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

(PRO-RIC Trial)

Overseen By Paul Szabolcs, MD

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Paul Szabolcs

Must be taking: Alemtuzumab, Melphalan, Thiotepa, Fludarabine

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of 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.

Who Is on the Research Team?

Paul Szabolcs, MD

Principal Investigator

UPMC Children's Hospital of Pittsburgh

Are You a Good Fit for This Trial?

Inclusion Criteria

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.

A. Primary Immunodeficiency Syndromes

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Regimen

Participants undergo a reduced-intensity conditioning regimen with alemtuzumab, hydroxyurea, fludarabine, melphalan, and thiotepa

2-3 weeks

Transplantation

Participants receive hematopoietic stem cell transplantation (HSCT) from a healthy donor

1 week

Follow-up

Participants are monitored for safety and effectiveness after treatment, including engraftment, GVHD, and immune reconstitution

up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

• Alemtuzumab
• BMT
• PBSCT
• Reduced-Intensity Chemotherapy
• UCBT

Who Is Running the Clinical Trial?

Paul Szabolcs

Lead Sponsor

Trials

8

Recruited

230+