NCT02110563

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

Phase I, Multicenter, Cohort Dose Escalation Trial to Determine the Safety, Tolerance, and Maximum Tolerated Dose of DCR-MYC, a Lipid Nanoparticle (LNP)-Formulated Small Inhibitory RNA (siRNA) Oligonucleotide Targeting MYC, in Patients With Refractory Locally Advanced or Metastatic Solid Tumor Malignancies, Multiple Myeloma, or Lymphoma

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 50 (actual)
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety and tolerability of the investigational anticancer drug DCR-MYC. DCR-MYC is a novel synthetic double-stranded RNA in a stable lipid particle suspension that targets the oncogene MYC. MYC oncogene activation is important to the growth of many hematologic and solid tumor malignancies. In this study the Sponsor proposes to study DCR-MYC and its ability to inhibit MYC and thereby inhibit cancer cell growth.

Detailed description

In this first-in human study, DCR-MYC will be administered by 2 hour intravenous (IV) infusion, once weekly for 2 weeks followed by a rest week (3 weeks = 1 cycle), to patients with either solid tumor malignancies, multiple myeloma, or non-Hodgkins lymphoma that have not responded to previous treatment. The highest safe dose of DCR-MYC that can be administered will be identified. In addition, the pharmacokinetic (PK) profile, potential pharmacodynamic (PD) effects, as well as the antitumor activity of DCR-MYC will be evaluated. There will be 2 expansion cohorts at the maximum tolerated dose (MTD) (or highest safe dose identified for further study which may be lower): * Biopsy Cohort: 6 patients, tumor biopsies to be performed pre-dosing and Cycle 2/Day 11; same assessments as dose escalation cohorts * PNET Cohort: Up to 20 patients with pancreatic neuroendocrine tumors; same assessments as dose escalation cohorts, however fewer PD (cytokine) assessments and no PK assessments

Conditions

• Solid Tumors
• Multiple Myeloma
• Non-Hodgkins Lymphoma
• Pancreatic Neuroendocrine Tumors
• PNET
• NHL

Interventions

Type	Name	Description
DRUG	DCR-MYC	Dosing: 2 hour IV infusion on Day 1 and 8 of each 21 day cycle. Starting dose: 0.1mg/kg/dose Dose escalation: 100%, 50%, or 25% increase in subsequent cohorts depending upon toxicity. Number of cycles: until progression or unacceptable toxicity develops.

Timeline

• Start date: 2014-04-01
• Primary completion: 2016-10-05
• Completion: 2016-11-03
• First posted: 2014-04-10
• Last updated: 2024-07-11

Locations

6 sites across 1 country: United States