MedTrace Logo

Immune Disorder HSCT Protocol

NCT01821781 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2026-02-19

No results posted yet for this study

Summary

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.

Conditions

  • Immune Deficiency Disorders
  • Severe Combined Immunodeficiency
  • Chronic Granulomatous Disease
  • X-linked Agammaglobulinemia
  • Wiskott-Aldrich Syndrome
  • Hyper-IgM
  • DiGeorge Syndrome
  • Chediak-Higashi Syndrome
  • Common Variable Immune Deficiency
  • Immune Dysregulatory Disorders
  • Hemophagocytic Lymphohistiocytosis
  • IPEX
  • Autoimmune Lymphoproliferative Syndrome
  • X-linked Lymphoproliferative Syndrome

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

Sponsors & Collaborators

  • Washington University School of Medicine

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-04-29
Primary Completion
2025-04-25
Completion
2026-04-30

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01821781 on ClinicalTrials.gov