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

• Sponsor: DeNova Research (Other)
• Overall Status: Completed
• CT.gov ID: NCT01614743
• Collaborator: Merz North America, Inc. (Industry)
• Enrollment: 9
• Location: 1
• Arms: 2
• Duration (Months): 17
• Patients Per Site Per Month: 0.5

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Rosacea	Drug: IncobotulinumtoxinA; Drug: Bacteriostatic saline	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 9 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Triple (Participant, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Study Start Date: Jun 1, 2012
• Actual Primary Completion Date: Nov 1, 2013
• Actual Study Completion Date: Nov 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: IncobotulinumtoxinA	Drug: IncobotulinumtoxinA; Experimental injection given at baseline and Week 16
Placebo Comparator: Placebo	Drug: Bacteriostatic saline; Placebo at baseline, incobotulinumtoxinA at 16 weeks

Outcome Measures

Primary Outcome Measures

1. 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).

2. 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 Outcome Measures

1. 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.

2. Patient Satisfaction [week 1, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks]

   4-point categorical assessment of patient satisfaction with treatment. Highly Satisfied- Optimal cosmetic result Very Satisfied- Obvious improvement in appearance form the initial condition, but not completely optimal for this subject Satisfied- Marked improvement in appearance from initial condition Unsatisfied- The appearance is essentially the same or worse as the original condition.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts 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

1. Willingness and ability to provide written photo consent and adherence to photography procedures (i.e., removal of jewelry and makeup).

2. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	DeNova Research	Chicago	Illinois	United States	60611

Sponsors and Collaborators

• DeNova Research
• Merz North America, Inc.

Investigators

• Principal Investigator: Steven H Dayan, MD, DeNova Research

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• DeNova Research Website

Publications

None provided.

• Responsible Party: Steven H. Dayan, Medical Director, DeNova Research
• ClinicalTrials.gov Identifier: NCT01614743
• Other Study ID Numbers: ROS-INC-12
• First Posted: Jun 8, 2012
• Last Update Posted: Oct 8, 2020
• Last Verified: Sep 1, 2020

• Keywords provided by Steven H. Dayan, Medical Director, DeNova Research: Rosacea
• Additional relevant MeSH terms: Rosacea; incobotulinumtoxinA

Study Results

Participant Flow

Arm/Group Title	IncobotulinumtoxinA at Baseline and Week 16	Placebo at Baseline, Then incobotulinumtoxinA at 16 Weeks
Arm/Group Description	IncobotulinumtoxinA: Experimental injection given at baseline and Week 16	Bacteriostatic saline: Placebo at baseline, incobotulinumtoxinA at 16 weeks
Period Title: Overall Study
STARTED	4	5
COMPLETED	3	5
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	Group 1 - IncobotulinumtoxinA	Group 2 - Bacteriostatic Saline	Total
Arm/Group Description	Experimental injection given at baseline and Week 16	Placebo at baseline, incobotulinumtoxinA at 16 weeks	Total of all reporting groups
Overall Participants	4	5	9
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	4 100%	5 100%	9 100%
>=65 years	0 0%	0 0%	0 0%
Age (Years) [Mean (Full Range) ]
Mean (Full Range) [Years]	42	53	47
Sex: Female, Male (Count of Participants)
Female	2 50%	5 100%	7 77.8%
Male	2 50%	0 0%	2 22.2%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	1 25%	0 0%	1 11.1%
White	3 75%	5 100%	8 88.9%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%

Outcome Measures

1. Primary Outcome

Title	Percent Change From Baseline in the Rosacea Clinical Score Card
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Group 1 - IncobotulinumtoxinA	Group 2 - Bacteriostatic Saline
Arm/Group Description	IncobotulinumtoxinA: Experimental injection given at baseline and Week 16	Bacteriostatic saline: Placebo at baseline, incobotulinumtoxinA at 16 weeks
Measure Participants	4	5
% Diff from baseline, Week 1	-0.63 (0.49)	-0.00063 (0.23)
% Diff from baseline, Week 4	-0.65 (0.60)	-0.34 (0.21)
% Diff from baseline, Week 12	-0.80 (0.61)	-0.13 (0.13)
% Diff from baseline, Week 16	-0.67 (0.48)	-0.51 (0.34)
% Diff from baseline, Week 17	-0.83 (0.62)	-0.83 (0.64)
% Diff from baseline, Week 20	-1.04 (8.85)	-0.7 (0.53)

2. Primary Outcome

Title	Number of Participants Experiencing Adverse Events
Description	Rate of adverse events
Time Frame	baseline, 1 week, 4 weeks, 12 weeks, 16 weeks, 17 weeks and 20 weeks

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Group 1 - IncobotulinumtoxinA	Group 2 - Bacteriostatic Saline
Arm/Group Description	IncobotulinumtoxinA: Experimental injection given at baseline and Week 16	Bacteriostatic saline: Placebo at baseline, incobotulinumtoxinA at 16 weeks
Measure Participants	4	5
Number [participants]	0 0%	0 0%

3. Secondary Outcome

Title	Mean Heatherton & Polivy State Self-Esteem (HPSS) Scale Score at Baseline, 1, 4, 12, 16, 17, and 20 Weeks
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Group 1 - IncobotulinumtoxinA	Group 2 - Bacteriostatic Saline
Arm/Group Description	IncobotulinumtoxinA: Experimental injection given at baseline and Week 16	Bacteriostatic saline: Placebo at baseline, incobotulinumtoxinA at 16 weeks
Measure Participants	4	5
Baseline	50.5 (8.19)	35.2 (10.0)
Week 1	53.0 (11.8)	36.0 (9.35)
Week 4	53.7 (15.7)	34.2 (10.1)
Week 12	52.8 (12.6)	32.2 (7.16)
Week 16	50.3 (11.0)	34.6 (8.88)
Week 17	52.0 (10.4)	33.6 (5.32)
Week 20	51.0 (12.2)	34.2 (8.38)

4. Secondary Outcome

Title	Patient Satisfaction
Description	4-point categorical assessment of patient satisfaction with treatment. Highly Satisfied- Optimal cosmetic result Very Satisfied- Obvious improvement in appearance form the initial condition, but not completely optimal for this subject Satisfied- Marked improvement in appearance from initial condition 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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Group 1 - IncobotulinumtoxinA	Group 2 - Bacteriostatic Saline
Arm/Group Description	IncobotulinumtoxinA: Experimental injection given at baseline and Week 16	Bacteriostatic saline: Placebo at baseline, incobotulinumtoxinA at 16 weeks
Measure Participants	4	5
Week 1	3.0 (0.76)	3.8 (0.42)
Week 4	2.3 (0.52)	3.6 (0.52)
Week 12	3.0 (0.0)	3.3 (0.49)
Week 16	2.7 (0.52)	3.2 (0.79)
Week 17	2.3 (1.0)	2.9 (0.32)
Week 20	1.7 (0.52)	2.2 (0.79)

Adverse Events

Arm/Group Title	Group 1 - IncobotulinumtoxinA		Group 2 - Bacteriostatic Saline
Arm/Group Description	Experimental injection given at baseline and Week 16		Placebo at baseline, incobotulinumtoxinA at 16 weeks
All Cause Mortality
	Group 1 - IncobotulinumtoxinA		Group 2 - Bacteriostatic Saline
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Group 1 - IncobotulinumtoxinA		Group 2 - Bacteriostatic Saline
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Other (Not Including Serious) Adverse Events
	Group 1 - IncobotulinumtoxinA		Group 2 - Bacteriostatic Saline
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)

Limitations/Caveats

The limitation of this study was a very small sample size.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.