NP2 Enkephalin For Treatment of Intractable Cancer Pain

A Phase II, Randomized, Double Blind, Placebo-controlled, Multicenter Study to Investigate the Impact of NP2 in Subjects With Intractable Pain Due to Malignancy

The purpose of this study is to examine the impact of intradermal delivery of NP2 on pain scores and pain medication usage in subjects with intractable pain due to malignant disease. A second purpose is to confirm safety and secondary efficacy measurements.

Study Overview

• Status: Completed
• Conditions: Neoplasms; Intractable Pain
• Intervention / Treatment: Biological: NP2; Biological: Placebo

Detailed Description

Chronic severe pain remains a significant unmet medical need in patients that have progressive cancer. Existing treatments have limited efficacy and also suffer significant side effects. This is a multi-center, randomized, double blind, placebo-controlled clinical trial designed to evaluate the impact of intradermal injection of NP2 in subjects who have intractable pain due to malignant disease. NP2 is a gene transfer vector engineered to express human preproenkephalin, a gene naturally involved in pain control. Delivery of NP2 directly to the site of pain caused by cancer is intended to provide increased Enkephalin peptides, which bind to opioid receptors, that may allow better pain control.

• Study Type: Interventional
• Enrollment (Actual): 33
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85050
      • Hope Research Institute
    • Tucson, Arizona, United States, 85715
      • Arizona Clinical Research Center
  • California
    • Corona, California, United States, 92879
      • Compassionate Cancer Care Medical Group, Inc.
    • Fresno, California, United States, 93720
      • Cancer Care Associates
    • La Mesa, California, United States, 91942
      • Triwest Research Associates
    • Los Angeles, California, United States, 90033
      • White Memorial Medical Center
    • Oakland, California, United States, 94609
      • Hematology Oncology Associates
  • Florida
    • Miami, Florida, United States, 33175
      • Advanced Pharma Cr
  • Georgia
    • Newnan, Georgia, United States, 30265
      • Better Health Clinical Research Inc
  • Illinois
    • Champaign, Illinois, United States, 61820
      • Christie Clinic
  • Indiana
    • Evansville, Indiana, United States, 47714
      • Global Scientific Innovations
  • Montana
    • Missoula, Montana, United States, 59802
      • Montana Cancer Institute Foundation
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27103
      • Center for Clinical Research
  • Ohio
    • Middletown, Ohio, United States, 45042
      • Signal Point Clinical Research Center
  • Oregon
    • Eugene, Oregon, United States, 97401
      • Pain Research of Oregon
  • Rhode Island
    • Cranston, Rhode Island, United States, 02920
      • Hematology Oncology Associatesof Rhode Island
  • Texas
    • San Antonio, Texas, United States, 78217
      • Medical Therapy and Research
  • Washington
    • Spokane, Washington, United States, 99208
      • Medical Oncology Associates

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• Histologically confirmed malignant disease.
• Intractable pain related to malignancy.
• Females must be postmenopausal or practicing birth control.
• Able to provide appropriate written consent.

Main Exclusion Criteria:

• Positive pregnancy test prior to receiving study treatment.
• Serious uncontrolled medical condition other than malignancy (e.g. congestive heart failure, coagulopathy, uncontrolled diabetes).
• Evidence of active Hepatitis B, Hepatitis C, or HIV infection.
• Evidence of viral, bacterial, or fungal infection in the planned treatment area.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Active NP2; Single intradermal dose of active NP2. An open label study extension will offer up to two additional doses of active NP2 between weeks 4-10 following the previous dose.	Biological: NP2; NP2 is a replication defective HSV-1 based gene transfer vector engineered to express human preproenkephalin. The drug will be injected intradermally corresponding to the distribution of the malignancy-related pain. The total amount to be injected will be a dose volume of 1.0 ml delivered in a single session on Study Day 0.
Placebo Comparator: Placebo; Single intradermal dose of placebo (vehicle). An open label study extension will offer up to two additional doses of active NP2 between weeks 4-10 following the previous dose.	Biological: Placebo; The placebo (vehicle) will be injected intradermally corresponding to the distribution of the malignancy-related pain. The total amount to be injected will be a dose volume of 1.0 ml delivered in a single session on Study Day 0.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pain Measured by the Numerical Rating Scale (NRS)	• Change from baseline of the average daily NRS pain score (scale of 0 to 10 ) of Placebo compared to Active NP2 cohorts.	Days -5 to -1 predosing and days 3 to 14 postdosing

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Opioid Pain Medication Usage Morphine Equivalent Units (MEU)	•Change from baseline of use of opioid pain medication average daily MEU of Placebo compared to Active NP2 cohorts	Days -5 to -1 predosing and 3 to 14 postdosing
Quality of Life ECOG	•Quality of Life measured by Eastern Cooperative Oncology Group Performance Status (ECOG) assessment at follow-up visits compared to baseline of Placebo compared to Active NP2 cohorts.	Baseline and Week 1, 2 and 4
Quality of Life SF-12	•Quality of Life measured by the 12-Item Short Form Health Survey (SF-12v2) at follow-up visits compared to baseline of Placebo compared to Active NP2 cohorts.	Baseline and Week 1, 2 and 4
Pain SF-MPQ	•Short Form McGill Pain Questionnaire (SF-MPQ-2) assessment at follow-up visits compared to baseline of Placebo compared to Active NP2 cohorts	Baseline and Week 1, 2 and 4

Collaborators and Investigators

• Sponsor: Diamyd Inc
• Collaborators: Paragon Biomedical; invivodata, Inc.
• Investigators: Study Director: Darren Wolfe, Ph.D., Diamyd Inc; Principal Investigator: David Fink, M.D., University of Michigan

Study record dates

Study Major Dates

• Study Start: January 1, 2011
• Primary Completion (Actual): July 1, 2012
• Study Completion (Actual): November 1, 2013

Study Registration Dates

• First Submitted: February 4, 2011
• First Submitted That Met QC Criteria: February 8, 2011
• First Posted (Estimate): February 9, 2011

Study Record Updates

• Last Update Posted (Estimate): August 8, 2014
• Last Update Submitted That Met QC Criteria: July 28, 2014
• Last Verified: July 1, 2014

More Information

Terms related to this study

• Keywords: opioid; pain; cancer; gene therapy; intractable; malignancy; enkephalin
• Additional Relevant MeSH Terms: Pain; Neurologic Manifestations; Pain, Intractable
• Other Study ID Numbers: NP2/P2/10/2