FindMyKOL
Clinical trialNCT01962415RecruitingPhase 2Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

SponsorPaul SzabolcsEnrollment100Congenital Bone Marrow Failure SyndromesHereditary AnemiasInflammatory ConditionsInherited Metabolic Disorders (IMD)Juvenile Rheumatoid Arthritis (JRA)Primary Immunodeficiency (PID)+1 more conditions
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
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

    Archana Ramgopal, DOPaulina Horvei, 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.

Alemtuzumab →Fludarabine →Hydroxyurea →Melphalan →Thiotepa
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
4 Feb 2014
Primary completion
30 Nov 2026
Completion
30 Nov 2027
Last update posted
15 Dec 2025
Registered enrollment
100
Registry id
NCT01962415
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.