Rosacea
Conditions
Keywords
Rosacea
Brief summary
This is a double-blinded, randomized placebo controlled pilot study comparing the efficacy and safety of incobotulinumtoxinA versus saline injections to the cheek region in patients with rosacea. The pilot study will enroll and treat a total of 10 subjects who present with rosacea of the cheek area. Upon study entry, subjects will be randomized to receive treatment with incobotulinumtoxinA or bacteriostatic saline to the cheek area. Study treatment will be prepared by an unblinded designee and both Physician Investigator (PI) and subject will remain blinded for the duration of the study. At the 16 week visit, control subjects will enter the rescue arm portion of the study and all study subjects will receive treatment with incobotulinumtoxinA to the cheek areas.
Interventions
Experimental injection given at baseline and Week 16
Placebo at baseline, incobotulinumtoxinA at 16 weeks
Sponsors
Merz North America, Inc.
DeNova Research
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Males and females between 18 and 65 years of age. 2. Subjects presenting with rosacea in the cheek area. 3. Willingness and ability to comply with protocol requirements, including returning for follow-up visits and abstaining from exclusionary procedures for the duration of the study. 4. Subjects of childbearing potential must have a negative urine pregnancy test result at Visit 1 and be willing able to use an acceptable method of birth control (e.g., barrier methods used with a spermicidal agent, hormonal methods, IUD, surgical sterilization, abstinence) during the study. Women will not be considered of childbearing potential if one of the following is documented on the medical history: * postmenopausal for at least 12 months prior to study drug administration * without a uterus and/or both ovaries * has had a bilateral tubal ligation for at least 6 months prior to study drug administration. * absence of an other physical condition according to the PI's discretion 5. Willingness and ability to provide written photo consent and adherence to photography procedures (i.e., removal of jewelry and makeup). 6. Willingness and ability to provide written informed consent prior to performance of any study related procedure.
Exclusion criteria
1. Subjects who are pregnant, nursing, planning to become pregnant, and/or not using a reliable form of birth control. 2. Subjects with a known allergy or sensitivity to any component of the study medications or anesthesia. 3. Topical or oral rosacea treatments within the past 2 weeks. 4. Subjects with a significant systemic illness or illness localized to the areas of treatment. 5. Botulinum toxin to the face within the past 6 months. 6. Significant concurrent illness such as diabetes, epilepsy, lupus, or congestive heart failure. 7. Concurrent skin condition affecting area to be treated. 8. Prior surgery on the area to be treated within 3 months of initial treatment or during the study. 9. History or evidence of keloids or hypertrophic scarring. 10. Subjects currently using aminoglycoside antibiotics, curare-like agents or other agents that might interfere with neuromuscular function. 11. Subjects with a diagnosis of Myasthenia Gravis, Eaton-Lambert Syndrome, Amyotrophic Lateral Sclerosis, or any other disease that might interfere with neuromuscular function or current facial palsy. 12. Current history of chronic drug or alcohol abuse. 13. Concurrent therapy that, in the investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study medication. 14. Subjects who, in the Investigator's opinion, have a history of poor cooperation, non¬compliance with medical treatment or unreliability. 15. Enrollment in any active study involving the use of investigational devices or drugs.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change From Baseline in the Rosacea Clinical Score Card | baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks | Live rosacea assessment for each side of the face using the Rosacea Clinical Scorecard for clinical assessment.The Rosacea Clinical Scorecard assesses the primary signs and symptoms of rosacea and is graded as absent (0), mild (1), moderate (2) or severe (3). |
| Number of Participants Experiencing Adverse Events | baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks | Rate of adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks | Self-esteem change will be determined by patient self-evaluation using the Heatherton & Polivy State Self-Esteem (HPSS) Scale. Heatherton & Polivy State Self-Esteem (HPSS) Scale includes three facets of self-esteem: Appearance, Performance, and Social The scale has a range of 20-100. A score closer to 100 signifies higher self-esteem, while a score closer to 20 signifies lower seld-esteem. |
| Patient Satisfaction | week 1, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks | 4-point categorical assessment of patient satisfaction with treatment. 1. Highly Satisfied- Optimal cosmetic result 2. Very Satisfied- Obvious improvement in appearance form the initial condition, but not completely optimal for this subject 3. Satisfied- Marked improvement in appearance from initial condition 4. Unsatisfied- The appearance is essentially the same or worse as the original condition. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Group 1 - IncobotulinumtoxinA Experimental injection given at baseline and Week 16 | 4 |
| Group 2 - Bacteriostatic Saline Placebo at baseline, incobotulinumtoxinA at 16 weeks | 5 |
| Total | 9 |
Baseline characteristics
| Characteristic | Group 1 - IncobotulinumtoxinA | Group 2 - Bacteriostatic Saline | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 5 Participants | 9 Participants |
| Age, Continuous | 42 Years | 53 Years | 47 Years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 3 Participants | 5 Participants | 8 Participants |
| Sex: Female, Male Female | 2 Participants | 5 Participants | 7 Participants |
| Sex: Female, Male Male | 2 Participants | 0 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 4 | 0 / 5 |
| serious Total, serious adverse events | 0 / 4 | 0 / 5 |
Outcome results
Primary
Number of Participants Experiencing Adverse Events
Rate of adverse events
Time frame: baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Group 1 - IncobotulinumtoxinA | Number of Participants Experiencing Adverse Events | 0 participants |
| Group 2 - Bacteriostatic Saline | Number of Participants Experiencing Adverse Events | 0 participants |
Primary
Percent Change From Baseline in the Rosacea Clinical Score Card
Live rosacea assessment for each side of the face using the Rosacea Clinical Scorecard for clinical assessment.The Rosacea Clinical Scorecard assesses the primary signs and symptoms of rosacea and is graded as absent (0), mild (1), moderate (2) or severe (3).
Time frame: baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 4 | -0.65 percent change | Standard Deviation 0.6 |
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 16 | -0.67 percent change | Standard Deviation 0.48 |
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 1 | -0.63 percent change | Standard Deviation 0.49 |
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 17 | -0.83 percent change | Standard Deviation 0.62 |
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 12 | -0.80 percent change | Standard Deviation 0.61 |
| Group 1 - IncobotulinumtoxinA | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 20 | -1.04 percent change | Standard Deviation 8.85 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 12 | -0.13 percent change | Standard Deviation 0.13 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 1 | -0.00063 percent change | Standard Deviation 0.23 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 4 | -0.34 percent change | Standard Deviation 0.21 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 20 | -0.7 percent change | Standard Deviation 0.53 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 16 | -0.51 percent change | Standard Deviation 0.34 |
| Group 2 - Bacteriostatic Saline | Percent Change From Baseline in the Rosacea Clinical Score Card | % Diff from baseline, Week 17 | -0.83 percent change | Standard Deviation 0.64 |
Secondary
Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks
Self-esteem change will be determined by patient self-evaluation using the Heatherton & Polivy State Self-Esteem (HPSS) Scale. Heatherton & Polivy State Self-Esteem (HPSS) Scale includes three facets of self-esteem: Appearance, Performance, and Social The scale has a range of 20-100. A score closer to 100 signifies higher self-esteem, while a score closer to 20 signifies lower seld-esteem.
Time frame: baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 4 | 53.7 units on a scale | Standard Deviation 15.7 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 16 | 50.3 units on a scale | Standard Deviation 11 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 1 | 53.0 units on a scale | Standard Deviation 11.8 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 17 | 52.0 units on a scale | Standard Deviation 10.4 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 12 | 52.8 units on a scale | Standard Deviation 12.6 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 20 | 51.0 units on a scale | Standard Deviation 12.2 |
| Group 1 - IncobotulinumtoxinA | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Baseline | 50.5 units on a scale | Standard Deviation 8.19 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 20 | 34.2 units on a scale | Standard Deviation 8.38 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Baseline | 35.2 units on a scale | Standard Deviation 10 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 1 | 36.0 units on a scale | Standard Deviation 9.35 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 4 | 34.2 units on a scale | Standard Deviation 10.1 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 12 | 32.2 units on a scale | Standard Deviation 7.16 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 16 | 34.6 units on a scale | Standard Deviation 8.88 |
| Group 2 - Bacteriostatic Saline | Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks | Week 17 | 33.6 units on a scale | Standard Deviation 5.32 |
Secondary
Patient Satisfaction
4-point categorical assessment of patient satisfaction with treatment. 1. Highly Satisfied- Optimal cosmetic result 2. Very Satisfied- Obvious improvement in appearance form the initial condition, but not completely optimal for this subject 3. Satisfied- Marked improvement in appearance from initial condition 4. Unsatisfied- The appearance is essentially the same or worse as the original condition.
Time frame: week 1, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 1 | 3.0 units on a scale | Standard Deviation 0.76 |
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 4 | 2.3 units on a scale | Standard Deviation 0.52 |
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 12 | 3.0 units on a scale | Standard Deviation 0 |
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 16 | 2.7 units on a scale | Standard Deviation 0.52 |
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 17 | 2.3 units on a scale | Standard Deviation 1 |
| Group 1 - IncobotulinumtoxinA | Patient Satisfaction | Week 20 | 1.7 units on a scale | Standard Deviation 0.52 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 17 | 2.9 units on a scale | Standard Deviation 0.32 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 1 | 3.8 units on a scale | Standard Deviation 0.42 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 16 | 3.2 units on a scale | Standard Deviation 0.79 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 4 | 3.6 units on a scale | Standard Deviation 0.52 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 20 | 2.2 units on a scale | Standard Deviation 0.79 |
| Group 2 - Bacteriostatic Saline | Patient Satisfaction | Week 12 | 3.3 units on a scale | Standard Deviation 0.49 |