MYOBLOC for Spasticity

Not currently recruiting at 8 trial locations

Overseen By Joseph Hull, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Supernus Pharmaceuticals, Inc.

Must not be taking: Aminoglycosides, Curare-like agents

Disqualifiers: Epilepsy, Neuromuscular disorders, others

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

Compensation: Varies

Number of Visits: 2

Duration: 13 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test MYOBLOC, a treatment for reducing upper limb spasticity (muscle stiffness) in adults. It will compare different doses of MYOBLOC to a placebo to evaluate its effectiveness and safety. Individuals with upper limb spasticity due to a stroke, brain injury, or spinal cord injury for more than six months might be suitable candidates. Participants must be able to follow study instructions and complete questionnaires, possibly with assistance from a caregiver.

As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval. It offers participants a chance to contribute to potentially groundbreaking advancements in treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications, but it does mention that you should not use certain medications like aminoglycoside antibiotics or muscle relaxants close to the trial start. It's best to discuss your current medications with the trial team to see if any adjustments are needed.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that MYOBLOC, a type of botulinum toxin, is already approved by the FDA for certain uses, indicating it has passed extensive safety tests. Studies have found that MYOBLOC can cause side effects like trouble swallowing and breathing. Although these side effects can be serious, they are uncommon in adults. Most people tolerate the treatment well, experiencing only mild or moderate reactions.

Safety reports from other MYOBLOC trials indicate that, in some cases, the effects of the toxin can last longer than expected. However, these situations are not typical. For those considering joining a trial, it's important to know that MYOBLOC's safety record is well-documented, and serious side effects are rare. Always consult a healthcare provider about any potential risks. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for spasticity?

Researchers are excited about MYOBLOC for treating spasticity because it offers a different approach compared to current options like botulinum toxin type A formulations. Unlike these standard treatments, MYOBLOC uses botulinum toxin type B, which might work more effectively for patients who don't respond well to type A or develop resistance over time. Additionally, MYOBLOC could potentially provide quicker relief due to its unique molecular characteristics, offering a new hope for those dealing with muscle stiffness and spasms.

What evidence suggests that MYOBLOC might be an effective treatment for spasticity?

Research has shown that MYOBLOC may help treat upper limb spasticity, a condition where muscles become stiff and hard to control. Previous studies found that it can improve muscle control and reduce stiffness. In one study, participants walked faster, increasing their speed by 0.044 meters per second, suggesting that MYOBLOC might enhance movement. It also effectively treats other issues, like neck muscle spasms, which supports its use for spasticity. Overall, evidence suggests MYOBLOC could be a helpful treatment option. Participants in this trial will receive either a low dose or high dose of MYOBLOC, or a placebo, to evaluate its effectiveness for spasticity. ^{1 2 3 6 7}

Who Is on the Research Team?

Jonathan Rubin, MD, MBA

Principal Investigator

Supernus Pharmaceuticals

Are You a Good Fit for This Trial?

Adults aged 18-80 with upper limb spasticity from stroke, brain injury, or spinal cord injury that happened at least 6 months ago. They must understand the study and consent to participate (or have a representative do so), be able to follow the protocol for a year, and have certain muscle stiffness levels. Excluded are those with severe lung disease, recent drug/alcohol abuse, other medical risks, recent major surgery or botulinum toxin treatment.

Inclusion Criteria

The investigator is willing and able to comply with all requirements of the protocol, including completion of study questionnaires

I have stiffness in at least two of these muscle groups: elbow, wrist, or fingers.

In the Investigator's opinion, the subject will be willing and able to comply with all requirements of the protocol, including completion of study questionnaires. A caregiver may be designated to assist with the physical completion of questionnaires/scales.

Exclusion Criteria

I have severe joint stiffness in my arm that limits movement.

You are allergic to botulinum toxin type A or B or any ingredients in MYOBLOC.

Presence of a spinal stimulator or intrathecal baclofen pump that has not been turned off within 30 days prior to screening.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single treatment of MYOBLOC or placebo for upper limb spasticity

13 weeks

Visits at baseline, Weeks 2, 4, 8, and 13

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants receive 5 separate MYOBLOC treatments approximately 13 weeks apart

65 weeks

What Are the Treatments Tested in This Trial?

Interventions

• MYOBLOC

Trial Overview The trial is testing MYOBLOC's effectiveness and safety in treating adult upper limb spasticity compared to placebo. It's a Phase 2/3 randomized controlled trial with double-blind procedures initially followed by an open-label extension for further safety evaluation.

How Is the Trial Designed?

Group I: Phase 3; MYOBLOC Experimental Treatment 1 Intervention

MYOBLOC is a single treatment and will be compared to volume-matched placebo

Group II: Phase 2; Low Dose MYOBLOC Experimental Treatment 1 Intervention

Low Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo

Group III: Phase 2; High Dose MYOBLOC Experimental Treatment 1 Intervention

High Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo

Group IV: Phase 2; Placebo Placebo Group 1 Intervention

Volume-matched placebo is a single treatment

Group V: Phase 3; Placebo Placebo Group 1 Intervention

Volume-matched placebo is a single treatment

Who Is Running the Clinical Trial?

Supernus Pharmaceuticals, Inc.

Lead Sponsor

Trials

49

Recruited

14,000+

Solstice Neurosciences

Industry Sponsor

Trials

7

Recruited

1,100+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04099667

NCT04099667 | Efficacy and Safety Study of MYOBLOC® ... The Phase 3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial will compare the efficacy and safety of MYOBLOC versus placebo in ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11274891/

Improving Spasticity by Using Botulin Toxin - PubMed Central Only a recent systematic review has shown an improvement in gait velocity of a 0.044 m/s increase (with an effect size of 0.193). The authors ...

3. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2009/103846s5120lbl.pdf

Myobloc® (rimabotulinumtoxinB) Injection - accessdata.fda.gov The data reflect the percentage of patients whose test results were considered positive for antibodies to MYOBLOC in both an in vitro and in vivo assay. The ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04815967?term=RIMABOTULINUMTOXINB&viewType=Table&rank=6

Efficacy and Safety Study of MYOBLOC® in the Treatment ... The Phase 3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial will compare the efficacy and safety of MYOBLOC versus placebo in ...

5. myobloc.com myobloc.com/files/Myobloc-Prescribing-Information.pdf

HIGHLIGHTS OF PRESCRIBING INFORMATION The primary efficacy outcome variable for both studies was the Toronto Western Spasmodic Torticollis Rating Scale. (TWSTRS)-Total Score (scale range of ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2019/103846s5190lbl.pdf

MYOBLOC (rimabotulinumtoxinB) - accessdata.fda.gov Postmarketing safety data from MYOBLOC and other approved botulinum toxins suggest that botulinum toxin effects may, in some cases, be observed beyond the ...

7. myoblochcp.com myoblochcp.com/

MYOBLOC - Botulinum Toxin Type B Therapy Swallowing and breathing difficulties can be life threatening and there have been reports of death. The risk of symptoms is probably greatest in children ...