Cannabidiol for Sickle Cell Disease

(SPICE Trial)

Overseen By Susanna Curtis

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Icahn School of Medicine at Mount Sinai

Must be taking: SCD modifying therapy

Must not be taking: Cannabinoids

Disqualifiers: Psychosis, Substance use disorder, others

Compensation: Varies

Number of Visits: 7

Duration: 4 weeks

52 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effects of cannabidiol (CBD) on individuals with sickle cell disease (SCD). Researchers aim to determine if taking CBD twice daily at varying doses can help manage SCD symptoms over four weeks. Participants will be randomly assigned to receive either a CBD dose (200mg, 400mg, or 600mg) or a placebo. Individuals with SCD who experience pain affecting daily life and are not regular cannabis users might be suitable for this trial. As a Phase 1, Phase 2 trial, this research seeks to understand how CBD functions in people and measure its effectiveness in managing SCD symptoms.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on a sickle cell disease modifying therapy or using opioids for pain, you must be on a stable dose for at least 3 months before joining the study.

Is there any evidence suggesting that cannabidiol is likely to be safe for humans?

Studies have shown that cannabidiol (CBD) is generally safe for most people. However, some reports mention possible side effects, such as changes in liver function and increased drowsiness, especially when combined with other treatments.

No evidence indicates that CBD worsens sickle cell disease symptoms. In fact, some research suggests it might help manage pain related to the condition. While there are some safety concerns, these mainly involve using CBD with other medications.

As the trial is in its early stages, the main focus is on assessing safety and determining the right dose. Researchers are closely monitoring for side effects to ensure it is safe for further testing. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sickle cell disease?

Researchers are excited about cannabidiol for sickle cell disease because it offers a novel approach by leveraging the potential therapeutic effects of CBD, a compound derived from cannabis. Unlike standard treatments, which often focus on managing symptoms and complications with medications like hydroxyurea or blood transfusions, cannabidiol may provide anti-inflammatory and pain-relieving properties, addressing some of the root causes of discomfort in sickle cell patients. The use of cannabidiol as an active ingredient opens new possibilities for pain management and improved quality of life without the psychoactive effects associated with THC, another cannabis compound. This could be a game-changer for individuals who struggle with the chronic pain and inflammation typical of sickle cell disease.

What evidence suggests that cannabidiol might be an effective treatment for sickle cell disease?

Studies have shown that cannabidiol (CBD) might help alleviate pain in people with sickle cell disease (SCD). Research suggests that CBD eases pain by calming certain cells and reducing swelling. In animal studies, CBD lessened pain without worsening symptoms. Other research has indicated that CBD might lower levels of a protein that causes swelling. While more studies in humans are needed, these findings suggest CBD could be a promising option for managing pain in SCD. This trial will evaluate different dosages of CBD—200 mg, 400 mg, and 600 mg—to determine its effectiveness and safety in managing pain for individuals with SCD. ^{2 3 5 6 7}

Who Is on the Research Team?

Susanna Curtis

Principal Investigator

Icahn School of Medicine at Mount Sinai

Are You a Good Fit for This Trial?

Adults over 18 with Sickle Cell Disease who can consent to research, have low pain interference scores, and don't use cannabis. They must not be pregnant or nursing and agree to birth control if applicable. Stable doses of opioids for pain or SCD therapies are required.

Inclusion Criteria

Able to consent for research

Baseline score of 60 or lower on the ASCQ-Me 7-day pain interference domain

One urine toxicology negative for cannabinoids within 30 days of randomization

Exclusion Criteria

No known intolerance to cannabinoids

No history of psychotic episode, psychosis, or active suicidality

Not a daily cannabis user

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive twice daily cannabidiol at 3 dose levels or placebo for 4 weeks

4 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Cannabidiol

Trial Overview

The trial is testing the effects of cannabidiol (CBD) at different doses (200mg, 400mg, and 600mg) versus a placebo in managing symptoms of Sickle Cell Disease over four weeks. It's randomized and double-masked so neither participants nor researchers know who gets CBD or placebo.

How Is the Trial Designed?

Group I: Cannabidiol 200 mg Active Control 1 Intervention

Twice daily 200 mg cannabidiol

Group II: Cannabidiol 400 mg Active Control 1 Intervention

Twice daily 400 mg cannabidiol

Group III: Cannabidiol 600 mg Active Control 1 Intervention

Twice daily 600 mg cannabidiol

Group IV: Placebo Placebo Group 1 Intervention

Twice daily matching placebo

Who Is Running the Clinical Trial?

Icahn School of Medicine at Mount Sinai

Lead Sponsor

Trials

933

Recruited

579,000+

Published Research Related to This Trial

A systematic review of 12 clinical trials involving 803 participants found that cannabidiol (CBD) is associated with a higher likelihood of withdrawal due to adverse effects compared to placebo, particularly in studies related to childhood epilepsy.

While CBD generally appears well tolerated, significant adverse effects such as abnormal liver function tests and sedation were noted, especially in combination with other medications, highlighting the need for careful monitoring of drug interactions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse effects of cannabidiol: a systematic review and meta-analysis of randomized clinical trials. Chesney, E., Oliver, D., Green, A., et al.[2021]

A systematic review of 4186 studies on cannabidiol (CBD) revealed that most research focuses on neurological outcomes, with significant adverse events reported in this area, highlighting the need for careful assessment of CBD's safety.

There is a notable gap in research regarding the reproductive and developmental toxicity of CBD, suggesting that future studies should prioritize these areas to establish safe intake levels for consumers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol Safety Data: A Systematic Mapping Study. Henderson, RG., Franke, KS., Payne, LE., et al.[2023]

In a study involving 40 children with drug-resistant epilepsy, cannabidiol was found to have a manageable safety profile, with the most common side effect being somnolence, which resolved in most cases.

Caregivers reported subjective improvements in their children's overall health, although the study's open-label design means that it's unclear if these benefits were directly due to cannabidiol treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol for treating drug-resistant epilepsy in children: the New South Wales experience. Chen, KA., Farrar, M., Cardamone, M., et al.[2020]

Serious suspected adverse reactions (SARs) to unlicensed cannabidiol (CBD) products were found to be 18.9% of all adverse events, with a higher frequency in men and adults, indicating a need for careful monitoring of its use.

The most common adverse effects associated with unlicensed CBD included mental disorders, hepatic disorders, and worsening of pre-existing epilepsy, particularly in patients also taking antiepileptic medications like clobazam and valproic acid.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacovigilance of unlicensed cannabidiol in European countries. Calapai, F., Esposito, E., Ammendolia, I., et al.[2023]

Nearly 50% of CBD users experienced adverse drug events (ADEs), with common issues including elevated liver enzymes, sedation, and sleep disturbances, indicating a significant safety concern.

CBD has a high potential for drug-drug interactions (DDIs) due to its effects on drug metabolism, particularly with enzymes like CYP3A4 and CYP2C19, suggesting that careful monitoring and dose adjustments may be necessary for patients on multiple medications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Potential Adverse Drug Events and Drug-Drug Interactions with Medical and Consumer Cannabidiol (CBD) Use. Brown, JD., Winterstein, AG.[2020]

In a study of 50 patients with multiple sclerosis, the combination of THC and CBD was effective in reducing spasticity in 80% of cases, suggesting it could be a beneficial alternative treatment for this symptom.

The treatment had an acceptable safety profile, with adverse effects reported in a minority of patients, including dizziness and somnolence, indicating that while some side effects occurred, they were manageable and did not outweigh the benefits for most patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical experiences with cannabinoids in spasticity management in multiple sclerosis. Lorente Fernández, L., Monte Boquet, E., Pérez-Miralles, F., et al.[2022]

In a randomized placebo-controlled trial involving 20 patients with Crohn's disease, cannabidiol (CBD) was found to be safe with no observed side effects, but it did not show any significant beneficial effects on disease activity after 8 weeks of treatment.

Despite the lack of efficacy, the study suggests that the ineffectiveness of CBD could be due to factors such as the small dosage used, the limited number of participants, or the need for a combination with other cannabinoids, indicating that further research is needed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low-Dose Cannabidiol Is Safe but Not Effective in the Treatment for Crohn's Disease, a Randomized Controlled Trial. Naftali, T., Mechulam, R., Marii, A., et al.[2022]

Citations

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Is CBD ready for prime time in sickle cell disease? - PMC

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NCT06930703 | Cannabidiol in Sickle Cell Disease

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sciencedirect.com

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Cannabis Use for Chronic Pain in Sickle Cell Disease

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Therefore, CBD may be effective in treating sickle cell pain, and our data support examining the analgesic effectiveness of Food and Drug ...

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withpower.com

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Cannabidiol for Sickle Cell Disease (SPICE Trial)

While CBD generally appears well tolerated, significant adverse effects such as abnormal liver function tests and sedation were noted, especially in combination ...