Botox for Trigeminal Neuralgia

Overseen By Amber Fu

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Stanford University

Must not be taking: Aminoglycosides

Disqualifiers: Secondary TN, Pregnant, Neuromuscular, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

Duration: 4 weeks

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if Botox (OnabotulinumtoxinA) can reduce pain for individuals with Trigeminal Neuralgia, a condition causing severe facial pain. Participants will receive either Botox injections or a placebo (a harmless, inactive substance) to compare effects. The trial seeks individuals who experience more than ten painful attacks per week and have not responded well to at least two different medications. Participants should have avoided or not want surgery and be able to attend regular in-person visits. As a Phase 4 trial, this research helps clarify how the already FDA-approved and effective treatment can benefit more patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it requires that your medication dose has been stable for the last 2 weeks. It's best to discuss your specific medications with the trial team.

What is the safety track record for OnabotulinumtoxinA?

Research shows that Botox, also known as OnabotulinumtoxinA, is generally safe for people with trigeminal neuralgia, a type of nerve pain in the face. Studies indicate that serious side effects rarely occur. One study found that 14 out of 108 patients (about 12.9%) experienced mild facial asymmetry, where one side of the face appeared slightly different from the other. This was the most common side effect reported.

Overall, Botox is well-tolerated, and many patients have found it effective in reducing pain. Notably, the FDA has already approved Botox for other conditions, supporting its safety. For those considering joining a trial, these findings suggest that the treatment is generally safe, with only a few mild side effects reported. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Unlike the standard treatments for trigeminal neuralgia, which often include medications like carbamazepine or oxcarbazepine that work by stabilizing nerve activity, OnabotulinumtoxinA offers a different approach. This treatment involves intradermal injections in specific facial areas, using a toxin that temporarily blocks nerve signals responsible for pain. Researchers are excited about OnabotulinumtoxinA because it targets pain directly at its source without the systemic side effects associated with traditional oral medications. Moreover, it could provide relief for patients who don't respond well to existing options, potentially offering faster and more localized pain control.

What is the effectiveness track record for OnabotulinumtoxinA in treating Trigeminal Neuralgia?

Research has shown that Botox, a well-known treatment, can effectively help with trigeminal neuralgia, a condition causing severe facial pain. One study found that 89.4% of patients experienced significant pain relief within two weeks of receiving Botox. This trial will compare the effects of OnabotulinumtoxinA, the active ingredient in Botox, with a placebo. This treatment not only eases pain but also reduces the frequency of painful episodes. Doctors have observed good results with Botox, especially for patients who cannot tolerate the side effects of other treatments. Overall, evidence strongly supports Botox as a promising option for managing trigeminal neuralgia. ^{3 4 5 6 7}

Who Is on the Research Team?

Meredith Barad, MD

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for individuals with Trigeminal Neuralgia, a condition causing facial pain. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and not have conditions that could interfere with the study.

Inclusion Criteria

I have tried at least 2 medications without success.

I am legally recognized as able to make my own decisions.

I can attend all required in-person visits.

Exclusion Criteria

I have been diagnosed with a specific type of facial pain.

I am not pregnant or breastfeeding.

I have a neuromuscular disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intradermal injections of OnabotulinumtoxinA or saline for trigeminal neuralgia

4 weeks

1 visit (in-person) for injection

Follow-up

Participants are monitored for changes in pain and attack frequency after treatment

3 months

1 visit (in-person) at the end of follow-up

What Are the Treatments Tested in This Trial?

Interventions

• OnabotulinumtoxinA

Trial Overview The trial is testing OnabotulinumtoxinA (Botox) against a saline solution (placebo). Participants will be randomly assigned to receive either Botox or placebo in a controlled setting to compare effectiveness.

How Is the Trial Designed?

Group I: OnabotulinumtoxinA Experimental Treatment 1 Intervention

Intradermal injections will be placed in the affected trigeminal territories according to a specific facial map that we have developed.

Group II: Saline Placebo Group 1 Intervention

The same procedure will be followed as above, but saline will be injected instead of onabotA

OnabotulinumtoxinA is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Botox for:

• Chronic migraine
• Involuntary muscle contractions or twitching
• Excessive sweating
• Increased saliva
• Overactive bladder
• Urinary incontinence
• Facial wrinkles
• Cervical dystonia
• Upper limb spasticity
• Lower limb spasticity

Approved in European Union as Botox for:

• Blepharospasm
• Strabismus
• Axillary hyperhidrosis
• Cervical dystonia
• Upper limb spasticity
• Lower limb spasticity
• Overactive bladder
• Urinary incontinence
• Facial wrinkles

Approved in Canada as Botox for:

• Chronic migraine
• Involuntary muscle contractions or twitching
• Excessive sweating
• Increased saliva
• Overactive bladder
• Urinary incontinence
• Facial wrinkles
• Cervical dystonia
• Upper limb spasticity
• Lower limb spasticity

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

Published Research Related to This Trial

In a study involving 36 patients with essential trigeminal neuralgia, a single injection of onabotulinum toxin A (BTX) significantly reduced pain compared to placebo, with a notable difference in pain scores observed three months post-treatment (VAS 4.75 vs 6.94; P = 0.01).

Onabotulinum toxin A was found to be well tolerated and safe, suggesting it could be an effective treatment option for managing pain in patients with this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute treatment of trigeminal neuralgia with onabotulinum toxin A. Zúñiga, C., Piedimonte, F., Díaz, S., et al.[2022]

In a study of 22 patients with refractory trigeminal neuralgia, botulinum toxin-A (BTX-A) significantly reduced pain scores at multiple time points after treatment, with the most notable improvement observed at 60 days post-injection.

The average pain score decreased from 7.14 before treatment to as low as 4.77 after BTX-A administration, indicating a percentage reduction in pain scores ranging from 20.4% to 33.1%, suggesting BTX-A is a viable option for managing refractory trigeminal neuralgia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Botulinum Toxin Type A in Trigeminal Neuralgia in a South Asian Cohort. Caldera, MC., Senanayake, SJ., Perera, SP., et al.[2022]

In a pilot study involving 13 patients with trigeminal neuralgia, treatment with botulinum-A neurotoxin (BoNT/A) significantly reduced pain levels and the area affected by pain across all branches of the trigeminal nerve.

The treatment was found to be effective with no major side effects reported, suggesting it is a safe option, but further confirmation through a placebo-controlled trial is necessary.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An open study of botulinum-A toxin treatment of trigeminal neuralgia. Piovesan, EJ., Teive, HG., Kowacs, PA., et al.[2022]

In a study of 88 patients with trigeminal neuralgia, botulinum toxin-A (BTX-A) demonstrated significant pain relief, with 100% of patients showing effectiveness within 2 months and 25% maintaining complete pain control after 14 months.

The treatment was found to be safe, with only minor side effects like swelling and facial asymmetry, which resolved on their own, indicating that BTX-A is a viable long-term option for managing trigeminal neuralgia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic effect of Botulinum toxin-A in 88 patients with trigeminal neuralgia with 14-month follow-up. Li, S., Lian, YJ., Chen, Y., et al.[2022]

OnabotulinumtoxinA is safe and well tolerated for preventing chronic migraines, with a low discontinuation rate of only 3.4% due to adverse events, based on a pooled analysis of 2436 patients from four clinical trials.

The most common side effects included neck pain (12.6%) and muscle weakness (8.0%), but serious adverse events were rare, occurring in only 5.4% of patients, indicating a favorable safety profile for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pooled analysis of the safety and tolerability of onabotulinumtoxinA in the treatment of chronic migraine. Diener, HC., Dodick, DW., Turkel, CC., et al.[2021]

In a study of 53 patients with trigeminal neuralgia, onabotulinumtoxinA (BTX-A) was effective in reducing pain severity and frequency in 52% of patients with multiple sclerosis-related trigeminal neuralgia (TN-MS) and 45% of patients with primary trigeminal neuralgia (TN-P).

The effectiveness of BTX-A treatment was influenced by patient factors, showing better results in those with continuous pain and less effectiveness in patients with a history of previous interventional treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of multiple sclerosis-related trigeminal neuralgia with onabotulinumtoxinA. Asan, F., Gündüz, A., Tütüncü, M., et al.[2023]

In a study of 90 patients with trigeminal neuralgia, local injections of onabotulinum toxin type A (botox) significantly reduced the frequency of pain attacks over three months, with the number of paroxysms decreasing from 31.2 to 9.2 per day (p<0.001).

While pain intensity showed a modest decrease initially, it improved significantly by the end of the three months, indicating that botox is a safe and effective symptomatic treatment for trigeminal neuralgia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[The effectiveness of botulinum therapy of trigeminal neuralgia]. Mingazova, LR., Orlova, OR., Soiher, MI., et al.[2021]

In a study of 27 patients with trigeminal neuralgia, injections of 100 Units of botulinum toxin type-A (BTX-A) significantly reduced pain intensity and frequency, with 88.9% of patients showing a ≥50% reduction in pain by the sixth month.

The treatment was well tolerated, with few adverse events reported, and 44% of patients experienced complete pain relief at the sixth month, indicating that this method is both effective and safe for managing trigeminal neuralgia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Botulinum toxin in the treatment of trigeminal neuralgia: 6-Month follow-up. Türk Börü, Ü., Duman, A., Bölük, C., et al.[2021]

Onabotulinum toxin A (BTX-A) treatment provided complete pain relief for a 60-year-old male with trigeminal neuralgia (TN) caused by an exostosis, demonstrating its efficacy as a last-resort option when other treatments failed.

The patient maintained a sustained analgesic effect over 28 months with regular BTX-A injections, indicating that it has a good safety profile and can be an effective treatment for TN in patients who are not candidates for conventional therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Botulinum Toxin as Monotherapy in Symptomatic Trigeminal Neuralgia. Lunde, HM., Torkildsen, Ø., Bø, L., et al.[2022]

In a study of 12 patients with idiopathic trigeminal neuralgia, botulinum toxin type A injections led to significant pain relief in 10 patients, with some experiencing complete pain disappearance for up to 60 days.

The injections were administered in specific trigger zones, including the masseter muscle for those with mandibular involvement, indicating a targeted approach that may enhance the treatment's efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Beneficial effects of botulinum toxin type A in trigeminal neuralgia. Zúñiga, C., Díaz, S., Piedimonte, F., et al.[2022]

Botulinum toxin type A (BTX-A) shows promising efficacy in treating trigeminal neuralgia, with response rates between 70-100% and significant reductions in pain intensity and frequency (60-100%) observed within 4 weeks after treatment across multiple studies.

The safety profile of BTX-A appears favorable, as major adverse events were not reported; however, more rigorous randomized controlled trials are needed to establish optimal treatment protocols and further assess its long-term efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Therapeutic efficacy and safety of botulinum toxin type A in trigeminal neuralgia: a systematic review. Hu, Y., Guan, X., Fan, L., et al.[2022]

Botulinum toxin type A is effective in reducing pain from idiopathic trigeminal neuralgia in patients over 70 years old, with significant pain reduction observed from an average score of 8.2 before treatment to 4.5 after treatment.

The treatment is considered safe, with no significant difference in side effects between older patients (5 minor side effects) and younger patients (2 minor side effects), indicating similar tolerability across age groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Evaluation of therapeutic effectiveness and safety of botulinum toxin type A in the treatment of idiopathic trigeminal neuralgia in patients older than 70 years]. Xu, Y., Zhang, Q., Mao, C., et al.[2018]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9375637/

Therapeutic Efficacy of Botulinum Toxin in Trigeminal ... An open retrospective study involving 152 patients showed an 89.4% effective rate within two weeks with botulinum toxin injection, with more ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06216886

OnabotulinumtoxinA for Trigeminal Neuralgia This study will offer onabotulinumtoxin A (Botox) delivered intradermally into the region of pain for the patient with trigeminal neuralgia.

3. practicalneurology.com practicalneurology.com/diseases-diagnoses/headache-pain/onabotulinumtoxina-for-trigeminal-neuralgia-treatment/32127/

OnabotulinumtoxinA for Trigeminal Neuralgia Treatment We have seen positive results overall with this treatment at our tertiary care headache center. Clinicians should consider onabotulinumtoxinA ...

4. thejournalofheadacheandpain.biomedcentral.com thejournalofheadacheandpain.biomedcentral.com/articles/10.1186/s10194-024-01926-9

Efficacy and safety profile of Onabotulinum toxin-A injection at ... Onabotulinum toxin-A injection at the SPG is effective in reducing pain symptoms in trigeminal neuralgia patients who cannot tolerate the side effects of ...

5. mdpi.com mdpi.com/2072-6651/15/11/654

The Therapeutic Effect of Botulinum Toxin Type A on ... Conclusions: Botulinum toxin type A effectively reduced the pain, paroxysm frequency, and PFPS scores of type 1 and type 2 trigeminal neuralgia ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4932020/

Therapeutic efficacy and safety of Botulinum Toxin A Therapy ... Despite limited data, our results suggest that BTX-A may be an effective and safe treatment option for patients with TN.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6190814/

Botulinum toxin-A for the treatment of neuralgia No serious adverse events associated with BTX-A were noted. Fourteen out of 108 patients (12.9%) with trigeminal neuralgia experienced mild facial asymmetry ...