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Clinical trialNCT04232085RecruitingPhase 2Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

SponsorSidney Kimmel Comprehensive Cancer Center at Johns HopkinsEnrollment27Bone Marrow FailureDyskeratosis CongenitaFanconi AnemiaHoyeraal-Hreidarsson SyndromeImmune Deficiency DiseaseNon Fanconi DNA-DSB Repair Disorder+4 more conditions
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
clinicaltrials.gov ↗

Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States
  • Johns Hopkins University

    Baltimore, Maryland, United States

    Heather J Symons, MD
    Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Alemtuzumab →Cyclophosphamide →Fludarabine →Melphalan →Mycophenolate mofetil →Tacrolimus →
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
12 Feb 2020
Primary completion
31 Dec 2027
Completion
31 Dec 2028
Last update posted
28 Nov 2025
Registered enrollment
27
Registry id
NCT04232085
clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.