Clinical Trial: Resiniferatoxin to Treat Severe Pain Associated With Advanced Cancer

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional

Official Title: A Phase I Study of the Intrathecal Administration of Resiniferatoxin for Treating Severe Refractory Pain Associated With Advanced Cancer

Brief Summary:

This study will examine the safety of giving the experimental drug, resiniferatoxin (RTX), to treat severe pain in patients with advanced cancer. RTX is a chemical extracted from a cactus-like plant. It is similar to capsaicin, the active ingredient in hot pepper. RTX has relieved pain and reduced the need for pain medication in several animal experiments. It works by destroying nerves that transmit pain information.

People at least 18 years of age with severe pain from advanced cancer at or below the level of the chest that cannot be controlled with standard treatments may be eligible for this study. Participants undergo the following procedures:

Pretreatment Visit

Before beginning treatment with RTX, patients give a medical history and undergo a physical examination that includes:

  • Electrocardiogram (EKG)
  • Blood draw
  • Urinalysis
  • Neurological examinations
  • Peak expiratory flow rate (PEFR)
  • Eye examination
  • MRI
  • Urology assessment
  • Pregnancy test, when appropriate

  • Questionnaires to collect information on health, personality, mood, pain levels and symptoms.

    2-Day Hospitalization

Patients are hospitalized for 2 days for RTX injection and monitoring, as follows:

  • Pain continues to be a major problem in patients with advanced cancer. Resiniferatoxin (RTX), a potent member of the family of drugs that includes capsaicin, selectively and irreversibly destroys the neurons (or their axons) transmitting chronic pain sensation. Intrathecal injection of RTX in several animal species has demonstrated a high level of safety, specificity, and efficacy in treating severe pain. This first-in-human, dose-escalation study will investigate the intrathecal administration of RTX in cancer patients with severe pain.

    PRIMARY OBJECTIVE:

    To investigate the safety and efficacy of RTX administered intrathecally in subjects with severe refractory pain associated with advanced cancer.

    STUDY POPULATION:

    Up to 45 subjects will be accrued across several research sites. Eligible subjects will be greater than or equal to 18 years of age, have a clinical and histological diagnosis of advanced malignancy, and have severe pain due to malignancy that is at or below the level of the chest and not adequately relieved by other pain control therapies.

    DESIGN:

    This will be a multi-site, non-randomized, open-label, dose-escalation study using a modified Fibonacci scheme. The starting dose of RTX will be 13 micrograms (mcg). Doses will then be increased in progressively smaller percentage increments. Dose escalation will occur in sequential groups of 3 subjects until 1 escalation above the effective dose in 100% of subjects (ED100), completion of the 166 mirco g dose level, or establishment of the maximum tolerated dose (MTD), whichever occurs first. The total duration of study participation for any subject will be up to 7 mont
    Sponsor: National Institute of Dental and Craniofacial Research (NIDCR)

    Current Primary Outcome: Phase I trial to demonstrate the safety of administering resiniferatoxin (RTX) directly into the human CNS (fluid bathing the spinal cord). [ Time Frame: 6 months from treatment ]

    Original Primary Outcome: Phase I trial to demonstrate the safety of administering resiniferatoxin (RTX) directly into the human CNS (fluid bathing the spinal cord).

    Current Secondary Outcome: Confirm that injection of RTX in CNS has pain-relieving properties (analgesia) resulting in lower pain scores, improvements in quality of life, and possibly opoid sparing properites in patients with refractory pain. [ Time Frame: 6 months from treatment ]

    Original Secondary Outcome: Confirm that injection of RTX in CNS has pain-relieving properties (analgesia) resulting in lower pain scores, improvements in quality of life, and possibly opoid sparing properites in patients with refractory pain.

    Information By: National Institutes of Health Clinical Center (CC)

    Dates:
    Date Received: December 5, 2008
    Date Started: December 3, 2008
    Date Completion: December 30, 2020
    Last Updated: May 20, 2017
    Last Verified: May 9, 2017