Table 6.

Summary of the current clinical trials targeting HLH syndrome

ACTIVE, RECRUITING PATIENTS
Clinical trial	Type of study	Targeted disease(s)	Drug regimen	Patient population	Sponsor
NCT01104025	Phase II	HLH	ATG, dexamethasone, etoposide	< 18 years	Children’s Hospital Medical Center, Cincinnati, OH, USA
NCT01547143	Phase II	HLH, excluding malignancy and MAS	cyclosporine, dexamethasone, etoposide, allo-SCT	18-80 years	Asan Medical Center, Seoul, Korea
NCT01818908	Phase II	NHL with HLH	DA-EPOCH	15-80 years	Jiangsu Province Hospital, Nanjing, Jiangsu, China
NCT01818492	Phase II	reactivated HLH, excluding MAS and malignancy	NI-0501, cyclosporine, dexamethasone	< 18 years	NovImmune SA, Geneva, Switzerland
NCT00368355	Phase II	HLH, XLP, (AML, ALL, MDS, NHL, CML)	T-cell depleted haploidentical- HSCT	< 55 years	Baylor College of Medicine, Houston, TX, USA
NCT01494103	Phase I	HLH, XLP, (AML, ALL, MDS, NHL, CML)	haplo-identical T cells, caspase 9 suicide gene, AP1903	Undefined	Baylor College of Medicine, Houston, TX, USA
NCT01125319	N/A	acquired HLH	Blood sampling to investigate NK and T cell function	> 18 years	Assistance Publique Hopitaux De Marseille, Marseille, France
NCT01095146	N/A	MAS, excluding HLH	retrospective study to define MAS diagnostic criteria	Undefined	Amrita Institute of Medical Sciences & Research Center, Kerala, India
NCT01652092	Phase II	HLH, SCID, XLP, Griscelli, Chediak-Higashi, etc	myeloablative and RIC allo- HSCT	< 50 years	Masonic Cancer Center, University of Minnesota, Minneapolis, MN, USA
NCT01821781	Phase II	HLH, SCID, XLP, Griscelli, Chediak-Higashi, etc	RIC-HSCT	< 21 years	Washington University School of Medicine, St. Louis, MO, USA
NCT00919503	Phase II	nonmalignant diseases	allo-HSCT, treosulfan	< 54 years	Fred Hutchinson Cancer Research Center, Seattle, WA, USA
NCT01050855	Phase II	nonmalignant diseases, immune deficiencies	RIC-HSCT	6 months - 25 years	Children’s Hospital of Philadelphia, Philadelphia, PA, USA
NCT00553098	Phase II	nonmalignant diseases, immune deficiencies	RIC-HSCT	< 54 years	Fred Hutchinson Cancer Research Center, Seattle, WA, USA
ACTIVE, NOT RECRUITING PATIENTS
Clinical trial	Type of study	Targeted disease(s)	Drug regimen	Patient population	Sponsor
NCT00426101	Phase III	HLH	HLH-2004, allo-HSCT	< 18 years	Karolinska University Hospital, Stockholm, Sweden
NCT00334672	Phase III	HLH	etoposide, dexamethasone, cyclosporine, allo HSCT	< 17 years	Children’s Cancer and Leukaemia Group, UK
NCT00006056	Phase II	HLH; XLP type 1, 2; Chediak- Higashi	allo-HSCT	< 55 years	Fairview University Medical Center, Minneapolis, MN, USA
NCT00710892	Phase I	HLH, AML, MDS, CML, ALL	T cell depletion, inducible caspase 9 suicide gene	< 65 years	Baylor College of Medicine, Houston, TX, USA
NCT00176826	Phase II/ III	HLH, XLP, Chediak-Higashi, Griscelli,	in vivo T cell depletion, allo- HSCT	< 55 years	Masonic Cancer Center, University of Minnesota, Minneapolis, MN
NCT00176865	Phase II	HLH, XLP, Chediak-Higashi, Griscelli,	non-myeloablative HSCT	< 35 years	Masonic Cancer Center, University of Minnesota, Minneapolis, MN

Abbreviations used: AML = acute myeloid leukemia

ALL = acute lymphoblastic leukemia

allo = allogeneic

ATG = anti-thymocyte globulin

AP-1903 = lipid-permeable tacrolimus analogue with homodimerizing activity, used to “activate” caspase 9 suicide gene

CML = chronic myeloid leukemia

DA EPOCH = dose adjusted EPOCH (etoposide, doxorubicin, vincristine, rituximab, cyclophosphamide, prednisone)

HLH = hemophagocytic lymphohistiocytosis

HLH-2004 = HLH-2004 treatment protocol (dexamethasone, etoposide, cyclosporine)

HSCT = hematopoietic stem cell transplant

MAS = macrophage activation syndrome

MDS= myelodysplastic syndrome

NHL = non-Hodgkin lymphoma

NI-0501 = anti-interferon γ monoclonal antibody

RIC = reduced intensity conditioning

SCID = severe combined immunodeficiency

XLP = X-linked lymphoproliferative disease