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Table 3 Completed and ongoing clinical trials on patients with neurofibromatosis type 2

From: The genetic landscape and possible therapeutics of neurofibromatosis type 2

ID

Initiation Date

Phase

Nation

N

Disease

Treatment

Primary Outcome

Class of inhibitor

Mechanism of action

Status

NCT02934256

7.2016

2

China

10

NF2

VS

Icotinib

Volume of tumor

RTKs

EGFR inhibitor

Completed

NCT02129647

4.2014

2

USA

13

NF2

VS

Axitinib

Expression Levels of p-S6,p-ERK, p-AKT

RTKs

VEGFR1/2/3

inhibitor

Completed

Has Results

NCT02104323

1.2014

2

China

20

NF2

VS

Endostatin

Volume of tumor

RTKs

VEGF expression inhibitor

Completed

NCT01345136

7.2015

2

USA

4

NF2

Everolimus

VS volume

mTOR

Inhibits mTORC1

Active, not recruiting

NCT01490476

1.2012

2

France

10

NF2

Everolimus

VS volume

mTOR

Inhibits mTORC1

Completed

NCT01880749

6.2013

1

USA

5

NF2

VS

Meningioma

Everolimus

Proportions of VS and meningiomas after exposure

mTOR

Inhibits mTORC1

Completed

NCT01419639

10.2011

2

USA

10

NF2

Everolimus

Radiographic Response

Change in Tumor Size

mTOR

Inhibits mTORC1

Completed, has results

NCT01767792

5.2013

2

USA

22

NF2

Progressive VS

Bevacizumab

Hearing

RTKs

VEGF-A inhibitor

Completed

Has Results

NCT01207687

10.2010

2

USA

14

NF2

VS

Bevacizumab

Hearing

RTKs

VEGF-A inhibitor

Completed

Has Results

NCT01125046

6.2010

2

USA

50

Acoustic Schwannoma

Meningioma

Ependymoma

NF1

NF2

Brain Tumor

Bevacizumab

Progression Free Survival

RTKs

VEGF-A inhibitor

Completed

Has Results

NCT04283669

2.2020

2

USA

19

NF2

Progressive VS

Crizotinib

Volumetric response rate

RTKs

c-MET inhibitor

Active, not recruiting

NCT02831257

8.2016

2

USA

18

NF2

Meningioma

AZD2014

Radiographic Response Rate

mTOR

mTOR kinase inhibitor

Completed

Has Results

NCT03071874

10.2017

2

USA

28

Grade II/ III NF2-mutated meningiomas

AZD2014

Progression Free Survival

mTOR

mTOR kinase inhibitor

Active, not recruiting

NCT04374305

6.2020

2

USA

80

NF2

VS

Brigatinib

Volumetric

response rate

RTKs

ALK and EGFR inhibitor

Recruiting

NCT02523014

8.2015

2

USA

124

NF2 Gene Mutation

GSK2256098

Progression free survival

FAK

FAK inhibitor

Recruiting

NCT03095248

5.2017

2

USA

34

NF2

VS

Meningioma

Ependymoma

Glioma

Selumetinib

Hearing

Response rate of NF2 related tumors

MEK

MEK1/2 inhibitor

Recruiting

NCT03079999

6.2018

2

USA

300

NF2

VS

Aspirin

Progressive free survival

Hippo pathway

Cyclooxygenase

Inhibitor

Recruiting

NCT01129193

1.2017

1

USA

44

Solid tumors

AR-42

Adverse events

AKT

Histone

deacetylase inhibitor

Completed

NCT02282917

12.2015

1

USA

7

NF2

VS

Meningioma

AR-42

Ratio of Phospho-AKT

AKT

Histone

deacetylase inhibitor

Terminated

Has Results

NCT05130866

6.2022

2/3

USA

89

NF2

AR-42

Progressive free survival

AKT

Histone

deacetylase inhibitor

Recruiting

NCT00863122

6.2009

1

USA

26

NF2

VS

Auditory Tumor

Lapatinib

Lapatinib Plasma Concentrations

RTKs

EGFR/ ErbB2

Inhibitor

Completed

Has Results

NCT00973739

9.2009

2

USA

21

NF2

VS

Lapatinib

Progression Free Survival

RTKs

EGFR/ ErbB2

Inhibitor

Completed

Has Results

NCT01201538

10.2010

2

Canada

2

Growing VS

Nilotinib

Volume change of VS

RTKs

Bcr-Abl inhibitor

Terminated

NCT00911248

7.2009

2

USA

11

NF2

PTC299

Volume of tumor

RTKs

VEGFA inhibitor

Terminated

NCT04085159

9.2019

1/2

China

100

Schwannomatosis NF1

NF2

Antigen-specific T cells CART/CTL and DCvac

Percentage of adverse effects

Immunotherapy

Immunotherapy

Recruiting

NCT05228015

7.2022

1

USA

158

NF2 Deficiency

IK-930

Safety and tolerability

Hippo pathway

TEAD inhibitor

Recruiting

NCT04857372

10.2021

1

USA

156

Advanced Mesothelioma and Other Solid Tumors

IAG933

Percentage of adverse effects

Hippo pathway

YAP/TEAD interaction inhibitor

Recruiting

  1. NF2: neurofibromatosis type 2; RTKs: receptor tyrosine kinases; EGFR: epidermal growth factor receptor; VS: vestibular schwannoma; VEGFR: vascular endothelial growth factor receptor; mTORC1: mechanistic target of rapamycin complex 1; cMET: c-mesenchymal-epithelial transition; ALK: anaplastic lymphoma kinase; MEK; Mitogen-activated protein kinase; AKT: Ak strain transforming