Efficacy and Safety of Cannabidiol for Gastroparesis and Functional Dyspepsia

Mayo Clinic

Status and phase: Completed; Phase 2

Conditions: Gastroparesis; Dyspepsia

Treatments: Drug: Cannabidiol; Other: placebo

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT03941288

R01DK122280 (U.S. NIH Grant/Contract)

19-002483

Details and patient eligibility

About

Researchers are looking at the effects of a cannabidiol medication on stomach function in people with gastroparesis (a paralyzed stomach) and people with dyspepsia (an upset stomach caused by improper functioning of the stomach's muscles or nerves).

Enrollment: 92 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients with gastroparesis or functional dyspepsia (FD)

• Age 18-70 years

• Patients will be identified from among Mayo Clinic patients.

• Patients will have symptoms consistent with gastroparesis based on a national guideline for gastroparesis (symptoms plus delayed gastric emptying of solids). Patients with Rome IV criteria for postprandial distress syndrome (a subset of functional dyspepsia) will be selected based on gastric emptying of solids which is NOT delayed, in addition to standard FD criteria:

  • Symptoms fulfilled for the last 3 months with onset greater than 6 months before diagnosis:
  • One or more symptoms being bothersome: postprandial fullness, early satiation, epigastric pain or burning
  • Must include one or both of the following at least 3 days per week: bothersome postprandial fullness (i.e., severe enough to impact on usual activities) or bothersome early satiation (i.e., severe enough to prevent finishing a regular-size meal)
  • No evidence of organic, systemic, or metabolic disease to explain the symptoms on routine investigations.
  • Participants will have previously undergone test of gastric emptying of solids using the standardized Mayo Clinic scintigraphic method

• Ability to provide informed consent

• Absence of other diseases (structural or metabolic) which could interfere with interpretation of the study results

• Body mass index of 18-35 kg/m2

• Several medication classes, particularly those affecting gastrointestinal transit or motor functions, will be excluded, including GLP-1 receptor or amylin agonists in patients with diabetes mellitus. Stable doses of thyroid replacement, estrogen replacement, low-dose aspirin for cardioprotection, and birth control (but with adequate backup contraception, as drug interactions with birth control have not been conducted for secretin PAM) are

Exclusion criteria

• Patients with current H. pylori infection will be excluded.
• Pregnancy or lactation
• Rapid metabolizers for CYP3A4 or CYP2C19 [estimated prevalence of 17% and 18% respectively
• based on literature review will be excluded since this could impact assessment of effects of cannabidiol
• Patients with abnormal baseline liver transaminases (any value above UNL), since up to 3-fold, dose-related elevations of liver transaminases (ALT and/or AST) occur in 13% of treated patients (vs. 1% placebo);
• Hypersensitivity to cannabidiol or any of the ingredients in EPIDIOLEX
• Concomitant use of valproate, central nervous system (CNS) depressants and alcohol, other hepatotoxic drugs

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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92 participants in 2 patient groups, including a placebo group

Pharmacodynamics and clinical effects of cannabidiol

Active Comparator group

Description:

Cannabidiol will be administered orally twice daily in equally divided doses starting at 2.5mg/kg per day and increasing by 2.5 to 5.0mg/kg every other day until the target dose of 20mg/kg is reached. Cannabidiol and the matching placebo solution (excipients alone) will be provided in identical 100ml amber glass bottles. At the end of the treatment period, the treatment solutions will be tapered (10% volume each day) over 10 days.

Treatment:

Drug: Cannabidiol

pharmacodynamics and clinical effects of placebo

Placebo Comparator group

Description:

Placebo will be administered orally twice daily in equally divided doses starting at 2.5mg/kg per day and increasing by 2.5 to 5.0mg/kg every other day until the target dose of 20mg/kg is reached. Cannabidiol and the matching placebo solution (excipients alone) will be provided in identical 100ml amber glass bottles. At the end of the treatment period, the treatment solutions will be tapered (10% volume each day) over 10 days.

Treatment:

Other: placebo