Table 2 Autophagy-modulating compounds in clinical trials

From: Autophagy and human diseases

Drug Autophagy target Disease Intervention ClinicalTrials.gov Identifier Phase
Chloroquine Lysosomal inhibitor Stage IV small cell lung cancer Chloroquine NCT00969306 Phase 1
Chloroquine Lysosomal inhibitor Ductal carcinoma in situ142 Chloroquine NCT01023477 Phase 1/2
Chloroquine Lysosomal inhibitor Relapsed and refractory multiple myeloma Chloroquine combined with cyclophosphamide and velcade NCT01438177 Phase 2
Chloroquine Lysosomal inhibitor Brain metastases from solid tumors143 Chloroquine plus whole-brain irradiation NCT01894633 Phase 2
Hydrochloroquine Lysosomal inhibitor Breast cancer Hydrochloroquine NCT01292408 Phase 2
Hydroxychloroquine Lysosomal inhibitor Primary renal cell carcinoma Hydroxychloroquine NCT01144169 Phase 1
Hydroxychloroquine Lysosomal inhibitor Previously treated renal cell carcinoma Hydroxychloroquine combined with mTOR inhibitor RAD001 NCT01510119 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Pancreatic cancer Hydroxychloroquine combined with gemcitabine NCT01506973 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Stage IIb or III adenocarcinoma of the pancreas Hydroxychloroquine combined with gemcitabine NCT01128296 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Non-small cell lung cancer Hydroxychloroquine combined with carboplatin, paclitaxel, and bevacizumab NCT00933803 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Recurrent advanced non-small cell lung cancer Hydroxychloroquine combined with carboplatin, paclitaxel, and bevacizumab NCT00728845 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Advanced/recurrent non-small cell lung cancer Hydroxychloroquine combined with paclitaxel, carboplatin, and bevacizumab NCT01649947 Phase 2
Hydroxychloroquine Lysosomal inhibitor Metastatic breast cancer Hydroxychloroquine combined with ixabepilone NCT00765765 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Colorectal cancer Hydroxychloroquine combined with oxaliplatin, leucovorin, 5-fluorouracil, and bevacizumab NCT01206530 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Metastatic colorectal cancer Hydroxychloroquine combined with capecitabine, oxaliplatin, and bevacizumab NCT01006369 Phase 2
Hydroxychloroquine Lysosomal inhibitor Unspecified adult solid tumor Hydroxychloroquine combined with temsirolimus NCT00909831 Phase 1
Hydroxychloroquine Lysosomal inhibitor Unspecified adult solid tumor Hydroxychloroquine combined with sunitinib NCT00813423 Phase 1
Hydroxychloroquine Lysosomal inhibitor Refractory or relapsed solid tumors Hydroxychloroquine combined with sorafenib NCT01634893 Phase 1
Hydroxychloroquine Lysosomal inhibitor Malignant solid tumor Hydroxychloroquine combined with vorinostat NCT01023737 Phase 1
Hydroxychloroquine Lysosomal inhibitor Advanced solid tumors or prostate or kidney cancer Hydroxychloroquine combined with Akt inhibitor MK2206 NCT01480154 Phase 1
Hydroxychloroquine Lysosomal inhibitor Castrate refractory prostate cancer Hydroxychloroquine combined with ABT-263 or abiraterone NCT01828476 Phase 2
Hydroxychloroquine Lysosomal inhibitor Metastatic prostate cancer Hydroxychloroquine combined with docetaxel NCT00786682 Phase 2
Hydroxychloroquine Lysosomal inhibitor Advanced cancers Hydroxychloroquine combined with sirolimus or vorinostat NCT01266057 Phase 1
Hydroxychloroquine Lysosomal inhibitor Relapsed or refractory multiple myeloma Hydroxychloroquine combined with bortezomib NCT00568880 Phase 1/2
Hydroxychloroquine Lysosomal inhibitor Lymphangioleiomyomatosis Hydroxychloroquine combined with sirolimus NCT01687179 Phase 1
Carbamazepine Autophagy inducer α1-antitrypsin deficiency liver cirrhosis117 Carbamazepine NCT01379469 Phase 2
Lithium carbonate Autophagy inducer Amyotrophic lateral sclerosis144 Lithium carbonate NCT00790582 Phase 2
Trehalose Autophagy inducer Vascular aging116 Low-dose and high-dose trehalose NCT01575288 N/A
  1. Source: The clinical trial information was queried from ClinicalTrial.gov website (http://clinicaltrials.gov/).