Study to Assess Epidural Resiniferatoxin for the Treatment of Intractable Pain Associated With Advanced Cancer

NCT05067257 | Suspended Phase 2 FDA Drug

• 1 other identifier: RTX-CAP-201
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 4

Brief Summary

This Phase 2 study assesses the safety and efficacy of a single injection of Resiniferatoxin versus placebo for the treatment of intractable advanced cancer pain.

Conditions

Pain, Intractable; Pain Cancer; Pain

Keywords

advanced cancer pain; cancer pain; pain; resiniferatoxin; RTX

Outcome Measures

Primary Outcomes (1)

• Assess the incidence of any treatment-emergent adverse events of epidural Resiniferatoxin (RTX) [safety and tolerability]

  To assess the safety of epidural RTX including incidence and severity of any short-term and long-term treatment-emergent adverse events (TEAEs) using the Common Terminology Criteria for Adverse Events (CTCAE) criteria

  Baseline through study completion at up to approximately 12 months

Secondary Outcomes (3)

• Assess efficacy of RTX on pain associated with advanced cancer

  Baseline through study completion at up to approximately 12 months

• Assess RTX effect on quality of life

  Baseline through study completion at up to approximately 12 months

• Assess RTX effect on opioid consumption

  Baseline through study completion at up to approximately 12 months

Study Arms (3)

Resiniferatoxin

EXPERIMENTAL

15 mcg, 20 mcg, or 25 mcg in 2mL injected once into the epidural space

Drug: Resiniferatoxin

Placebo

PLACEBO COMPARATOR

2mL injected once into the epidural space

Drug: Placebo

Concurrent Control

NO INTERVENTION

No intervention

Interventions

Resiniferatoxin DRUG

Resiniferatoxin is a compound purified from natural sources

Also known as: RTX

Resiniferatoxin

Placebo DRUG

Vehicle solution

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically or cytologically confirmed advanced cancer
• Targeted pain area of involvement that is at or below the lower thoracic or chest level down to lower extremities, attributed to the cancer
• If pain is present in other areas, able to differentiate pain in the target area is the primary source of pain
• Intractable pain that has not responded to standard therapies
• Be opioid tolerant, defined as an average daily opioid consumption \> 30 mg oral morphine equivalent dose during the screening and which has been stable for the month prior to screening
• Have a Karnofsky Performance Scale score ≥ 50 at Screening
• In the Investigator's opinion, a reasonable expectation that the subject will be able to complete the study
• Able to comply with the study procedures and give informed consent
• Willing to follow contraception guidelines

You may not qualify if:

• Be undergoing or have plans to undergo changes to current cancer treatment from D-7 through M3
• Had an implantation of intrathecal pump or spinal cord stimulator less than 6 weeks prior to D1 or planning to undergo such a placement during the study prior to M3
• Have leptomeningeal metastases in the lumbar area
• Unless approachable via the caudal route, have the level of intended epidural injection within site of prior lumbar spine surgical procedures that could disrupt the epidural space or otherwise impair ability of the injection to reach nerves
• Has evidence of a non-correctable coagulopathy or hemostasis problem including a low platelet count, prothrombin time, abnormal PT or PTT, or on anticoagulant or antiplatelet therapies before and during investigational product (IP) administration
• Have evidence or history of bleeding disorder or disseminated intravascular coagulation, any recent hemorrhage or bleeding event within 4 weeks prior to D1
• Have abnormal neutrophil or serum creatinine
• Is febrile or has other evidence of infection within 24 hours of D1
• Has recently been diagnosed as COVID-19 positive or evidence of active infection. The subject may participate if full recovery has occurred with a negative RT-PCR test (any EUA cleared test) at least 1 week prior to D1
• Has an allergy or hypersensitivity to TRPV1 agonists, bupivacaine, radiographic contrast agents, fentanyl, hydromorphone, or morphine
• Pregnant at Screening or planning on becoming pregnant or currently breastfeeding
• Has an intrathecal shunt, increased intracranial pressure or evidence of brain pathology as determined by symptoms, history, physical examination, and/or magnetic resonance imaging (MRI)
• Is unable or distinguish the target pain from any additional loci of pain at screening
• Non-study related minor surgical procedure ≤ 2 days or major surgical procedure ≤ 7 days prior to screening and must be sufficiently recovered and stable prior to D1
• Has not recovered from toxicities from previous cancer treatment, including chemotherapy, hormone therapy, immunotherapy, radiotherapy or bisphosphonates. Participants are not eligible if they have received such therapy within the month prior to D1

Sponsors & Collaborators

• Sorrento Therapeutics, Inc.: lead

Study Sites (4)

Altman Clinical and Translational Research Institute (ACTRI)

La Jolla, California, 92037, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

HD Research

Bellaire, Texas, 77401, United States

Location

MeSH Terms

Conditions

Pain, Intractable; Cancer Pain; Pain

Interventions

resiniferatoxin

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Mike Royal, MD

  Sorrento Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2021
• First Posted: October 5, 2021
• Study Start: September 1, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: March 18, 2024

Record last verified: 2024-03

Locations

US (4)