TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

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Recruiting
participants needed

17
sponsor

Johns Hopkins All Children's Hospital

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Updated on 19 February 2024

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deficiency

glomerular filtration rate

progressive disease

stem cell transplantation

hla-a

cell transplantation

adhesion

autoimmune disease

immunodeficiency

metabolic disorders

malnutrition

enteropathy

neurodegenerative disorders

wiskott-aldrich syndrome

granulomatous disease

hyper igm syndrome

ipex

haploidentical transplantation

dock8

pulse oximetry

hemophagocytic lymphohistiocytosis

chronic granulomatous disease

osteopetrosis

abnormal hematopoiesis

zap70 deficiency

ctla4 deficiency

mucopolysaccharidoses

monomac syndrome

open protocol

Summary

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

Details

Condition	Metabolic disorder, Primary Immune Deficiency Disorders
Age	21years or below
Treatment	Haploidentical Hematopoietic Cell Transplantation
Clinical Study Identifier	NCT04414046
Sponsor	Johns Hopkins All Children's Hospital
Last Modified on	19 February 2024

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	Patient with any form of primary immune deficiency/dysregulatory disorders characterized by aberrant immune function, abnormal hematopoiesis, systemic or organ specific autoimmunity and/or non-malignant lymphoproliferation. This includes, but not limited to
	Disorders of phagocytes: Chronic granulomatous disease, Leukocyte adhesion deficiency, defects of IL-10 pathway, MonoMac syndrome
	II. Defects of cellular and humoral immunity: Severe Combined Immunodeficiency
	Disorder (infants with classic SCID up to 2 years of age will be excluded due
	to other open protocol), X-linked hyper-IgM syndrome, DOCK8 deficiency, ZAP70
	deficiency, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome
	NEMO deficiency
	III. Disorder of immune dysregulation: Immunodysregulation polyendocrinopathy
	enteropathy X-linked (IPEX) syndrome, CTLA4 deficiency, LRBA deficiency, STAT1
	GOF, STAT3 GOF, X-linked lymphoproliferative disease etc
	IV. Other PIDs and immune dysregulatory disorders who can be benefitted by HCT
	as deemed appropriate by the PI and the treating immunologist
	\. Histiocytic disorders including hemophagocytic lymphohistiocytosis
	(familial HLH (types 1-5), secondary HLH (refractory to therapy or with
	recurrent episodes of hyper inflammation) and multisystem refractory
	Langerhans cell histiocytosis
	\. Metabolic disorders that could improve or stabilize after stem cell
	transplantation such as mucopolysaccharidoses, neurodegenerative disorders
	osteopetrosis, etc
	Inclusion Criteria
	Patient lacks a suitable conventional donor (HLA-identical sibling or 10/10 matched unrelated donor evaluated using the genetic loci- HLA-A,-B, -C, -DRB1, -DQB1) or has rapidly progressive disease not permitting time to identify an unrelated donor
	Patient must have a minimum genotypic identical match of 5/10
	Patients must have adequate organ function measured by
	Cardiac: asymptomatic or if symptomatic then LVEF at rest must be 40% or SF 26%
	Pulmonary: asymptomatic or if symptomatic DLCO 40% of predicted (corrected for hemoglobin) or pulse oximetry 92% on room air if the patient is unable to perform pulmonary function testing
	Renal: Creatinine clearance (CrCl) or glomerular filtration rate (GFR) must be > 50 mL/min/1.73 m2
	Hepatic: Serum conjugated (direct) bilirubin < 2.0 x ULN for age; AST and ALT < 5.0 x ULN for age
	Karnofsky or Lansky (age-dependent) performance score 50
	Signed written informed consent
Exclusion Criteria
	Pregnant or breastfeeding females
	Patient has HIV or uncontrolled fungal, bacterial or viral infections
	Patient has received prior solid organ transplant
	Patient has active GVHD (> grade II) or chronic extensive GVHD due to a previous allograft at the time of inclusion

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deficiency

glomerular filtration rate

progressive disease

stem cell transplantation

neurodegenerative disorders

wiskott-aldrich syndrome

granulomatous disease

hyper igm syndrome

ipex

haploidentical transplantation

dock8

pulse oximetry

hemophagocytic lymphohistiocytosis

chronic granulomatous disease

osteopetrosis

abnormal hematopoiesis

zap70 deficiency

ctla4 deficiency

mucopolysaccharidoses

monomac syndrome

open protocol

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Study Definition

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TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children