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

ConditionsPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)Hereditary AnemiasInflammatory Conditions
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-60
SponsorPaul Szabolcs

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patient, parent, or legal guardian must have given written informed consent.

Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.

Severe Combined Immune Deficiency (SCID) with NK cell activity

Omenn Syndrome

Disqualifiers

None

Trial population

patients with Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • 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.

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

incidence of acute graft versus host disease (GVHD)

grades 3-4, chronic extensive GVHD

Time frame
up to 5 years
2

overall survival after HSCT

review of the existing medical records to check on the participant's survival status

Time frame
up to 5 years

Secondary outcomes

1

Describe degree of engraftment, based upon chimerism data

review of chimerism test results in the existing medical records to check on degree of donor engraftment measured by the percentage of donor-derived blood cells in the HSCT recipient

Time frame
up to 5 years
2

Describe probability to discontinue systemic immunosuppression medications

review of the existing medical records to check on the participant's current medications

Time frame
by 6, 9, and 12 months post-HSCT
3

Describe the tempo of immune reconstitution

review of the various test results in existing medical records to check on the participant's immune system recovery rate

Time frame
over the first year post transplant
4

Describe the use of donor leukocyte infusion (DLI)

review of the existing medical records to check on the participant's need for DLI

Time frame
up to 5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Paul Szabolcs

Lead sponsor

University of Pittsburgh

Sponsor institution

Source ClinicalTrials.gov
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