Immune Disorder HSCT Protocol

Phase 2

Recruiting

• Conditions: Immune Deficiency Disorders; Severe Combined Immunodeficiency; Chronic Granulomatous Disease; Common Variable Immune Deficiency; Hemophagocytic Lymphohistiocytosis; X-linked Lymphoproliferative Syndrome; X-linked Agammaglobulinemia; Wiskott-Aldrich Syndrome; Hyper-IgM; Autoimmune Lymphoproliferative Syndrome
• Interventions: Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

• Registration Number: NCT01821781
• Lead Sponsor: Washington University School of Medicine

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-03
• Study First Submitted: 2013-03-19
• Study First Submitted QC: 2013-03-26
• Study First Posted: 2013-04-01
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-01
• Last Update Posted: 2024-07-03
• Primary Completion: 2027-03
• Study Completion: 2027-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• </= 28 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)

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Preparative	Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan	-

Primary Outcome Measures

Name	Time	Method
Number of participants with donor engraftment	1 year post transplant

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Washington University; 🇺🇸 Saint Louis, Missouri, United States