yes No Not Sure

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
Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or
-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
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact



Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider


Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 



Reply by • Private

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note