Epidiolex (CBD) in Patients With Biochemically Recurrent Prostate Cancer

NCT04428203 | Completed Phase 1 Results FDA Drug

• 1 other identifier: MCC-20-GU-74-PMC
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this phase I/Ib study is to determine the safety profile of Epidiolex (CBD oil) in biochemically recurrent prostate cancer patients. The study consists of a dose escalation part and dose expansion part. The dose expansion part of the study will use the maximum tolerated dose (MTD) determined in the dose escalation part to assess the activity, safety and tolerability of the investigational product in patients with biochemically recurrent prostate cancer after localized therapy with either surgery or radiation.

Conditions

Prostate Cancer Recurrent; Prostate Cancer; Prostate Adenocarcinoma

Keywords

Prostate; Rising PSA; CBD Oil; Epidiolex

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Dose-limiting Toxicities (Treatment-related Adverse Events) as Assessed by the CTCAE v5.0

  DLT was defined as grade ≥3 nausea, vomiting, diarrhea that persists \>72 h despite optimal anti-emetics and anti-diarrhea treatment, grade ≥3 hematological adverse events (AEs), or grade ≥2 suicidal ideation.Treatment-related adverse events are those that comprise a dose-limiting toxicity within 30 days after initiation of Epidiolex (i.e., acute DLT). Additionally, Treatment-related adverse events will continue to be monitored for a total of 90 days.

  up to 90 days

Secondary Outcomes (7)

• Participants With Biochemical Response.

  within 90 days

• Change in PSA Velocity From Baseline Throughout the Treatment Period as an Indication of Biochemical Response.

  within 90 days

• Change in Testosterone Levels From Baseline Throughout the Treatment Period as an Indication of Biochemical Response

  Baseline, Day 1 of Cycle 1 (each cycle is 4 weeks), Day 1 of Cycle 2, Day 1 of Cycle 3, and 1 month post treatment (up to 16 weeks)

• Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)

  Baseline

• Health-related Quality of Life (EORTC Quality of Life Questionnaire-C30)

  12 weeks

Study Arms (3)

Dose Escalation: Epidiolex 600 mg

EXPERIMENTAL

Cohort 1 participants with rising PSA after failure of localized therapy will receive 600 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex

Drug: Epidiolex Oral Liquid Product

Dose Escalation: Epidiolex 800 mg

EXPERIMENTAL

Cohort 2 participants with rising PSA after failure of localized therapy will receive 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolex

Drug: Epidiolex Oral Liquid Product

Dose Expansion: Epidiolex 800 mg

EXPERIMENTAL

Expansion Cohort participants with rising PSA after failure of localized therapy will receive the MTD of 800 mg oral solution Epidiolex once daily for a total of 90 days in the absence of disease progression or unacceptable toxicity followed by a 10-day taper with 30 days of follow up after the discontinuation (last dose) of Epidiolexdose

Drug: Epidiolex Oral Liquid Product

Interventions

Epidiolex Oral Liquid Product DRUG

600 mg Oral solution

Also known as: CBD oil

Dose Escalation: Epidiolex 600 mg

Eligibility Criteria

• Age: 18 Years+
• Gender Eligibility Details: prostate cancer only occurs in male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Completion of localized therapy (prostatectomy or radiotherapy) for prostate adenocarcinoma (either histologically or cytologically confirmed)
• Biochemical (PSA) recurrence, defined as: \* PSA of \>= 0.2 ng/ml that has increased above nadir following radical prostatectomy OR \* PSA increase of 2.0 ng/ml above post-therapy nadir after radiotherapy NOTE: PSA measured at two consecutive time points (separated by 4 or more weeks) is required in order to demonstrate the requisite increase in PSA
• Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
• Absolute neutrophil count \>= 1,500/microliters (at baseline \[pre-study\])
• Platelets \>= 80,000/microliters (at baseline \[pre-study\])
• Total bilirubin =\< institutional upper limit of normal (at baseline \[pre-study\])
• Aspartate aminotransferase (AST)(serum glutamic-oxaloacetic transaminase/alanine aminotransferase (ALT)(serum glutamate pyruvate transaminase) =\< institutional upper limit of normal (at baseline \[pre-study\])
• Glomerular filtration rate (GFR) \>= 30 mL/min/1.73 m\^2 using the Cockcroft-Gault formula (at baseline \[pre-study\])
• Patients with a prior or concurrent malignancy (non-prostate) whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen as determined by the treating physician are eligible
• Given that worsening of an underlying state of mental depression or suicidal ideation has been reported with Epidiolex, patients should be carefully screened for depression at baseline and if there are indications or a history of depression it is strongly recommended that these patients be closely followed together with behavioral health or psychiatric medical support. Patients with an established diagnosis of depression that, in the assessment of the investigator may make the administration of Epidiolex hazardous, should not be enrolled on this protocol
• Concurrent use of over-the-counter CBD oil, Marinol or marijuana is not permitted. Patients with a history of current over-the-counter CBD oil, Marinol or marijuana use for any reason are eligible only if they do the following: \* Complete a one-week washout period prior to study initiation \* Refrain from non-study related CBD oil, Marinol or marijuana use while on-study
• Ability to understand and the willingness to sign a written informed consent document

You may not qualify if:

• History of hypersensitivity to Epidiolex (cannabidiol) or sesame seeds (one of the inactive ingredients in Epidiolex)
• Any radiological evidence of metastatic disease (determined by standard of care computed tomography \[CT\] scans of abdomen. pelvis, chest, whole body bone scan or Axium positron emission tomography scan). Questionable lesions on bone scan will be confirmed by standard of care methods such as plain X-rays or Axium positron emission tomography scan, if not previously performed
• Receipt of prior cytotoxic chemotherapy for recurrent prostate cancer
• Use of androgen deprivation therapy (for example, bicalutamide, flutamide, nilutamide, or leuprolide acetate) concurrently or within the previous 3 months.
• Uncontrolled intercurrent illness such as active infections. Other illnesses will be evaluated and eligibility status determined at the discretion of the treating physician and the investigator
• Psychiatric illness/social situations that would limit compliance with study requirements
• Concomitant use of valproate or clobazam
• Concurrent use of over-the-counter CBD oil, Marinol or marijuana
• Epidiolex is a moderate inhibitor of CYP2C19 and a moderate/strong inhibitor of CYP3A4, therefore concurrent use of CYP2C19 substrates is not allowed

Sponsors & Collaborators

• Zin W Myint: lead

Study Sites (1)

Markey Cancer Center

Lexington, Kentucky, 40536, United States

Location

MeSH Terms

Conditions

Prostatic Neoplasms

Condition Hierarchy (Ancestors)

Genital Neoplasms, Male; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Genital Diseases, Male; Genital Diseases; Urogenital Diseases; Prostatic Diseases; Male Urogenital Diseases

Results Point of Contact

• Title: Dr. Zin Myint
• Organization: University of Kentucky

zin.myint@uky.edu

8593233964

Study Officials

• Zin W. Myint, MD

  University of Kentucky

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor of Medicine

Study Record Dates

• First Submitted: June 6, 2020
• First Posted: June 11, 2020
• Study Start: August 3, 2020
• Primary Completion: August 20, 2021
• Study Completion: August 20, 2021
• Last Updated: March 5, 2025
• Results First Posted: March 5, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)