Lower-dose stem cell transplant for children and young adults with inherited blood disorders

Plain-English translation of NCT01962415 on ClinicalTrials.gov ↗ · Source last updated December 2025 · Translation generated June 2026 · How we translate trials

Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.

What is this trial?

This trial is testing a gentler approach to stem cell transplantation for children and young adults (up to age 55) who have inherited blood disorders, bone marrow failure, metabolic disorders, or immune system conditions. Instead of using standard high-dose chemotherapy before the transplant, researchers are using reduced-intensity conditioning — significantly lower doses of chemotherapy — to prepare the body for healthy donor stem cells. The goal is to achieve successful transplant outcomes while reducing severe side effects on the brain, heart, lungs, liver, and other organs.

Many young patients with these inherited disorders have never received chemotherapy before, which can make stem cell transplantation challenging. Researchers believe that lower chemotherapy doses combined with the right immunosuppressive medications may allow donor stem cells to successfully take hold while causing fewer harmful side effects than traditional high-dose approaches.

Do you qualify?

You likely qualify if…

you are between ages 4 and 55
you have been diagnosed with a non-malignant inherited disorder such as a primary immunodeficiency, bone marrow failure syndrome, inherited metabolic disorder, hereditary anemia, or inflammatory condition
you have a matched stem cell donor available (either from cord blood, bone marrow, or peripheral blood)
your heart, lungs, liver, and kidneys are functioning at acceptable levels
you are HIV negative and not pregnant (if applicable)

You likely don't qualify if…

you have a malignant (cancer-related) disorder
you have severe organ damage that would make chemotherapy unsafe
your kidneys are not filtering adequately or your liver enzymes are very elevated
you are pregnant or breastfeeding
you do not have a matched donor available

What participation looks like

If you are enrolled, you will receive a conditioning regimen consisting of lower doses of chemotherapy medications (including , , , and ) to prepare your body for the transplant. You will then receive healthy stem cells from your matched donor. You will be monitored closely during your hospital stay and in follow-up visits to track how well the new stem cells take hold and to manage any side effects. The exact timeline and number of visits will depend on your specific situation and how you respond to treatment.

AI-generated summary from trial data · Jun 2, 2026 · Not medical advice

Trial locations(1 site)

United States

UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania