Immune Disorder HSCT Protocol

Overview

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Full Title of Study: “A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime”

Study Type

• Study Type: Interventional
• Study Design
  • Allocation: N/A
  • Intervention Model: Single Group Assignment
  • Primary Purpose: Treatment
  • Masking: None (Open Label)
• Study Primary Completion Date: March 2022

Interventions

• Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
  • Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF

Arms, Groups and Cohorts

• Experimental: Preparative

Clinical Trial Outcome Measures

Primary Measures

• Number of participants with donor engraftment
  • Time Frame: 1 year post transplant

Secondary Measures

• Major Transplant Related Toxicities
  • Time Frame: 1 years post transplant
• Time to neutrophil recovery
  • Time Frame: within 100 days post transplant
• Number of patient with acute GVHD
  • Time Frame: 180 days post transplant
• Number of participants with infectious complications
  • Time Frame: 2 years post transplant
• Time to immune reconstitution
  • Time Frame: 2 years post transplant
• Overall survival
  • Time Frame: 2 years post transplant
• Time to platelet recovery
  • Time Frame: within 100 days post transplant
• Number of patients with chronic GVHD
  • Time Frame: 2 years post transplant
• Disease free survival
  • Time Frame: 2 years post transplant

Participating in This Clinical Trial

Inclusion Criteria

• </= 21 years of age – Performance status >/= 40 – DLCO >/= 40% – LVEF >/=40% or LVSF >/=26% – Serum creatinine < 2x ULN – Liver enzymes </= 5x ULN – Negative pregnancy test – Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord) Exclusion Criteria:

• Known diagnosis of HIV I/II – Pregnant or breastfeeding – Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab – Uncontrolled viral infection within 1 week prior to starting alemtuzumab

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: 21 Years

Are Healthy Volunteers Accepted: No

Investigator Details

• Lead Sponsor
  • Washington University School of Medicine
• Provider of Information About this Clinical Study
  • Sponsor