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

Sponsor

Sorrento Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05067257

Collaborator

(none)

120

Enrollment

Location

Arms

Anticipated Duration (Months)

7.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Pain, Intractable Pain Cancer Pain	Drug: Resiniferatoxin Drug: Placebo	Phase 2

Detailed Description

This is a global multicenter, randomized Phase 2 study for subjects with advanced cancer experiencing intractable pain to assess the safety and efficacy of a single epidural injection of Resiniferatoxin versus vehicle control. Additional subjects who elect to not enroll but are willing to be followed will comprise a Concurrent Control Group. Subjects will be followed for twelve months.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

120 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

A Multicenter, Phase 2 Study to Assess the Safety and Efficacy of Epidural Resiniferatoxin for the Treatment of Intractable Pain Associated With Advanced Cancer

Anticipated Study Start Date :

Aug 1, 2022

Anticipated Primary Completion Date :

Jun 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Resiniferatoxin 15 mcg, 20 mcg, or 25 mcg in 2mL injected once into the epidural space	Drug: Resiniferatoxin Resiniferatoxin is a compound purified from natural sources Other Names: RTX
Placebo Comparator: Placebo 2mL injected once into the epidural space	Drug: Placebo Vehicle solution
No Intervention: Concurrent Control No intervention

Arm

Intervention/Treatment

Experimental: Resiniferatoxin

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

Drug: Resiniferatoxin

Resiniferatoxin is a compound purified from natural sources

Other Names:

RTX

Placebo Comparator: Placebo

2mL injected once into the epidural space

Drug: Placebo

Vehicle solution

No Intervention: Concurrent Control

No intervention

Outcome Measures

Primary Outcome Measures

Assess the incidence of any treatment-emergent adverse events of epidural Resiniferatoxin (RTX) [safety and tolerability] [Baseline through study completion at up to approximately 12 months]
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

Secondary Outcome Measures

Assess efficacy of RTX on pain associated with advanced cancer [Baseline through study completion at up to approximately 12 months]
To assess efficacy of each epidural RTX dose in comparison to control groups on the subject's intractable pain due to advanced cancer using the Brief Pain Inventory, Short Form (BPI-SF) score (1-10, with 10 associated with worse pain)
Assess RTX effect on quality of life [Baseline through study completion at up to approximately 12 months]
To assess the efficacy of each epidural RTX dose in comparison to control groups on the quality of life using Treatment Helpfulness Questionnaire score (graded 1-7, with 7 being most helpful)
Assess RTX effect on opioid consumption [Baseline through study completion at up to approximately 12 months]
To assess the effect of each epidural RTX dose on opioid consumption, measuring the average reduction compared to baseline of daily opioid consumption, calculated as an oral morphine equivalent dose

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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

Exclusion Criteria:

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
Non-study related minor surgical procedure ≤ 5 days or major surgical procedure ≤ 21 days prior to enrollment. In all cases, subjects must be sufficiently recovered and stable prior to IP administration on D1
Arterial thrombi, myocardial infarction, admission for unstable angina, within 3 months prior to screening
Clinically significant electrocardiogram abnormalities
Have any medical condition that could adversely impact subject's participation or safety or interfere with pain assessments
Participation in another investigational trial during the study