NCT02010775

Botulinum Toxin Type A (BOTOX®) Treatment of Masseter Muscle Hypertrophy

Completed Phase 2 Results posted Last updated 28 May 2019

What this trial tests

Phase 2 trial testing botulinum toxin Type A in Masseter Muscle Hypertrophy in 187 participants. Completed in 29 November 2017.

Timeline

20 January 2014

Primary endpoint
7 March 2017

29 November 2017

Quick facts

Lead sponsor	Allergan
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	187
Start date	20 January 2014
Primary completion	7 March 2017
Estimated completion	29 November 2017
Sites	14 locations across Taiwan, Canada, Australia

Drugs / interventions tested

• botulinum toxin Type A (BOTULINUM TOXIN TYPE A) — full drug profile →
• Normal saline

Conditions studied

• Masseter Muscle Hypertrophy — all drugs for Masseter Muscle Hypertrophy →

Sponsor

Allergan — full company profile →

Who can join

Adults 18 to 50, any sex, with Masseter Muscle Hypertrophy. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline in Lower Facial Volume Using VECTRA 3D Images Primary · Baseline (Day 1) to Day 90 of Treatment Cycle 1

Lower facial volume was calculated from 3-dimensional (3D) images captured with the VECTRA M3 3D Stereophotogrammetry imaging system and was analyzed using computer assisted systems and predetermined facial landmarks. The difference in volume was measured between the select region of the baseline surface 3D model and the select region of the posttreatment surface 3D model. A negative change from Baseline (decrease in volume) indicates improvement.

Group	Value	95% CI
BOTOX® 96U	-8.20	-15.6 – -1.4
BOTOX® 72U	-7.41	-16.5 – -0.8
BOTOX® 48U	-6.84	-17.4 – 1.6
BOTOX® 24U	-4.40	-13.2 – 2.0
Placebo	-0.52	-6.7 – 9.0

Percentage of Participants Who Achieved a Masseter Muscle Prominence Scale (MMPS) Grade ≤ 3 as Assessed by the Investigator Secondary · Day 90 of Treatment Cycle 1

The investigator used visual inspection and palpation to grade the prominence of the participant's masseter muscle on the left and right sides of the face using the MMPS where: 1=minimal prominence (best), 2=mild prominence, 3=moderate prominence, 4=marked prominence, 5=very marked prominence (worst). The percentage of participants with grade 3 or less is reported.

Group	Value	95% CI
BOTOX® 96U	89.5	75.2 – 97.1
BOTOX® 72U	89.5	75.2 – 97.1
BOTOX® 48U	83.3	67.2 – 93.6
BOTOX® 24U	66.7	49.0 – 81.4
Placebo	35.1	20.2 – 52.5

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline (Day 1) to the end of study (Month 12). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

BOTOX® 96U

Serious: 1/38 (3%)

Deaths: 0/38

BOTOX® 72U

Serious: 0/38 (0%)

Deaths: 0/38

BOTOX® 48U

Serious: 0/37 (0%)

Deaths: 0/37

BOTOX® 24U

Serious: 2/37 (5%)

Deaths: 0/37

Placebo

Serious: 3/37 (8%)

Deaths: 0/37

Serious adverse events (8 terms)

Reaction	System	BOTOX® 96U	BOTOX® 72U	BOTOX® 48U	BOTOX® 24U	Placebo
Appendicitis perforated	Infections and infestations	—	—	—	—	—
Peritonitis	Infections and infestations	—	—	—	—	—
Kidney infection	Infections and infestations	—	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—	—
Wrist fracture	Injury, poisoning and procedural complications	—	—	—	—	—
Hepatic cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—
Uterine leiomyomax	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—
Abortion induced	Surgical and medical procedures	—	—	—	—	—

Other adverse events (14 terms — click to expand)

Reaction	System	BOTOX® 96U	BOTOX® 72U	BOTOX® 48U	BOTOX® 24U	Placebo
Nasopharyngitis	Infections and infestations	—	—	—	—	—
Influenza	Infections and infestations	—	—	—	—	—
Facial paresis	Nervous system disorders	—	—	—	—	—
Mastication disorder	Musculoskeletal and connective tissue disorders	—	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—
Irritable bowel syndrome	Gastrointestinal disorders	—	—	—	—	—
Muscle strain	Injury, poisoning and procedural complications	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—
Pain in jaw	Musculoskeletal and connective tissue disorders	—	—	—	—	—
Injection site pain	General disorders	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—
Ligament sprain	Injury, poisoning and procedural complications	—	—	—	—	—
Food allergy	Immune system disorders	—	—	—	—	—

Most-reported serious reactions: Appendicitis perforated, Peritonitis, Kidney infection, Fall, Wrist fracture, Hepatic cancer, Uterine leiomyomax, Abortion induced.

Data from ClinicalTrials.gov NCT02010775 adverse events section.

Sponsor's own description

This study will evaluate the safety and efficacy of a range of doses of botulinum toxin Type A (BOTOX®) for the treatment of patients with bilateral Masseter Muscle Hypertrophy (MMH).

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

1. A Controlled Phase 2b Trial to Assess the Efficacy and Safety of a Single Intervention of OnabotulinumtoxinA for Treating Masseter Muscle Prominence.
   Liew S, Jones D, Dayan S, Fabi S, et al · · 2025 · cited 1× · PMID 40111171 · DOI 10.1093/asj/sjaf042
2. OnabotulinumtoxinA Treatment for Masseter Muscle Prominence: 6-Month Safety and Efficacy Results, Including Patient-Reported Outcomes, From a Phase 3, Randomized, Placebo-Controlled, Multiregional Trial.
   Sun J, Wang L, Wu Y, Humphrey S, et al · · 2026 · PMID 41092464 · DOI 10.1093/asj/sjaf204
3. Longitudinal Computed Tomography Indicates No Negative Impact of OnabotulinumtoxinA on Mandibular Bone Density in a 12-Month, Double-Blind, Randomized, Repeat Treatment, Placebo-Controlled Study in Healthy Adults With Masseter Muscle Prominence.
   Kostenuik P, Mallya SM, Hopfinger R, Aghaloo T, et al · · 2025 · PMID 40843518 · DOI 10.1093/asj/sjaf167
4. Improvement of Lower Facial Shape after Treatment with OnabotulinumtoxinA: Secondary Results from a Phase 2 Dose Escalation Study.
   Liew S, Rivers JK, Humphrey S, Carruthers J, et al · · 2026 · PMID 40801407 · DOI 10.1097/prs.0000000000012345