Study to Evaluate Safety and MTD of Epidural Resiniferatoxin Injection for Treatment of Intractable Cancer Pain

NCT03226574 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: RTX-001
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 4

Brief Summary

The study is a multicenter, open-label Phase 1b single dose escalation safety study for adult subjects with intractable pain associated with cancer in any area below the mid-thoracic level who meet all other eligibility criteria.

Conditions

Intractable Cancer Pain

Keywords

Resiniferatoxin; RTX; Cancer pain

Outcome Measures

Primary Outcomes (2)

• Dose limiting toxicity (DLT)

  Grade 3 or 4 Toxicity associated with RTX administration

  3-month

• Maximum tolerated dose (MTD)

  Maximum dose without a Grade 3 or 4 toxicity

  3-month

Secondary Outcomes (1)

• Numeric Pain Rating Scale (NPRS)

  Day-28 to Day90 daily

Other Outcomes (5)

• Daily analgesic consumption (DAC) log

  Day-28 to Day90 daily

• Brief Pain Inventory-Short Form (BPI-SF)

  Day-28, Day1, Day2/3, Day8, Day15, Day30, Day60 and Day90

• Patient Global Impression of Change (PGIC)

  Day2/3, Day8, Day15, Day30, Day60 and Day90

Study Arms (1)

RTX epidural injection

EXPERIMENTAL

Epidural injection of 1.5mL/min RTX under the guidance of epidurogram.

Drug: Resiniferatoxin

Interventions

Resiniferatoxin DRUG

single dose (0.4, 1, 2, 4, 8,15, 25 or 35 mcg), epidural injection

Also known as: RTX

RTX epidural injection

Eligibility Criteria

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

You may qualify if:

• Histologically confirmed advanced cancer or metastasis, which has not responded to standard therapy, producing intractable chronic pain in any area below the mid-thoracic level.
• Male or female subjects must be at least 18 years of age.
• Must have a worst pain score ≥6 on the NPRS at Screening visit.
• Subjects not seeking or receiving potentially curative therapies for cancer. Palliative therapy is acceptable if the therapy started and is stable prior to IP administration.
• Sexually active female subjects of childbearing potential and male subjects capable of fathering a child must be willing to use an effective method of contraception to avoid pregnancies.
• Must be willing and capable of understanding and cooperating with the requirements of the study.
• Must be able to understand and complete study-related forms and adequately communicate with the investigator and/or site staff.
• Must have provided written informed consent prior to participating in any study-related activity.
• Subjects able to complete the study duration.

You may not qualify if:

• Subjects with leptomeningeal metastases in lumbar area.
• Undergoing or have plans to undergo changes to current cancer treatment during the study through the Day15 assessment.
• Had prior lumbar spine surgical procedures that could impair the ability to perform the injection.
• Evidence of brain pathology or increase intracranial pressure.
• Presence of an IT shunt.
• Has evidence or a coagulopathy or hemostasis problem.
• Subjects with a total neutrophil count \<1500 cells/mm3.
• Subjects with serum creatinine ≥1.5 mg/dL.
• Is febrile or has other evidence of an infection within 7 days of planned injection.
• Has an allergy or hypersensitivity to chili peppers, capsaicin, or radiographic contrast agents.
• Female subjects who are pregnant, are planning on becoming pregnant, or are currently breastfeeding.
• Subjects with any medical condition that could adversely impact study participation or assessments.
• Subjects who have received new anti-cancer treatments and there is less than one week or four half-lives of the investigational drug, whichever is greater, between the last dose of the new drug and the planned day of IP administration; or had a change in the dose or schedule of the anti-cancer treatments within one week or four half-lives, whichever is greater, between the last dose of the anti-cancer treatment and the planned day of IP administration; or are scheduled to receive a new anti-cancer therapy or investigational product prior to completion of the Day 15 visit.
• Subjects with additional loci of pain above the mid-thoracic level or other pain disorder due to non-cancer etiology, unless both the investigator and the subject are clearly able to distinguish the additional pain from the target pain due to cancer.
• Liver cirrhosis or severe hepatic impairment, with liver function test 3 times above ULN.

Sponsors & Collaborators

• Sorrento Therapeutics, Inc.: lead

Study Sites (4)

University of Miami/Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

Location

Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Hermann Drive Surgical Hospital

Houston, Texas, 77004, United States

Location

MeSH Terms

Conditions

Cancer Pain

Interventions

resiniferatoxin

Condition Hierarchy (Ancestors)

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

Study Officials

• Monica Luchi, MD

  Sorrento Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The design uses a traditional "3+3" rule-based design that does not stipulate any prior assumption of the dose-toxicity curve. Dose escalation is permitted between successive cohorts based upon a specified algorithm, using discrete dosage steps.

Study Record Dates

• First Submitted: July 6, 2017
• First Posted: July 24, 2017
• Study Start: September 1, 2017
• Primary Completion: March 18, 2020
• Study Completion: June 18, 2020
• Last Updated: October 20, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)