Stem cell transplant for inherited immune system disorders

Plain-English translation of NCT07284641 on ClinicalTrials.gov ↗ · Source last updated May 2026 · 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 whether a stem cell transplant can help people with inherited immune system disorders—particularly Common Variable Immunodeficiency (CVID) and related conditions—whose bodies cannot fight infections or control inflammation properly. The transplant uses healthy stem cells from a donor (a family member or matched stranger) along with medications to prepare your body to accept them. This treatment aims to replace your faulty immune system with a working one.

Many people with these inherited immune disorders do not improve enough with current medications like mycophenolate mofetil or rituximab, leaving them vulnerable to serious infections and autoimmune problems. Researchers believe a stem cell transplant could offer a more permanent solution by rebuilding the immune system from scratch.

Do you qualify?

You likely qualify if…

you are between 5 and 40 years old
you have been diagnosed with an inherited immune disorder (such as CVID, DiGeorge syndrome, chronic granulomatous disease, or related conditions) confirmed by genetic testing
you have already tried and failed standard treatments like mycophenolate mofetil or rituximab due to lack of improvement or side effects
your kidneys, heart, and lungs are working well enough to handle the transplant procedure
you are free of HIV and HTLV infections
you have a suitable stem cell donor (a family member or matched unrelated donor)

You likely don't qualify if…

you have an allergy to DMSO (a chemical used to preserve stem cells)
you are pregnant or unable to use reliable birth control for 12 months after the transplant
you have had a solid organ transplant within the last 8 weeks
your heart, lungs, or kidneys are not strong enough (based on specific medical tests)

What participation looks like

You will undergo a stem cell transplant procedure at a single hospital center. First, you'll receive conditioning therapy—a combination of chemotherapy drugs and whole-body radiation designed to prepare your body to accept the new stem cells. Then you will receive healthy stem cells from your donor (obtained from bone marrow or circulating blood). After transplantation, you will be closely monitored for 2 years with regular visits to watch for success and manage any side effects, following standard medical care practices.

AI-generated summary from trial data · Jun 7, 2026 · Not medical advice

Trial locations(1 site)

United States

UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania