Transplant conditioning for non-malignant diseases using immune suppression

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates a preparative regimen that maximizes host immunosuppression without myeloablation to facilitate the engraftment of donor hematopoietic cells in children with non-malignant disorders. It employs reduced intensity conditioning to minimize early and late toxicities associated with transplant, such as graft versus host disease (GVHD). The trial evaluates the safety and efficacy of this approach by monitoring major toxicities within the first 100 days post-transplantation, utilizing matched and mismatched donor stem cells from both bone marrow and umbilical cord blood sources.

Why it matters

Who can join: the full rules

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

Where it's running

• Washington University School of Medicine (in St. Louis)
  St Louis, Missouri, United States Recruiting
• Phoenix Children's Hospital
  Phoenix, Arizona, United States Active not recruiting
• Children's Hospital of Orange County
  Orange, California, United States Active not recruiting
• University of California
  San Diego, California, United States Active not recruiting
• Yale School of Medicine
  New Haven, Connecticut, United States Active not recruiting
• George Washington University School of Medicine
  Washington D.C., District of Columbia, United States Active not recruiting

Study contacts

• Principal investigator: Shalini Shenoy, MD — Washington University School of Medicine (in St. Louis)
• Study coordinator: Lissy Keller, CCRP
• Email: kellerl@wustl.edu
• Phone: 3142861168

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.