Clinical experiences with systemically administered siRNA-based therapeutics in cancer.

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Published in Nat Rev Drug Discov on November 16, 2015

Authors

Jonathan E Zuckerman1, Mark E Davis2

Author Affiliations

1: Department of Pathology and Laboratory Medicine, University of California Los Angeles, Los Angeles, California 90095, USA.
2: Chemical Engineering, California Institute of Technology, Pasadena, California 91125, USA.

Associated clinical trials:

A Multicenter Phase I Study of MRX34, MicroRNA miR-RX34 Liposomal Injection | NCT01829971

Safety Study of CALAA-01 to Treat Solid Tumor Cancers | NCT00689065

Study With Atu027 in Patients With Advanced Solid Cancer | NCT00938574

A Study to Determine Safety, Pharmacokinetics and Pharmacodynamics of Intravenous TKM 080301 in Neuroendocrine Tumors (NET) and Adrenocortical Carcinoma (ACC) Patients | NCT01262235

EphA2 Gene Targeting Using Neutral Liposomal Small Interfering RNA Delivery | NCT01591356

I5NP for Prophylaxis of Delayed Graft Function in Kidney Transplantation | NCT00802347

Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment | NCT01158079

Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver Involvement | NCT00882180

A Phase II Study of siG12D LODER in Combination With Chemotherapy in Patients With Unresectable Locally Advanced Pancreatic Cancer | NCT01676259

Phase I - Escalating Dose Study of siG12D LODER (Local Drug EluteR) in Patients With Locally Advanced Adenocarcinoma of the Pancreas, and a Single Dose Study of siG12D LODER (Local Drug EluteR) in Patients With Non-operable Adenocarcinoma of the Pancreas | NCT01188785

TKM 080301 for Primary or Secondary Liver Cancer | NCT01437007

Atu027 Plus Gemcitabine in Advanced or Metastatic Pancreatic Cancer (Atu027-I-02) (Atu027-I-02) | NCT01808638

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis | NCT01960348

A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers | NCT01814839

Phase I, Multicenter, Dose Escalation Study of DCR-MYC in Patients With Solid Tumors, Multiple Myeloma, or Lymphoma | NCT02110563

Phase Ib/2, Multicenter, Dose Escalation Study of DCR-MYC in Patients With Hepatocellular Carcinoma | NCT02314052

Safety, Pharmacokinetics and Preliminary Anti-Tumor Activity of Intravenous TKM-080301 in Subjects With Advanced Hepatocellular Carcinoma | NCT02191878

Safety and Tolerability Study of SNS01-T in Relapsed or Refractory B Cell Malignancies (Multiple Myeloma, B Cell Lymphoma, or Plasma Cell Leukemia (PCL) | NCT01435720

A Study of PNT2258 in Patients With Advanced Solid Tumors | NCT01191775

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | NCT02292186

Study of PNT2258 for Treatment of Relapsed or Refractory Non-Hodgkin's Lymphoma | NCT01733238

PNT2258 for Treatment of Patients With r/r DLBCL (Wolverine) | NCT02226965

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