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

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

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 design

Treatments tested in this trial

data collection

Treatment groups

No treatment groups listed

Sponsors and collaborators

Paul Szabolcs

Lead sponsor

University of Pittsburgh

Sponsor institution

Source ClinicalTrials.gov