New transplant approach aims to fix broken immune systems

NCT ID NCT04232085

Recruiting now Disease control Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 participants will receive the transplant and be monitored for donor cell engraftment and survival at one year.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: stem cell transplant with reduced-intensity chemotherapy and post-transplant cyclophosphamide
What this could lead to: If successful, this could offer a safer way to replace a faulty immune system with a donor's healthy one, reducing long-term complications for people with severe immune disorders.
What could go wrong: This is a small, early-phase trial (27 people) and the transplant still carries risks like graft-versus-host disease, infection, and organ damage. It may not work for all conditions tested.

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Conditions

The condition(s) this trial relates to.

Bone Marrow Failure Disorders bone marrow failure syndrome dyskeratosis congenita Fanconi anemia Hoyeraal-Hreidarsson syndrome Immune Deficiency Disease immunodeficiency disease inborn error of immunity Primary Immunodeficiency Diseases telomere syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BONE MARROW FAILURE DYSKERATOSIS CONGENITA FANCONI ANEMIA HOYERAAL HREIDARSSON SYNDROME IMMUNE DEFICIENCY DISEASE NON FANCONI DNA-DSB REPAIR DISORDER PRIMARY IMMUNE DEFICIENCY DISORDER SHORT TELOMERE LENGTH SHORT TELOMERE SYNDROME TELOMERE BIOLOGY DISORDER

New transplant approach aims to fix broken immune systems

Summary

What this could mean

Get notified about this study

Conditions

As listed by the trial registrant

Contacts and locations

Locations

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