Clinical trial NCT01821781: Immune Disorder HSCT Protocol
Official title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
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
Keywords: Immune deficiency
Immune disorders
Immune dysregulatory
Reduced Intensity
Alemtuzumab
Campath
Lead sponsor: Washington University School of Medicine
Sponsor class: OTHER
First posted: 2013-04-01
Enrollment: 20
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with donor engraftment
timeFrame: 1 year post transplant
Secondary outcomes:
measure: Major Transplant Related Toxicities
timeFrame: 1 years post transplant
measure: Time to neutrophil recovery
timeFrame: within 100 days post transplant
measure: Number of patient with acute GVHD
timeFrame: 180 days post transplant
measure: Number of participants with infectious complications
timeFrame: 2 years post transplant
measure: Time to immune reconstitution
timeFrame: 2 years post transplant
measure: Overall survival
timeFrame: 2 years post transplant
measure: Time to platelet recovery
timeFrame: within 100 days post transplant
measure: Number of patients with chronic GVHD
timeFrame: 2 years post transplant
measure: Disease free survival
timeFrame: 2 years post transplant
Other outcomes:
Locations:
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
Study officials:
References:
citation: Bhatt ST, Schulz G, Hente M, Slater A, Murray L, Shenoy S, Bednarski JJ. A single-center experience using alemtuzumab, fludarabine, melphalan, and thiotepa as conditioning for transplantation in pediatric patients with chronic granulomatous disease. Pediatr Blood Cancer. 2020 Jan;67(1):e28030. doi: 10.1002/pbc.28030. Epub 2019 Oct 10.