Cannabidiol in Sickle Cell Disease

NCT06930703 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: STUDY-24-00903IN123001222
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Sickle Cell Disease

Keywords

chronic pain; epidiolex; cannabidiol

Outcome Measures

Primary Outcomes (1)

• Tumor Necrosis Factor-alpha level

  Plasma levels of tumor necrosis factor-alpha, a marker of inflammation in sickle cell disease will be collected.

  at 4 weeks

Secondary Outcomes (16)

• Levels of Markers of Inflammation

  at 4 weeks

• White Blood Cell with differential

  at 4 weeks

• C-reactive protein level

  at 4 weeks

• Tryptase levels

  at 4 weeks

• Substance P levels

  at 4 weeks

Study Arms (4)

Placebo

PLACEBO COMPARATOR

Twice daily matching placebo

Drug: Placebo

Cannabidiol 200 mg

ACTIVE COMPARATOR

Twice daily 200 mg cannabidiol

Drug: Cannabidiol

Cannabidiol 400 mg

ACTIVE COMPARATOR

Twice daily 400 mg cannabidiol

Drug: Cannabidiol

Cannabidiol 600 mg

ACTIVE COMPARATOR

Twice daily 600 mg cannabidiol

Drug: Cannabidiol

Interventions

Cannabidiol DRUG

Cannabidiol (CBD) twice daily taken orally for 4-weeks

Also known as: Epidiolex

Cannabidiol 200 mg; Cannabidiol 400 mg; Cannabidiol 600 mg

Placebo DRUG

Placebo equivalent twice daily taken orally for 4-weeks

Placebo

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead

Study Sites (1)

Icahn School of Medicine at Mount Sinai

Manhattan, New York, 10029, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell; Chronic Pain

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Susanna Curtis

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Susanna Curtis

CONTACT

212-241-3650; susanna.curtis@mssm.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Model Details: Twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, for 4 weeks, with 1:1:1:1 allocation of placebo to 3 drug doses.

Study Record Dates

• First Submitted: April 9, 2025
• First Posted: April 16, 2025
• Study Start: April 3, 2025
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027
• Last Updated: June 25, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: Specify Other Time FrameOn request.
• Access Criteria: Researchers who provide a methodologically sound proposal. Any purpose. Proposals should be directed to Susanna.curtis@mssm.edu. To gain access, data requestors will need to sign a data access agreement. Data are available for 5 years at a third party website (Link TBD).

Locations

US (1)