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Cannabidiol in Sickle Cell Disease

Cannabidiol in Sickle Cell Disease

Recruiting
18 years and older
All
Phase 1/2

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Overview

Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

Description

The researchers will conduct a randomized, double blind, placebo-controlled, study of cannabidiol in an oral formulation. Participants will be enrolled when they are not in pain crisis and have demonstrated a urine toxicology test free from cannabinoids in the past 30 days. The sample size will be 52 participants, aged ≥18, with 1:1:1:1 allocation of placebo to 3 drug doses. This is a dose finding study with a primary outcome of reduction of inflammatory cytokine TNFα.

Eligibility

Inclusion Criteria:

  • Age >18 years
  • Clinical diagnosis of SCD (HbSS, HbSC, HbSβ+; Thal, HbSβ0Thal, HbS variants)
  • Baseline score of 60 or lower on the ASCQ-Me 7-day pain interference domain
  • If on a SCD modifying therapy (hydroxyurea, regular blood transfusions, L-glutamine, voxelotor, crizanlizumab), on stable dose for at least 3 months
  • If using opioids for pain at home, on stable dose for at least 3 months
  • One urine toxicology negative for cannabinoids within 30 days of randomization
  • Willing to abstain from cannabis, medical and illicit, during study weeks 1 through 4 • Not pregnant or nursing
  • If a woman capable of becoming pregnant, willing to use a medically accepted form of birth control for the duration of study participation. Accepted forms include oral contraception, medroxyprogesterone, contraceptive implants or patch, surgical sterilization, total abstinence.
  • Able to consent for research

Exclusion Criteria:

  • No known intolerance to cannabinoids
  • No history of psychotic episode, psychosis, or active suicidality
  • No contraindication to epidiolex with attention to potential side effects, concurrent medications/substances, and concurrent medical problems, as evaluated by a physician
  • Not a daily cannabis user
  • No diagnosis of active substance use disorder
  • No ALT>3 times the upper limit of normal

Study details
    Sickle Cell Disease

NCT06930703

Icahn School of Medicine at Mount Sinai

15 October 2025

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FAQs

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A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

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