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Table 4 Interventions targeting lysosomes

From: Role of lysosomes in physiological activities, diseases, and therapy

Disease

Intervention

Stage of development

Comment

NCT number

Atherosclerosis

Temsirolimus ± Dexamethasone

Phase II

mTOR inhibitor

NCT03942601

Temsirolimus

Phase III

mTOR inhibitor

NCT04433572

Chloroquine

Not applicable

Autophagy/lysosome inhibitor

NCT00455403

Hydroxychloroquine

Phase IV

Autophagy/lysosome inhibitor

NCT04161339

Alzheimer's disease

Trehalose

Phase I

Autophagy inducer

NCT04663854

Hydralazine

Phase III

Autophagy inducer

NCT04842552

Rapamune

Phase I

Autophagy inducer

NCT04200911

Rapamycin

Phase II

Autophagy inducer

NCT04629495

Parkinson’s disease

Exablate BBBD with Cerezyme

Not applicable

Enzyme replacement therapy

NCT04370665

Systemic lupus erythematosus

Hydroxychloroquine or chloroquine

Phase II

Autophagy/lysosome inhibitor

NCT01946880

Sirolimus

Phase II

Autophagy inducer

NCT04582136

Sirolimus

Phase II

Autophagy inducer

NCT04736953

Rapamycin

Phase II

Autophagy inducer

NCT00779194

Crohn’s disease

Rapamycin

Not applicable

Autophagy inducer

NCT02675153

Ciprofloxacin + Doxycycline + Hydroxychloroquine + Budesonide

Phase II

Combination of hydroxychloroquine with others

NCT01783106

Rheumatoid arthritis

Temsirolimus

Phase II

mTOR inhibitor

NCT00076206

Sirolimus

Phase I/II

Autophagy inducer

NCT00392951

Infliximab + DMARDs (methotrexate; chloroquine; leflunomidum; cyclosporin A; sulfasalazine; OM 89)

Phase III

Combination of chloroquine with others

NCT00521924

Multiple sclerosis

Temsirolimus

Phase II

mTOR inhibitor

NCT00228397

Sirolimus

Phase I/II

Autophagy inducer

NCT00095329

Fabry disease

Agalsidase alfa

Phase III

Enzyme replacement therapy

NCT01298141

Agalsidase beta

Phase IV

Enzyme replacement therapy

NCT00081497

Tay-Sachs disease

AXO-AAV-GM2

Phase I

Gene therapy

NCT04669535

Mucopolysaccharidosis diseases

Aldurazyme (for type I)

Phase III

Enzyme replacement therapy

NCT00258011

Idursulfase (for type II)

Phase II/III

Enzyme replacement therapy

NCT00630747

SAF-301 (for type III)

Phase I/II

Gene therapy

NCT02053064

ABO-102 (for type III)

Phase I/II

Gene therapy

NCT04088734

Naglazyme (for type IV)

Phase IV

Enzyme replacement therapy

NCT00299000

elosulfase alfa (for type IV)

Not applicable

Enzyme replacement therapy

NCT03204370

AAV2/8.TBG.hARSB (for type IV)

Phase I/II

Gene therapy

NCT03173521

UX003 (for type VII)

Phase I/II

Enzyme replacement therapy

NCT01856218

Pompe disease

Alglucosidase alfa

Phase IV

Enzyme replacement therapy

NCT04676373

Gaucher disease

Imiglucerase

Phase IV

Enzyme replacement therapy

NCT04656600

Velaglucerase alfa

Phase IV

Enzyme replacement therapy

NCT04718779

taliglucerase alfa

Phase IV

Enzyme replacement therapy

NCT04002830

Non-small cell lung cancer

Binimetinib + Hydroxychloroquine

Phase II

Combination Therapy

NCT04735068

Paclitaxel + Carboplatin ± Bevacizumab ± Hydroxychloroquine

Phase II

Combination Therapy

NCT01649947

Bevacizumab + Carboplatin + Paclitaxel + Hydroxychloroquine

Phase I/II

Combination Therapy

NCT00728845

Sunitinib + Rapamycin

Phase I

Combination Therapy

NCT00555256

Neratinib ± Temsirolimus

Phase II

Combination Therapy

NCT01827267

Small cell lung cancer

Chloroquine

Phase I

Autophagy/lysosome inhibitor

NCT00969306

Etoposide + Carboplatin + Atezolizumab + BNT411 (TLR7/8 agonist)

Phase I/II

Combination Therapy

NCT04101357

Colon cancer

Bupivacaine liposome suspension (for pain control)

Phase IV

Bupivacaine slowly released from lysosomes

NCT02052557

FOLFOX/bevacizumab ± Hydroxychloroquine

Phase I/II

Combination Therapy

NCT01206530

Vorinostat + Hydroxychloroquine

Phase II

Combination Therapy

NCT02316340

nab-rapamycin + mFOLFOX6 and Bevacizumab

Phase I

Combination Therapy

NCT03439462

Temsirolimus + Cetuximab

Phase I

Combination Therapy

NCT00593060

Pembrolizumab + Poly-ICLC (TLR3 agonist)

Phase I/II

Combination Therapy

NCT02834052

FOLFIRI + Cetuximab + IMO-2055 (TLR9 agonist)

Phase I

Combination Therapy

NCT00719199

Breast cancer

Hydrochloroquine

Phase II

Autophagy/lysosome inhibitor

NCT01292408

Ixabepilone + Hydroxychloroquine

Phase I/II

Combination Therapy

NCT00765765

Letrozole + Palbociclib + Hydroxychloroquine

Phase I/II

Combination Therapy

NCT03774472

Chloroquine

Phase II

Autophagy/lysosome inhibitor

NCT02333890

Zoledronic acid + Odanacatib (cathepsin K inhibitor)

Phase I/II

Combination Therapy

NCT00399802

Trastuzumab + Rapamycin

Phase II

Combination Therapy

NCT00411788

Inetetamab + Rapamycin + Chemotherapy

Phase III

Combination Therapy

NCT04736589

Rapamycin

Phase II

mTOR inhibitor

NCT02642094

Radiation + Cyclophosphamide + Imiquimod (TLR7 agonist)

Phase I/II

Combination Therapy

NCT01421017

Hepatocellular cancer

Sorafenib ± Hydroxychloroquine

Phase II

Combination Therapy

NCT03037437

temsirolimus

Phase II

mTOR inhibitor

NCT01079767

RO7119929

Phase I

TLR7 agonist

NCT04338685

Cholangiocarcinoma

ABC294640 ± Hydroxychloroquine

Phase II

Combination Therapy

NCT03377179

Gastrointestinal cancer

Cobimetinib + Atezolizumab + Hydroxychloroquine

Phase I/II

Combination Therapy

NCT04214418

Prostate cancer

Hydroxychloroquine

Early Phase 1

Autophagy/lysosome inhibitor

NCT02421575

Docetaxel ± Hydroxychloroquine

Phase II

Combination Therapy

NCT00786682

Odanacatib

Phase II

Cathepsin K inhibitor

NCT00691899

Temsirolimus

Phase II

mTOR inhibitor

NCT00919035

Bevacizumab + Temsirolimus

Phase I/II

Combination Therapy

NCT01083368

Temsirolimus + Diphenhydramine

Phase II

Combination Therapy

NCT00887640

Biliary cancer

Trametinib + Hydroxychloroquine

Phase II

Combination Therapy

NCT04566133

Ovarian cancer

Temsirolimus

Phase II

mTOR inhibitor

NCT00926107

Cisplatin + Pembrolizumab + Rintatolimod (TLR3 agonist)

Phase I/II

Combination Therapy

NCT03734692

OC-L + Ampligen (TLR3 agonist)

Phase I/II

Combination Therapy

NCT01312389

Pancreatic cancer

LY3214996 ± Hydroxychloroquine sulfate

Phase II

Combination Therapy

NCT04386057

Gemcitabine + Abraxane + Hydroxychloroquine

Phase I/II

Combination Therapy

NCT01506973

Binimetinib + Hydroxychloroquine

Phase I

Combination Therapy

NCT04132505

Sirolimus

Phase II

mTOR inhibitor

NCT00499486

Bevacizumab + Temsirolimus

Phase II

Combination Therapy

NCT01010126

Radiation Therapy + Nivolumab + SD-101 (TLR9 agonist)

Phase I

Combination Therapy

NCT04050085

INCAGN01949 + CMP-001 (TLR9 agonist)

Phase I

Combination Therapy

NCT04387071

Melanoma

Dabrafenib + Trametinib ± Hydroxychloroquine

Phase II

Combination Therapy

NCT04527549

Sorafenib + Temsirolimus

Phase I

Combination Therapy

NCT00349206

MART-1 Antigen ± GLA-SE (TLR4 agonist)

Early Phase I

Combination Therapy

NCT02320305

NY-ESO-1 protein + Montanide + Poly ICLC (TLR3 agonist)

Phase I/II

Vaccine

NCT01079741

Multiple myeloma

Bortezomib + hydroxychloroquine

Phase I

Combination Therapy

NCT00568880

Brain neoplasms

Temsirolimus

Phase I

mTOR inhibitor

NCT00949026

Head and neck cancer

Temsirolimus + Weekly Paclitaxel + Carboplatin

Phase I/II

Combination Therapy

NCT01016769

Cetuximab + EMD 1201081 (TLR9 agonist)

Phase II

Combination Therapy

NCT01040832

Renal cell cancer

Sunitinib + Temsirolimus

Phase I

Combination Therapy

NCT01122615

Bladder cancer

Temsirolimus

Phase II

mTOR inhibitor

NCT01827943

Sirolimus

Early Phase I

mTOR inhibitor

NCT02753309

Glioma

Tumor-lysate pulsed DC vaccination + adjuvant poly ICLC (TLR3 agonist)

Phase II

Vaccine

NCT01204684

Esophageal cancer

URLC10-177 + TTK-567 + CpG-7909 (TLR9 agonist)

Phase I/II

Vaccine

NCT00669292

Follicular Lymphoma

Radiation Therapy + Ibrutinib + SD-101 (TLR9 agonist)

Phase I/II

Combination Therapy

NCT02927964

Non-Hodgkin Lymphoma

local irradiation + CPG 7909 (TLR9 agonist)

Phase I/II

Combination Therapy

NCT00185965

Advanced or metastatic tumor combined with COVID-19

Avdoralimab + Monalizumab + GNS651 (autophagy inhibitor)

Phase II

Combination Therapy

NCT04333914

Nonspecific cancer

GSK1795091

Phase I

TLR4 agonist

NCT02798978

Anti-Cancer Agent + SHR2150 (TLR7 agonist)

Phase I/II

Combination Therapy

NCT04588324

Echopulse + PD-1 + Imiquimod (TLR7 agonist)

Phase I

Combination Therapy

NCT04116320

Durvalumab + MEDI9197 (TLR7/8 agonist)

Phase I

Combination Therapy

NCT02556463

Ipilimumab + Nivolumab + Tilsotolimod (TLR9 agonist)

Phase I

Combination Therapy

NCT04270864

NY-ESO-1 protein + Montanide ± Resiquimod (TLR7/8 agonist)

Phase I

Vaccine

NCT00821652

BMS 986178 + SD-101 (TLR9 agonist)

Phase I

Combination Therapy

NCT03831295

  1. Function of several repeatedly mentioned chemical: mTOR inhibitor: Temsirolimus, Sirolimus, Rapamycin, Rapamune; autophagy/lysosome inhibitor: Hydroxychloroquine, Chloroquine. +: combination; +/−: with or without