A Study to Evaluate the Safety and Efficacy of CB-03-01 Cream, 1% in Subjects With Facial Acne Vulgaris (25)

• ClinicalTrials.gov Identifier: NCT02608450
• Recruitment Status: Completed
• First Posted: November 18, 2015
• Results First Posted: November 20, 2020
• Last Update Posted: November 20, 2020
• Sponsor: Cassiopea SpA
• Information provided by (Responsible Party): Cassiopea SpA

• Study Type: Interventional
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
• Condition: Acne Vulgaris
• Interventions: Drug: CB-03-01 cream, 1%; Drug: Vehicle cream
• Enrollment: 708

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Period Title: Overall Study
Started	353	355
Completed	287	290
Not Completed	66	65
Reason Not Completed
Lost to Follow-up	39	32
Withdrawal by Subject	21	15
Adverse Event	3	6
Withdrawal by parent	2	3
Lack of Efficacy	0	3
Noncompliance with study drug	0	2
Physician Decision	0	1
Pregnancy	0	1
Progressive disease	1	0
Multiple reasons	0	2

Baseline Characteristics

Arm/Group Title		CB-03-01 Cream	Vehicle Cream	Total
Arm/Group Description		CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.	Total of all reporting groups
Overall Number of Baseline Participants		353	355	708
Baseline Analysis Population Description		Number of participants is based on the Intent to treat dataset.
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	353 participants	355 participants	708 participants
		20.0 (6.7)	19.9 (6.8)	19.9 (6.7)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	353 participants	355 participants	708 participants
	Female	221 62.6%	215 60.6%	436 61.6%
	Male	132 37.4%	140 39.4%	272 38.4%
Ethnicity (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	353 participants	355 participants	708 participants
	Hispanic or Latino	94 26.6%	80 22.5%	174 24.6%
	Not Hispanic or Latino	259 73.4%	275 77.5%	534 75.4%
	Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	353 participants	355 participants	708 participants
	American Indian or Alaska Native	0 0.0%	1 0.3%	1 0.1%
	Asian	9 2.5%	10 2.8%	19 2.7%
	Native Hawaiian or Other Pacific Islander	2 0.6%	0 0.0%	2 0.3%
	Black or African American	31 8.8%	38 10.7%	69 9.7%
	White	298 84.4%	297 83.7%	595 84.0%
	More than one race	9 2.5%	7 2.0%	16 2.3%
	Unknown or Not Reported	4 1.1%	2 0.6%	6 0.8%
Baseline IGA [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	353 participants	355 participants	708 participants
	0 - Clear	0 0.0%	0 0.0%	0 0.0%
	1 - Almost Clear	0 0.0%	0 0.0%	0 0.0%
	2- Mild	0 0.0%	0 0.0%	0 0.0%
	3 - Moderate	292 82.7%	291 82.0%	583 82.3%
	4 - Severe	61 17.3%	64 18.0%	125 17.7%
		[1] Measure Description: Investigator's Global Assessment (IGA)
Baseline Acne Lesion Counts; Mean (Standard Deviation); Unit of measure: Lesions	Number Analyzed	353 participants	355 participants	708 participants
Non-inflammatory lesions		59.1 (22.2)	60.7 (22.1)	59.9 (22.1)
Inflammatory lesions		42.4 (11.8)	42.9 (12.3)	42.7 (12.1)
Total Lesion Count		101.5 (25.1)	103.6 (26.1)	102.6 (25.6)

Outcome Measures

1. Primary Outcome

Title	Percentage of Participants Achieving Success in Investigator's Global Assessment (IGA)
Description	Percentage of subjects in each treatment group achieving "success" at Week 12, with "success" defined as an IGA score of "clear (score=0)" or "almost clear (score=1)" and at least a two-point reduction in IGA compared to Baseline.
Time Frame	Week 12

Outcome Measure Data

Analysis Population Description

Intent-to-treat (ITT) population included all randomized subjects.

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Measure Type: Number; Unit of Measure: Percentage of participants
	18.4	9.0

2. Primary Outcome

Title	Change From Baseline in Non-inflammatory Lesion Counts
Description	Absolute change from Baseline in non-inflammatory lesion counts in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population.

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: Non-inflammatory Lesions
	-19.4; (-22.6 to -16.7)	-13.0; (-15.7 to -10.3)

3. Primary Outcome

Title	Change From Baseline in Inflammatory Lesion Counts
Description	Absolute change from Baseline in inflammatory lesion counts in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population.

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: Inflammatory Lesions
	-19.3; (-21.1 to -17.5)	-15.5; (-17.3 to -13.6)

4. Secondary Outcome

Title	Change From Baseline in Total Lesion Counts
Description	Absolute change from Baseline in total lesions counts in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: Total lesions
	-39.1; (-43.0 to -35.1)	-28.8; (-32.6 to -24.9)

5. Secondary Outcome

Title	Percent Change From Baseline in Total Lesion Counts
Description	Percent change from Baseline in total lesions counts in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: percent change
	-37.0; (-40.9 to -33.2)	-28.4; (-32.3 to -24.6)

6. Secondary Outcome

Title	Percent Change From Baseline in Non-inflammatory Lesion (NIL) Counts
Description	Percent change from Baseline in non-inflammatory lesions count in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population.

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: percent change
	-30.6; (-35.6 to -25.7)	-21.6; (-26.4 to -16.8)

7. Secondary Outcome

Title	Percent Change From Baseline in Inflammatory Lesion (IL) Counts
Description	Percent change from Baseline in inflammatory lesions count in each treatment group at Week 12.
Time Frame	Baseline and Week 12

Outcome Measure Data

Analysis Population Description

ITT population.

Arm/Group Title	CB-03-01 Cream	Vehicle Cream
Arm/Group Description:	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.	Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
Overall Number of Participants Analyzed	353	355
Mean (95% Confidence Interval); Unit of Measure: percent change
	-44.8; (-49.0 to -40.5)	-36.5; (-40.8 to -32.2)

8. Other Pre-specified Outcome

Title	Local Site Reactions
Description	Local Site Reactions (LSRs) including telangiectasia, skin atrophy, striae rubrae, erythema, edema, scaling/dryness, stinging/burning, and pruritus scored by frequency and severity at every visit (Baseline, Weeks 4, 8, and 12).
Time Frame	Baseline, Weeks 4, 8, and 12

Outcome Measure Data Not Reported

Adverse Events

Time Frame	Adverse events (AEs) and serious adverse events (SAEs) were collected from screening visit, Baseline (Day 1) and up to Week 12/early termination.
Adverse Event Reporting Description	The Safety population was used for all analyses which comprised of all participants enrolled in the study and applied at least on dose of the test article.
Arm/Group Title	CB-03-01 Cream		Vehicle Cream
Arm/Group Description	CB-03-01 cream, 1% applied twice daily for 12 weeks Cortexolone 17α-propionate (USAN/INN: clascoterone) is a steroidal antiandrogen that is being developed as a 1% cream for the topical treatment of acne vulgaris.		Vehicle cream applied twice daily for 12 weeks Vehicle cream: Vehicle cream manufactured to mimic look and feel of CB-03-01 but without the active ingredient cortexolone 17α-propionate.
All-Cause Mortality
	CB-03-01 Cream		Vehicle Cream
	Affected / at Risk (%)		Affected / at Risk (%)
Total	0/353 (0.00%)		0/355 (0.00%)
Serious Adverse Events
	CB-03-01 Cream		Vehicle Cream
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/353 (0.00%)		1/355 (0.28%)
Infections and infestations
Pneumonia † 1 [1]	0/353 (0.00%)	0	1/355 (0.28%)	1
1 Term from vocabulary, MedDRA version 18.1; † Indicates events were collected by systematic assessment; [1] The SAE was deemed not related to test article.
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0.6%
	CB-03-01 Cream		Vehicle Cream
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	9/353 (2.55%)		18/355 (5.07%)
General disorders
Application pain † 1	1/353 (0.28%)	1	3/355 (0.85%)	4
Infections and infestations
Nasopharyngitis † 1	6/353 (1.70%)	7	12/355 (3.38%)	12
Injury, poisoning and procedural complications
Application site pruritus † 1	2/353 (0.57%)	2	3/355 (0.85%)	3
1 Term from vocabulary, MedDRA version 18.1; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS 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.

The Sponsor has first right to publish pooled study data. In the event that such manuscript has not been submitted for publication within 18 months from study completion/termination at all participating sites, the PI shall have the right to single center publications provided they submit any data for presentation, oral or written, to the Sponsor for review 60 days prior to public disclosure. The PI may not disclose previously undisclosed confidential information other than study results.

Results Point of Contact

• Name/Title: Cassiopea R&D
• Organization: Cassiopea SPA
• Phone: +39 02 868 911 24
• EMail: r&d@cassiopea.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Piszczatoski CR, Powell J. Topical Clascoterone: The First Novel Agent for Acne Vulgaris in 40 Years. Clin Ther. 2021 Oct;43(10):1638-1644. doi: 10.1016/j.clinthera.2021.08.007. Epub 2021 Oct 2.

Hebert A, Thiboutot D, Stein Gold L, Cartwright M, Gerloni M, Fragasso E, Mazzetti A. Efficacy and Safety of Topical Clascoterone Cream, 1%, for Treatment in Patients With Facial Acne: Two Phase 3 Randomized Clinical Trials. JAMA Dermatol. 2020 Jun 1;156(6):621-630. doi: 10.1001/jamadermatol.2020.0465.

• Responsible Party: Cassiopea SpA
• ClinicalTrials.gov Identifier: NCT02608450
• Other Study ID Numbers: CB-03-01/25
• First Submitted: November 17, 2015
• First Posted: November 18, 2015
• Results First Submitted: September 23, 2020
• Results First Posted: November 20, 2020
• Last Update Posted: November 20, 2020