Nabiximols for Neuromyelitis Optica Spectrum Disorder

(SENS-NMO Trial)

Overseen By Rebecca Salky

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Michael, Levy M.D.,Ph.D.

Must be taking: Anti-spasticity medications

Must not be taking: Cannabinoids, CYP3A4 inhibitors, others

Disqualifiers: Cannabis use, Schizophrenia, Heart disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

44 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you maintain a stable dose of any non-study-related anti-spasticity medication you are currently taking. However, you must stop taking certain medications like some antibiotics, antifungals, and other specific drugs listed in the exclusion criteria for the duration of the study.

How is the drug Nabiximols different from other drugs for neuromyelitis optica spectrum disorder?

Nabiximols is unique because it is derived from cannabis and contains both THC (tetrahydrocannabinol) and CBD (cannabidiol), which may offer a different mechanism of action compared to traditional immunosuppressants used for neuromyelitis optica spectrum disorder. Unlike other treatments that target specific immune cells or antibodies, Nabiximols may work by modulating the endocannabinoid system, potentially providing symptom relief through a novel pathway. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial tests a cannabinoid spray called nabiximols to help adults with a condition affecting the nervous system who have severe muscle stiffness. These patients often don't respond well to other treatments. The spray works by relaxing muscles through the body's natural system that controls movement and pain. Nabiximols, also known as Sativex, is a cannabis plant extract approved in several countries for various uses including muscle stiffness related to multiple sclerosis and as additional pain relief in advanced cancer.

Research Team

Michael Levy, MD, PhD

Principal Investigator

Massachusetts General Hospital

Anastasia Vishnevetsky, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

Adults diagnosed with Neuromyelitis Optica Spectrum Disorders (NMOSD) who experience moderate to severe spasticity for at least 6 months can join. They must be stable on current medications, not planning pregnancy, and able to use electronic devices for remote study participation. Exclusions include certain psychiatric conditions, substance abuse history, significant heart or liver issues, recent cannabis use, and known allergies to trial medication components.

Inclusion Criteria

I agree to use contraception or remain abstinent for 3 months after my last dose.

My condition has been stable with no relapses for at least 6 months.

Able to use the necessary electronic applications (either via smartphone, tablet, or desktop) and has an email address

Exclusion Criteria

I do not have severe kidney or liver problems.

My immediate family has a history of schizophrenia or similar conditions.

Intention to travel internationally during the study

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

1 visit (virtual)

Treatment Period 1

Participants receive either nabiximols or placebo spray with a 2-week dose escalation followed by a 4-week stable treatment period

6 weeks

4 visits (virtual)

Washout

Participants undergo a 2-week washout period between treatment periods

2 weeks

Treatment Period 2

Participants receive the alternate treatment (nabiximols or placebo) with a 2-week dose escalation followed by a 4-week stable treatment period

6 weeks

4 visits (virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (virtual)

Treatment Details

Interventions

• Nabiximols

Trial Overview The trial is testing nabiximols (a cannabinoid spray) against a placebo in treating spasticity from NMOSD. It aims to see if nabiximols improve symptoms like muscle stiffness, pain frequency of spasms, mood swings walking ability and sleep quality compared to a dummy spray. Participants will receive treatments by mail and complete questionnaires remotely.

Participant Groups

Group I: Placebo, then Nabiximols Experimental Treatment 2 Interventions

During Period 1, participants receive daily matched placebo spray, delivered by a pump action oromucosal spray. The first 2 weeks of Period 1 are the titration period with participants following pre-specified uptitration schedule, until they reach their individualized optimum daily dosage, with a maximum of 12 daily sprays. Following these 2 weeks, they continue the optimum dose for 4 weeks. Then, they undergo a 2-week washout period, and then, enter Period 2 where they receive the active nabiximols spray and again undergo a 2-week titration period, and a 4-week consistent daily dosage period.

Group II: Nabiximols, then Placebo Experimental Treatment 2 Interventions

During Period 1, participants receive daily nabiximols spray, delivered by a pump action oromucosal spray. The first 2 weeks of Period 1 are the titration period with participants following pre-specified uptitration schedule, until they reach their individualized optimum daily dosage, with a maximum of 12 daily sprays. Following these 2 weeks, they continue the optimum dose for 4 weeks. Then, they undergo a 2-week washout period, and then, enter Period 2 where they receive the matched placebo spray and again undergo a 2-week titration period, and a 4-week consistent daily dosage period.

Who Is Running the Clinical Trial?

Michael, Levy M.D.,Ph.D.

Lead Sponsor

Trials

1

Recruited

40+

Jazz Pharmaceuticals

Industry Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

References

1. England pubmed.ncbi.nlm.nih.gov

Batoclimab as an add-on therapy in neuromyelitis optica spectrum disorder patients with acute attacks. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Neuromyelitis Optica. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Unexpected exacerbations following initiation of disease-modifying drugs in neuromyelitis optica spectrum disorder: Which factor is responsible, anti-aquaporin 4 antibodies, B cells, Th1 cells, Th2 cells, Th17 cells, or others? [2018]

4. Spain pubmed.ncbi.nlm.nih.gov

Experience with tocilizumab in patients with neuromyelitis optica spectrum disorders. [2021]

5. Spain pubmed.ncbi.nlm.nih.gov

Experience with tocilizumab in patients with neuromyelitis optica spectrum disorders. [2022]