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

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Paul Szabolcs
Study ID
NCT04528355
Status
Recruiting
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Conditions

  • Congenital Bone Marrow Failure Syndromes
  • Hereditary Anemias
  • Inflammatory Conditions
  • Inherited Metabolic Disorders (IMD)
  • Primary Immunodeficiency (PID)

Eligibility Criteria

Sex
ALL
Age
2 Months - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • data collection — DRUG
    Study subjects will receive alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen in accordance with clinical practice at UPMC Children's Hospital of Pittsburgh at the discretion of the treating physician. Medical data will be abstracted from subject's medical charts once the patient signs the informed consent.

Study Details

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.

Key Dates

Start date
Aug 20, 2020
Status verified
Jan 2026
Primary completion
Dec 31, 2027
Completion
Jun 30, 2028

Study Design

Enrollment
50 participants (estimated)

Primary Outcome Measure

incidence of acute graft versus host disease (GVHD) [ Time Frame: up to 5 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224
Shawna McIntyre, RN
[email protected]
412-692-5552
Jessie Barnum, MD (SUB_INVESTIGATOR)
Craig Byersdorfer, MD (SUB_INVESTIGATOR)
Maria Escolar, MD (SUB_INVESTIGATOR)
Elizabeth Stenger, MD (SUB_INVESTIGATOR)
Randy Windreich, MD (SUB_INVESTIGATOR)

Find similar trials in Pittsburgh, PA

By research site
UPMC Children's Hospital of Pittsburgh· Pittsburgh, PA

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