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Trial registered on ANZCTR


Registration number
ACTRN12617001127392
Ethics application status
Approved
Date submitted
25/07/2017
Date registered
1/08/2017
Date last updated
14/02/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
Study of BTX 1503 Solution in Patients with Acne Vulgaris
Scientific title
An Open-Label Study to Evaluate the Safety and Tolerability of BTX 1503 Solution in Patients with Acne Vulgaris
Secondary ID [1] 292521 0
BTX.2017.002
Universal Trial Number (UTN)
Nil
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acne 304165 0
Condition category
Condition code
Skin 303493 303493 0 0
Dermatological conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Approximately 16 participants with acne vulgaris will be enrolled and receive 3ml dose of BTX 1503 5% Solution twice daily.
Participants will receive their initial application of study drug on Day 1 at the clinical sites administered by the clinical site staff. Participants will be instructed in how to apply study drug when not at the clinical site. Starting on the evening of Day 1, participants will apply their study drug at home. Each application of study drug will occur at approximately the same time in the morning with the second application 12 hours later. Participants will apply study drug twice daily through Day 27. Participants will apply study drug at the clinical site on the morning of Day 14 and on the morning of Day 28 (final dose).
A diary will be maintained documenting compliance with application of the self-administered applications. Each dose of study drug will be applied to the entire face. Participants will be asked to apply the total amount of study drug per application evenly, as best as possible, to cover all the face.
Intervention code [1] 298705 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 302870 0
To evaluate the safety and tolerability of BTX 1503 5% solution for 28 days administered as a single dose twice daily (BID) in participants with acne vulgaris.
Assessed by: collection of vital signs (temperature, blood pressure and pulse), AEs, cutaneous tolerability assessments, and laboratory findings (CBC, chemistry, and urinalysis). Urine drug tests will be conducted for drugs of abuse, including the presence of tetrahydrocannabinol (THC).
Timepoint [1] 302870 0
Adverse events will be monitored from time of consent through the end of study (35 days). Cutaneous tolerability (erythema, scaling, dryness, burning/stinging, and irritant/allergic contact dermatitis) will be collected at Baseline, Day 14, Day 28, and Day 35 and graded using the following scale: 0, None; 1, Slight; 2, Moderate; 3, Severe. Vital signs (temperature, blood pressure, and pulse) will be obtained at Baseline, Day 14, Day 28 and Day 35. Complete blood count (CBC), chemistry, and urinalysis will be conducted at Baseline and at Day 28. Urine drug tests will be conducted at the Day 1, Day 28 and Day 35 Visits to evaluate for levels of THC.
Blood levels of study drug will be measured.
Pregnancy testing will be conducted for women of child-bearing potential (WOCBP) prior to treatment and at the Day 28 Visit.
Secondary outcome [1] 337271 0
The effects of treatment with BTX 1503 5% Solution on lesion counts will be evaluated by the treating dermatologist (investigator).
Assessed by: collection of inflammatory lesion counts at Baseline, Day 28, and Day 35. Photographs will be obtained at Baseline, Day 28, and Day 35.
Timepoint [1] 337271 0
The absolute and percent change from Baseline in the inflammatory lesion count at Day 28 and Day 35.
Secondary outcome [2] 337480 0
The effects of treatment with BTX 1503 5% Solution on lesion counts will be evaluated by the treating dermatologist (investigator).
Assessed by: collection of non-inflammatory lesion counts at Baseline, Day 28, and Day 35. Photographs will be obtained at Baseline, Day 28, and Day 35.
Timepoint [2] 337480 0
The absolute and percent change from Baseline in the non-inflammatory lesion count at Day 28 and Day 35.
Secondary outcome [3] 337481 0
The effects of treatment with BTX 1503 5% Solution on lesion counts will be evaluated by the treating dermatologist (investigator).
Assessed by: collection of total lesion counts.
Timepoint [3] 337481 0
The absolute and percent change from Baseline in the total lesion count at Day 28.
Secondary outcome [4] 337482 0
The effects of treatment with BTX 1503 5% Solution on Investigator Global Assessment (IGA) will be evaluated by the treating dermatologist (investigator).
An independent group of dermatologists will also review the photographs for IGA scoring. On Day 28 a Patient Reported Outcome (PRO) instrument will assess the participant’s perception of the change in their acne relative to baseline.
Timepoint [4] 337482 0
The proportion of participants with an IGA score of “clear” or “almost clear” and at least a 2-grade reduction at Day 28 and Day 35.

Eligibility
Key inclusion criteria
To be included in the study, participants with acne vulgaris must meet the following inclusion criteria.
1. Participant (or legal guardian) has the ability and willingness to sign a written informed consent.
2. Participant is of either gender between 18 and 65 years of age, inclusive.
3. Participant is in good general health without clinically significant haematological, cardiac, respiratory, renal, endocrine, gastrointestinal, psychiatric, hepatic, or malignant disease, as determined by the investigator.
4. Participant has suitable venous access for blood sampling.
5. Participant is able and willing to complete the study and to comply with all study instructions and attend the necessary visits.
6. Participant has acne vulgaris of the face defined as:
a. 20 to 50 (inclusive) inflammatory lesions on the face
b. 20 to 100 (inclusive) non-inflammatory lesions on the face
c. An Investigator Global Assessment (IGA) score for acne severity of 3 or 4 (moderate or severe) assessed on the face.
7. Participant has <= 3 nodular/cystic acne lesions (>5 mm in diameter)
8. Participant must be willing to use the facial cleanser provided (Neutrogena) throughout the study.
9. Participant must refrain from the use of other treatments for acne during the study.
10. Participant must agree to not wash their face for 4 hours after application of study medication.
11. Participant must agree that shaving cream and provided facial cleansers will not be used within 5 minutes prior to, or for 4 hours after, application of study medication.
12. Participant must agree to maintain their regular use of sunscreens, moisturisers, and facial makeup throughout the entire course of the study and not apply sunscreens, moisturisers, or facial makeup within 4 hours prior to, or 1 hour after, study drug application.
13. Participant agrees to not use marijuana products throughout the study.
14. Male participants and their partners must agree and commit to use a barrier method of contraception during the study.
15. A negative urine pregnancy test (UPT) result for all WOCBP at the Screening Visit and Baseline visit, if applicable. A WOCBP is one who is permanently sterilised or is postmenopausal. Postmenopausal is defined as 12 months with no menses without an alternative medical cause.
16. Sexually active women must agree to use:
a. One of these highly effective contraception methods - Intrauterine device (IUD); hormonal (injections, implants, transdermal patch, vaginal ring; tubal ligation; partner vasectomy, OR
b. Oral contraceptives WITH a barrier method (listed below), OR
c. Two barrier forms of contraception (listed below)
Male or female condom; diaphragm; cervical cap.
17. Male participants must refrain from sperm donation during the study treatment period until 90 days after final study drug administration.
18. Male participants must agree to keep their face clean shaven (no moustache or goatee; short sideburns acceptable) throughout the study and use the same method for shaving as was used for the 4 weeks prior to the Screening Visit.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
If a participant meets any of the following exclusion criteria, they may not participate in the study.
1. Female participant who is breast feeding, pregnant, or planning to become pregnant any time during the course of the study.
2. Participant with history of known or suspected intolerance to dimethicone containing products, or drug product excipients (hexamethyldisiloxane, polymethylsiloxane, and polypropylene glycol (PPG) 15 stearyl ether).
3. Participant has used any marijuana products, via any route, within 4 weeks prior to the Screening Visit. A urine drug test positive for THC will exclude the participant.
4. Participant has known HIV infection.
5. Participant has acne conglobata, acne fulminans, secondary acne (chloracne), drug-induced acne, pseudo-folliculitis, or severe acne requiring systemic treatment.
6. Participant has severe truncal acne.
7. Participant has excessive facial hair that would interfere with the evaluation of safety or with the diagnosis or assessment of acne vulgaris.
8. Participant has sunburns, unevenness in skin tones, tattoos, scars, excessive hair, freckles, birthmarks, moles, or other skin damage or abnormality that would result in the inability to evaluate the skin of the face.
9. Participant has any skin condition of the face other than acne vulgaris.
10. Participant started a topical medication regimen for treatment of acne vulgaris on the face within 14 days of the Screening Visit.
11. Participant has used systemic corticosteroids, oral antibiotics, anti-inflammatory drugs (NSAIDs are permitted) or a prescription topical retinoid on the face within 4 weeks prior to the Screening Visit.
12. Participant has initiated hormonal therapy or had a dose change to hormonal therapy within 12 weeks prior to the Screening Visit.
13. Participant uses hormonal therapy solely for the control of acne.
14. Participant has used androgen receptor blockers (eg, spironolactone) within 12 weeks prior to the Screening Visit.
15. Participant has used oral retinoids (eg, isotretinoin) within 24 months prior to the Screening Visit.
16. Participant has had facial procedures (eg, microdermabrasion, chemical or laser peel) within 8 weeks prior to the Screening Visit.
17. Participant has had photodynamic therapy within 8 weeks prior to the Screening Visit.
18. Participant has an underlying disease that requires the use of interfering topical or systemic therapy.
19. Participant has other dermatological conditions that require the use of interfering topical or systemic therapy or that might interfere with study assessments such as, but not limited to, atopic dermatitis, psoriasis, perioral dermatitis, or rosacea.
20. Participant has had excessive sun exposure (in the opinion of the investigator) within one week prior to the Screening Visit and an unwillingness to refrain from excessive sun exposure during the study.
21. Participant has a clinically relevant history or currently suffering from any disease or condition that, in the opinion of the investigator, may affect the evaluation of the study product or place the participant at undue risk. This may include respiratory (including chronic asthma requiring repetitive drug interventions), gastrointestinal, renal, hepatic, haematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, or connective tissue diseases or disorders.
22. Participant has a clinically relevant history of, or current evidence of, abuse of alcohol or other drugs. If the urine drug screen (UDS) at the Screening Visit is positive for any drugs of abuse, the participant is not eligible to participate. Participants may be deemed eligible if the UDS identifies participant-reported, prescribed drugs or appropriate levels of alcohol, as determined by the investigator.
23. Participant has used systemic or other immunosuppressive medications within 4 weeks of the Screening Visit (inhaled corticosteroid <= 1000 µg daily dose is acceptable).
24. Participant is currently using any medication that, in the opinion of the investigator, may affect the evaluation of the study product or place the participant at undue risk.
25. Participant has participated in another investigational drug or device research study within 30 days of the Screening Visit or five half-lives of the drug, whichever is longer.
26. Any other reason that would make the participant, in the opinion of the investigator or sponsor, unsuitable for the study

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
This is an open-label, multi-centre, single-arm, Phase 1b study. All participants will receive 3 mL of study drug (BTX 1503 5% Solution) applied to the entire face. No escalation of dose will occur. There are no pre-planned dose adjustments, modifications, or delays. Participants will receive BID application of study drug for 27 days with a final application on the morning of Day 28 for a total of 55 doses.
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
All statistical processing will be performed using SAS® unless otherwise stated.
Safety Analyses
All participants who receive at least one confirmed dose of study drug, and have at least one post-baseline assessment will be included in the safety analyses.
All treatment-emergent adverse events (TEAEs) occurring during the study will be recorded and classified based on MedDRA terminology. Treatment-emergent adverse events are those AEs with an onset on or after the first application of study medication. All reported TEAEs will be summarised by treatment group, the number of participants reporting events, system organ class, preferred term, severity, relationship to study drug, and seriousness. When summarising events by causality and severity, each participant will be counted only once within a system organ class or a preferred term by using the event with the greatest relationship and highest severity within each classification.
Serious adverse events (SAEs) will be summarised by cohort, system organ class, preferred term, severity, outcome and relationship to study drug; and all SAEs will be listed by participant. In addition, a list of participants who prematurely discontinue from the study due to an AE and the reason for discontinuation will be provided.
Concomitant medication will be mapped to ATC Level 2 using the WHODrug dictionary. The number and percentage of participants reporting each medication will be summarised. Medications taken by each participant will be listed.
Cutaneous tolerability scores for each parameter (erythema, scaling, dryness, burning/stinging, and irritant/allergic contact dermatitis) will be summarised for each visit. In addition, the change from baseline in the mean scores will be summarised for each visit.
Exploratory Analyses:
Lesion counts will be collected by the clinical site along with photographs of the participant’s face. Inflammatory and non-inflammatory lesion counts will be made separately. The IGA will be conducted by the study investigator at each site. Each participant will have the IGA done by the same investigator throughout the study. IGA will also be assessed by the central panel of reviewers.
Demographics will be summarised by age, gender, race, ethnicity height and weight. Summary statistics will be prepared for the change from baseline in lesion counts (inflammatory and non-inflammatory separate and combined) and IGA separately for the investigators and the central panel (IGA only). For continuous variables, the mean, standard deviation (SD), median, and range will be presented along with the 95% confidence interval (CI). Categorical variables will be summarised by proportions along with the 95% CI.
Lesion counts, changes in lesion counts, and IGA scores will be presented by reviewer (clinical site or central panel [IGA Only]) and concurrence will be assessed.
The following exploratory analyses will be conducted.
• The absolute and percent change from Baseline in the inflammatory lesion count at Day 28 and Day 35
• The absolute and percent change from Baseline in the non-inflammatory lesion count at Day 28 and Day 35
• The absolute and percent change from Baseline in the total lesion count at Day 28 and Day 35
• The proportion of participants with an IGA score of “clear” or “almost clear” and at least a 2-grade reduction at Day 28 and Day 35
Participant’s assessment of the change in their acne from baseline to Day 28 using the PRO assessment will be summarized.
Drug Levels:
Blood levels of study drug will be summarised by time point. The mean, SD, median and range will be presented.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
10/10/2017
Actual
24/10/2017
Date of last participant enrolment
Anticipated
31/10/2017
Actual
8/12/2017
Date of last data collection
Anticipated
Actual
18/01/2018
Sample size
Target
16
Accrual to date
Final
21
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 8609 0
Veracity Clinical Research - Woolloongabba
Recruitment hospital [2] 8610 0
Sinclair Dermatology - East Melbourne
Recruitment hospital [3] 8658 0
The Skin Centre - Benowa
Recruitment hospital [4] 10011 0
Fremantle Dermatology - Fremantle
Recruitment postcode(s) [1] 16717 0
4102 - Woolloongabba
Recruitment postcode(s) [2] 16718 0
3002 - East Melbourne
Recruitment postcode(s) [3] 16766 0
4217 - Benowa
Recruitment postcode(s) [4] 19337 0
6160 - Fremantle

Funding & Sponsors
Funding source category [1] 297090 0
Commercial sector/Industry
Name [1] 297090 0
Botanix Pharmaceuticals Ltd
Country [1] 297090 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Botanix Pharmaceuticals Ltd
Address
68 Aberdeen Street
Northbridge, WA 6006, Australia
Country
Australia
Secondary sponsor category [1] 296098 0
None
Name [1] 296098 0
Not Applicable
Address [1] 296098 0
Not Applicable
Country [1] 296098 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 298268 0
Bellberry Limited
Ethics committee address [1] 298268 0
Ethics committee country [1] 298268 0
Australia
Date submitted for ethics approval [1] 298268 0
12/07/2017
Approval date [1] 298268 0
16/08/2017
Ethics approval number [1] 298268 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 76534 0
Dr Lynda J Spelman
Address 76534 0
Veracity Clinical Research Pty Ltd. Suite 18, Level 1, 250 Ipswich Road, Woolloongabba, QLD 4102
Country 76534 0
Australia
Phone 76534 0
+61 7 3039 1311
Fax 76534 0
+61 7 3391 6239
Email 76534 0
spelchat@iinet.net.au
Contact person for public queries
Name 76535 0
Michael Thurn
Address 76535 0
Chief Operating Officer
Botanix Pharmaceuticals Limited
68 Aberdeen Street, Northbridge WA 6003
Country 76535 0
Australia
Phone 76535 0
+61 2 6362 7663
Fax 76535 0
Email 76535 0
mthurn@botanixpharma.com
Contact person for scientific queries
Name 76536 0
Mark Davis
Address 76536 0
Botanix Pharmaceuticals Limited
VP Clinical and Regulatory
610 W. Germantown Pike, Suite 400, Plymouth Meeting, PA 19462
Country 76536 0
United States of America
Phone 76536 0
+1 925 3361055
Fax 76536 0
Email 76536 0
mdavis@botanixpharma.com

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