Immune Disorder HSCT Protocol

Chronic Granulomatous Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01821781
• Other study ID #: 201301135
• Status: Recruiting
• Phase: Phase 2
• Start date: March 2013
• Est. completion date: March 2027

Study information

• Verified date: August 2023
• Source: Washington University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: March 2027
• Est. primary completion date: March 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

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)

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

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
United States	Washington University	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Washington University School of Medicine

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with donor engraftment		1 year post transplant
Secondary	Major Transplant Related Toxicities		1 years post transplant
Secondary	Time to neutrophil recovery		within 100 days post transplant
Secondary	Number of patient with acute GVHD		180 days post transplant
Secondary	Number of participants with infectious complications		2 years post transplant
Secondary	Time to immune reconstitution		2 years post transplant
Secondary	Overall survival		2 years post transplant
Secondary	Time to platelet recovery		within 100 days post transplant
Secondary	Number of patients with chronic GVHD		2 years post transplant
Secondary	Disease free survival		2 years post transplant