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Table 1 Targeted therapy for hemophagocytic lymphohistiocytosis (HLH) in ongoing clinical trials

From: Hemophagocytic lymphohistiocytosis: current treatment advances, emerging targeted therapy and underlying mechanisms

Target

NCT Number

HLH type

Interventions

Phase

No. Patient

Start Date

Study status

IFN-γ

NCT06038422

R/R HLH

GTP

3

15

2023/9/15

Not yet recruiting

IFN-γ

NCT05744063

Primary HLH

Emapalumab

4

13

2023/2/3

Active not recruiting

IFN-γ

NCT05001737

MAS

Emapalumab

3

41

2021/12/15

Recruiting

JAK1/2

NCT06244862

Severe HLH

Ruxolitinib

2

42

2024/2/1

Not yet recruiting

JAK1/2

NCT06160791

HLH

Ruxolitinib + ED

2

36

2024/2/1

Not yet recruiting

JAK1/2

NCT05762640

Primary HLH

Ruxolitinib

2

20

2024/3/1

Not yet recruiting

JAK1/2

NCT05491304

Pediatric HLH

Ruxolitinib + ED

4

400

2022/9/1

Recruiting

JAK1/2

NCT05137496

MAS

Ruxolitinib + methylprednisolone

3

40

2022/6/1

Not yet recruiting

JAK1/2

NCT04999878

Lymphoma-associated HLH

RUE-DDGP

4

30

2021/5/30

Recruiting

JAK1/2

NCT04551131

HLH

Ruxolitinib + ED

1/2

62

2021/7/13

Recruiting

JAK1/2

NCT04120090

R/R HLH

ruxolitinib

3

80

2019/7/1

Unknown

JAK1/2

NCT03795909

R/R HLH

Ruxolitinib + Dex

1/2

50

2017/3/1

Unknown

JAK1

NCT05063110

Non-severe HLH

Itacitinib

2

63

2022/5/1

Recruiting

JAK2

NCT04326348

HLH

TQ05105

1

40

2020/7/17

Unknown

IL-6

NCT02007239

HLH

Tocilizumab

2

NA

2013/12/1

Withdrawn

IL-1

NCT02780583

MAS

Anakinra

1

40

2016/5/15

Active not recruiting

IL-18

NCT03512314

NLRC4-MAS, XIAP Deficiency

Tadekinig alfa (IL-18BP)

3

10

2018/1/24

Active not recruiting

CD52

NCT01821781

HLH

Conditioning regimen before HSCT: Flu + Mel + Alem + thiotepa

2

20

2013/3/1

Recruiting

CD20

NCT05384743

EBV-HLH

Rituximab

3

30

2022/2/1

Unknown

CD20

NCT05258136

EBV-HLH

CD20 monoclonal antibody

NA

20

2021/6/1

Enrolling by invitation

PD-1

NCT05008666

ENKTL-HLH

Sintilimab + chidamide + azacitidine

2

37

2021/12/1

Unknown

PD-1

NCT05775705

HLH

PD-1 antibody + L-DEP

3

25

2023/8/1

Not yet recruiting

PD-1

NCT05315336

HLH

PD-1 antibody + L-DEP

3

50

2022/6/1

Not yet recruiting

PD-1

NCT05164978

HLH

PD-1 antibody + DEP

NA

20

2021/5/1

Unknown

PD-1

NCT05039580

HLH

PD-1 antibody + L-DEP

4

36

2021/5/15

Unknown

PD-1

NCT04084626

HLH

PD-1 antibody + lenalidomide

3

40

2019/9/15

Unknown

PD-1

NCT04944511

HLH after allo-HSCT

Toripalimab

NA

20

2021/7/1

Unknown

PD-1

NCT04690036

EBV-HLH after transplantation

Toripalimab

1

20

2021/7/1

Unknown

IL-18 + IL-1β

NCT04641442

NLRC4-GOF, XIAP Deficiency, CDC42 Mutations

MAS825 (anti-IL-1β/IL-18)

2

18

2020/12/18

Recruiting

  1. IFN-γ interferon-γ; JAK1/2 Janus kinase 1/2; IL-1 interleukin-1; IL-6 interleukin-6; IL-18 interleukin-18; PD-1 programmed death 1; IL-1β interleukin-1 beta; R/R relapsed/refractory; MAS macrophage activation syndrome; Allo-HSCT allogeneic hematopoietic stem cell transplant; EBV Epstein-Barr virus; GTP emapalumab + teniposide + methylprednisolone; ED etoposide + dexamethasone; Dex dexamethasone; RUE-DDGP Ruxolitinib + Etoposide + cis-platinum + Dexamethasone + Gemcitabine + Pegaspargase; Flu + Mel + Alem + thiotepa fludarabine + melphalan + alemtuzumab + thiotepa; DEP liposomal doxorubicin, etoposide, and methylprednisolone; L-DEP PEG-asparaginase combined with liposomal doxorubicin, etoposide, and methylprednisolone; IL-18BP IL-18 binding protein