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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03226574
Recruitment Status : Recruiting
First Posted : July 24, 2017
Last Update Posted : July 1, 2019
Information provided by (Responsible Party):
Sorrento Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Intractable Cancer Pain Drug: Resiniferatoxin Phase 1

Detailed Description:
All subjects who received RTX will be included in the analyses and summaries of safety, efficacy, PD, and PK assessments.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Intervention Model: Sequential Assignment
Intervention Model Description: 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.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label, Phase 1b Study to Assess the Safety and Define the Maximally Tolerated Dose of Epidural Resiniferatoxin Injection for the Treatment of Intractable Pain Associated With Cancer
Actual Study Start Date : September 1, 2017
Estimated Primary Completion Date : August 2019
Estimated Study Completion Date : November 2019

Arm Intervention/treatment
Experimental: RTX Drug: Resiniferatoxin
single dose (0.4, 1, 2, 4, 8, or 15 mcg), epidural injection
Other Name: RTX

Primary Outcome Measures :
  1. Dose limiting toxicity (DLT) [ Time Frame: 3-month ]
  2. Maximum tolerated dose (MTD) [ Time Frame: 3-month ]

Secondary Outcome Measures :
  1. Numeric Pain Rating Scale (NPRS) [ Time Frame: Day-28 to Day90 daily ]
    Daily average pain and daily worst pain on a 0-10 scale

Other Outcome Measures:
  1. Daily analgesic consumption (DAC) log [ Time Frame: Day-28 to Day90 daily ]
    Consumption of analgesics compared to baseline

  2. Brief Pain Inventory-Short Form (BPI-SF) [ Time Frame: Day-28, Day1, Day2/3, Day8, Day15, Day30, Day60 and Day90 ]
    Quality of life compared to baseline by the total score

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • Sensory/peripheral neuropathy of CTCAE Grade 2 or higher.
  • Nonstudy related minor surgical procedure ≤5 days or major surgical procedure ≤21 days prior to Screening visit.
  • Subjects who have not completely recovered from any toxicities from previous chemotherapy, hormone therapy, immunotherapy, or radiotherapies that are CTCAE Grade 3 or higher.
  • Arterial thrombi (including stroke), myocardial infarction, admission for unstable angina, cardiac angioplasty, or stenting within 3 months prior to Screening visit.
  • Corrected QT using Fridericia's formula (QTcF) prolongation.
  • Evidence or history of bleeding disorder within 4 weeks prior to IP administration.
  • Known HIV or acquired immunodeficiency syndrome-related illness, acute or history of chronic hepatitis B or C.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03226574

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Contact: Laurie Rosenberg, PhD (858) 203-4100 ext 3604

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United States, Florida
University of Miami/Sylvester Comprehensive Cancer Center Recruiting
Miami, Florida, United States, 33136
Contact: Alessandro Colantuno    305-585-7677   
Principal Investigator: Roy C Levitt, MD         
United States, Massachusetts
Brigham & Women's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Vicky Bai    617-732-9437   
Principal Investigator: Srdjan Nedeljkovic, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Magdi Elgasim    919-681-4056   
Principal Investigator: Richard Boortz-Marx, MD         
United States, Ohio
The Ohio State University Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43210
Contact: January Kim    614-366-1943   
Principal Investigator: Tristan Weaver, MD         
United States, Texas
Hermann Drive Surgical Hospital Recruiting
Houston, Texas, United States, 77004
Contact: Lauren Howard    713-367-8548   
Principal Investigator: Daneshvari Solanki, MD         
Sponsors and Collaborators
Sorrento Therapeutics, Inc.
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Study Director: Ken Takeshita, MD Sorrento Therapeutics, Inc.

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Responsible Party: Sorrento Therapeutics, Inc. Identifier: NCT03226574     History of Changes
Other Study ID Numbers: RTX-001
First Posted: July 24, 2017    Key Record Dates
Last Update Posted: July 1, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sorrento Therapeutics, Inc.:
Cancer pain

Additional relevant MeSH terms:
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Cancer Pain
Neurologic Manifestations
Signs and Symptoms