Last updated on April 2019

Periganglionic Resiniferatoxin for the Treatment of Intractable Pain Due to Cancer-induced Bone Pain


Brief description of study

Background

Cancer-induced bone pain (CIBP) is common in people with cancer. Bone cancer can also lead to anxiety, depression, and reduced mobility and quality of life. Researchers believe a research drug called resiniferatoxin (RTX) may be able to help.

Objective

To learn whether RTX is safe and can reduce cancer induced bone pain.

Eligibility

People ages 18 and older with CIBP that is not relieved by standard treatments

Design

Participants will have up to 6 outpatient visits over about 7 months. These will include:

Medical history

Physical exam

Blood and urine tests.

Thermal testing: a disk placed on the skin to test ability to sense temperature in and around the area of pain

Chest x-ray

EKG: stickers are placed on the chest to measure heart signals

ECG: measures electrical activity of the heart

Participants will have 1 inpatient visit lasting 2-4 days. This will include:

Catheter inserted into a vein in the arm. They are given anesthesia, sedation, and x-ray contrast.

A needle is passed through the skin of the back to inject the RTX.

Participants will keep a log of the pain medications they take after surgery.

Participants will be called 1 week and 2, 3, and 4 months after the injection.

Participants will be mailed surveys and questionnaires to complete 2, 3, and 4 months after the injection.

Detailed Study Description

Cancer-induced bone pain (CIBP) is a common clinical problem.1 While primary osteosarcoma is relatively uncommon, bone metastases frequently cause cancer-related pain with metastatic spread to bone in 60-84% of cases. 2 Resiniferatoxin (RTX) is an ultrapotent agonist analog of capsaicin that targets a receptor expressed on specific dorsal root sensory ganglia (DRG) neurons and is expected to reduce pain within the targeted zone. The overall program goal is to develop a new treatment for intractable chronic pain below the mid-thoracic level resulting from CIBP that has not been controlled with conservative treatments.

Primary Objectives

To determine the maximum tolerated dose (MTD) of RTX when injected near one or more DRGs, and to characterize its safety/toxicity profile and identify any dose-limiting toxicity (DLT).

Study Population

Up to 30 adult subjects are estimated to be necessary for enrollment to have up to 16 subjects who will receive RTX and provide study-related assessment results through the 30-day time point.

Design

The study is a single center, open-label Phase 1b dose escalation safety and efficacy trial for adult subjects with intractable pain due to CIBP below the mid-thoracic level who meet all other eligibility criteria.

Subjects who have undergone the informed consent (IC) process and signed the approved IC form for the study will be assigned a Screen # (S1, S2, etc.). Those subjects who meet all inclusion and exclusion criteria will undergo various study procedures and then be scheduled for the unilateral periganglionic (PG) DRG injection(s) (a maximum of 3 contiguous levels) under fluoroscopic guidance to treat the targeted DRGs demonstrated to be responsible for the chronic CIBP.

Subjects who are screen failures and those who prematurely terminate participation will be replaced until 16 subjects have received the injection(s) and completed study-related assessments through the day 30 (D30) time point.

Outcome Measures

The primary outcome is to achieve a dose-response relationship for safety, although data for pain reduction will be obtained as a secondary outcome measure. All subjects will receive RTX. There is no placebo group because of the invasive nature of the injection and the dose escalation performed in the study.

This dose escalation safety study is based upon 4 progressive dose levels (0.8 microg/ganglion; 1.6 microg/ganglion; 3.2 microg/ganglion, and 6.4 microg/ganglion.

The adaptive dose selection design is employed to establish the primary outcome, the MTD for RTX. MTD will be defined as 1 dose level below that at which DLT is observed in more than one-third of the subjects.

The efficacy outcome variable will be evaluated by establishing dose-response curves, with dose plotted on the x-axis and changes in efficacy endpoints (between before and D30, D60, D90 and D180 after treatment) plotted on the y-axis. If, at the end of the study, all dose levels of RTX have equal pain-relieving efficacy, the Data Safety Monitoring Committee (DSMC) may determine it is appropriate to include a dose lower than 0.8 mcg and/or a control group. On the other hand, if the dose-response curves show that higher doses could result in greater pain-relieving efficacy, the protocol may be amended to include higher doses of RTX.

Clinical Study Identifier: NCT02522611

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Recruitment Status: Open


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