Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan for Severe Combined Immunodeficiency (SCID)

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Severe Combined Immunodeficiency (SCID)+13 MoreTransplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new, less intense immunosuppressive regimen to see if it can establish engraftment of donor hematopoietic cells while still being safe.

Eligible Conditions
  • Common Variable Immunodeficiency
  • Severe Combined Immunodeficiency (SCID)
  • Chediak-Higashi Syndrome
  • Hemophagocytic Lymphohistiocytosis
  • DiGeorge Syndrome
  • Immune Dysregulation Disorders
  • Autoimmune Lymphoproliferative Syndrome
  • Wiskott-Aldrich Syndrome
  • Chronic Granulomatous Disease
  • X-linked Agammaglobulinemia
  • Immune Deficiency Disorders
  • Immune dysregulation, Polyendocrinopathy, Enteropathy, X-linked Syndrome (IPEX)
  • Hyper IgM Syndrome
  • X-Linked Lymphoproliferative Syndrome

Treatment Effectiveness

Study Objectives

1 Primary · 9 Secondary · Reporting Duration: 2 years post transplant

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

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

Preparative
1 of 1

Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan · No Placebo Group · Phase 2

Preparative
Drug
Experimental Group · 1 Intervention: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 2 years post transplant

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor
1,803 Previous Clinical Trials
2,279,429 Total Patients Enrolled

Eligibility Criteria

Age < 65 · All Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
% The person has a performance status of 40% or more.
A suitable donor is someone who is six out of six for matched stem cells for an umbilical cord blood transplant, eight out of eight for matched stem cells for a bone marrow transplant, or five to six out of six for matched stem cells for an unrelated umbilical cord blood transplant
People younger than 21 years old.
You have serum creatinine < 2x ULN.
The patient took a negative pregnancy test.
Liver enzymes > 5x ULN.