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Clinical trialNCT04528355RecruitingObservational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

SponsorPaul SzabolcsEnrollment50Congenital Bone Marrow Failure SyndromesHereditary AnemiasInflammatory ConditionsInherited Metabolic Disorders (IMD)Primary Immunodeficiency (PID)
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.
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
  • Upmc Children'S Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, United States

    Craig Byersdorfer, MDElizabeth Stenger, MDJessie Barnum, MDMaria Escolar, MDRandy Windreich, 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.

Data collection
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
20 Aug 2020
Primary completion
31 Dec 2027
Completion
30 Jun 2028
Last update posted
13 Jan 2026
Registered enrollment
50
Registry id
NCT04528355
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.