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Trial registered on ANZCTR


Registration number
ACTRN12618001047280
Ethics application status
Approved
Date submitted
22/05/2018
Date registered
22/06/2018
Date last updated
28/02/2020
Date data sharing statement initially provided
2/11/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
A Randomized, Double-Blind, Vehicle-Controlled Study to Evaluate the Safety and Efficacy of BTX 1503 in Patients with Moderate to Severe Acne Vulgaris
Scientific title
A Randomized, Double-Blind, Vehicle-Controlled Study to Evaluate the Safety and Efficacy of BTX 1503 in Patients with Moderate to Severe Acne Vulgaris
Secondary ID [1] 294815 0
None
Universal Trial Number (UTN)
U1111-1213-5813
Trial acronym
None
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acne vulgaris 307747 0
Condition category
Condition code
Skin 306802 306802 0 0
Dermatological conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Participants will be randomised to one of five treatment groups:
- BTX 1503 5% applied topically twice daily for 84 days
• BTX 1503 5% applied topically once daily for 84 days
• BTX 1503 2.5% applied topically once daily for 84 days
Participants will be provided with a kit containing 4 pumps of investigational product. Each pump will contain enough doses for seven days of dosing. Participants will be asked to record their doses in a diary and study personnel will perform drug accountability at each study visit.
Intervention code [1] 301129 0
Treatment: Drugs
Comparator / control treatment
-Vehicle comparator applied topically twice daily for 84 days
-Vehicle comparator applied topically once daily for 84 days
The vehicle comparator contains five excipients also used in the active study drug, which have a history of use in topical products.
Control group
Placebo

Outcomes
Primary outcome [1] 305790 0
Absolute change from Baseline in inflammatory lesion counts
The counts will be performed by a dermatologist or other trained study investigator.
Timepoint [1] 305790 0
Day 84 post-baseline visit
Secondary outcome [1] 346548 0
Absolute change from Baseline in non-inflammatory lesion counts
The counts will be performed by a dermatologist or other trained study investigator.
Timepoint [1] 346548 0
Day 84 post-first baseline viit
Secondary outcome [2] 346549 0
The percent change from Baseline in the inflammatory lesion counts.
The counts will be performed by a dermatologist or other trained study investigator
Timepoint [2] 346549 0
Day 84 post-baseline visit
Secondary outcome [3] 346550 0
The percent change from Baseline in the non-inflammatory lesion counts.
The counts will be performed by a dermatologist or other trained study investigator.
Timepoint [3] 346550 0
Day 84 post-baseline
Secondary outcome [4] 346551 0
The proportion of subjects with at least a 2-grade reduction from the Baseline IGA score
Timepoint [4] 346551 0
Day 84 post-baseline
Secondary outcome [5] 346552 0
The proportion of subjects with an IGA score of “clear” or “almost clear”.
Timepoint [5] 346552 0
Day 84 post-baseline
Secondary outcome [6] 346553 0
Adverse events (AEs) reported from time of consent/assent through the end of the study.
Known adverse events include mild irritation, dryness or itchiness at the site of application. Participants will be questioned at each outpatient visit as to whether there has been any changes to their health.
Timepoint [6] 346553 0
Adverse events will be monitored throughout the study, from the time of consent up to and including Day 84
Secondary outcome [7] 347656 0
Changes in the haematological status through complete blood counts (red cells, white cells (with differential), and platelets).
Timepoint [7] 347656 0
At baseline and at Day 84 post-baseline
Secondary outcome [8] 348486 0
Presence of urinary tract infection through analysis of urine (e.g. color, clarity, specific gravity, glucose, protein, and pH).
Timepoint [8] 348486 0
At baseline and at Day 84 post-baseline
Secondary outcome [9] 348488 0
Changes in glucose through analysis of blood chemistry.
Timepoint [9] 348488 0
At baseline and at Day 84 post-baseline
Secondary outcome [10] 348514 0
Changes in electrolytes through analysis of blood chemistry.
Timepoint [10] 348514 0
At baseline and at Day 84 post-baseline
Secondary outcome [11] 348515 0
Changes in renal function through analysis of blood chemistry.
Timepoint [11] 348515 0
At baseline and at Day 84 post-baseline
Secondary outcome [12] 348516 0
Changes in liver function through analysis of blood chemistry.
Timepoint [12] 348516 0
At baseline and at Day 84 post-baseline
Secondary outcome [13] 348517 0
Changes in renal function through analysis of urine (e.g. color, clarity, specific gravity, glucose, protein, and pH).
Timepoint [13] 348517 0
At baseline and at Day 84 post-baseline
Secondary outcome [14] 348518 0
Presence of metabolic disorders through analysis of urine (e.g. color, clarity, specific gravity, glucose, protein, and pH).
Timepoint [14] 348518 0
At baseline and at Day 84 post-baseline

Eligibility
Key inclusion criteria
To be included in the study, subjects with acne vulgaris must meet the following inclusion criteria.
- Subject is of either gender and 12 to 40 years of age.
- Subject is in good general health without clinically significant haematological, cardiac, respiratory, renal, endocrine, gastrointestinal, psychiatric, hepatic, or malignant disease, as determined by the investigator.
- Subject is able and willing to complete the study and to comply with all study instructions and attend the necessary visits.
-Subject 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.
-Subject has less than or <3 nodular/cystic acne lesions (>5 mm in diameter).
-Subject must refrain from the use of other treatments for acne during the study.
-Subject must agree to not wash or shave their face, swim or otherwise get their face wet for at least 1 hour after application of study medication.
-Subject must agree to maintain their regular use of sunscreens, moisturizers, shaving cream, and facial make up throughout the entire course of the study.
- Sexually active women of childbearing potential must use appropriate birth control methods.
Minimum age
12 Years
Maximum age
40 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
-People who would otherwise qualify for the study but are living in the same household as a study subject, are not allowed to participate in the study.
-Female subject who is breast feeding, pregnant, or planning to become pregnant any time during the course of the study.
-Subject has acne conglobata, acne fulminans, secondary acne (chloracne), pseudo-folliculitis, severe acne requiring systemic treatment, or is taking a medication known to induce or exacerbate acne.
-Subject has severe truncal acne.
-Subject has excessive facial hair that would interfere with the evaluation of safety or with the diagnosis or assessment of acne vulgaris.
-Subject 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.
-Subject has any skin condition of the face other than acne vulgaris.
-Subject has used oral retinoid (e.g. isotretinoin) within 6 months (180 days) prior to the
-Subject has used Vitamin A supplements greater than 10,000 units/day within 6 months (180 days) prior to the Baseline Visit.
-Subject has used androgen receptor blockers (such as spironolactone or flutamide) within 3 months (90 days) prior to the Baseline Visit.
-Subject has initiated treatment with hormonal therapy or changed dosing with hormonal therapy within 3 months (90 days) prior to the Baseline Visit. Note: Dose and frequency of any hormonal therapy started more than 3 months (90 days) prior to the Baseline Visit must remain unchanged throughout the study. Hormonal therapies include, but are not limited to, anabolic steroids and birth control pills. Subjects taking testosterone therapy are excluded from participation.
-Subject has had facial procedures (chemical or laser peel, microdermabrasion, etc.) within 8 weeks (56 days) prior to the Baseline Visit.
-Subject has had treatment with systemic antibiotics within 4 weeks (28 days) prior to the Baseline Visit.
-Subject has had treatment with systemic anti-acne drugs within 4 weeks (28 days) prior to the Baseline Visit.
-Subject has had treatment with systemic anti-inflammatory drugs within 4 weeks (28 days) prior to the Baseline Visit.
Note: Occasional non-steroidal anti-inflammatory drugs (NSAIDs) and aspirin use on an as-needed basis, and if not used consecutively for >14 days prior to the Baseline Visit, is acceptable and does not require washout. Subjects receiving intranasal and inhaled corticosteroids may participate. Low dose (81mg) aspirin taken daily is acceptable.
-Subject has had treatment with systemic (oral) corticosteroids other immunosuppressive medications within 4 weeks (28 days) prior to the Baseline Visit.
-Subject has had treatment with prescription topical retinoid use on the face (e.g. tretinoin, tazarotene) within 4 weeks (28 days) prior to the Baseline Visit.
-Subject has had treatment with topical prescription antibiotics (e.g. dapsone, clindamycin, erythromycin, or sulfacetamide) or combination products that include a topical antibiotic within 2 weeks (14 days) prior to the Baseline Visit.
-Subject has had treatment with over-the-counter (OTC) topical medications for the treatment of acne vulgaris including benzoyl peroxide, topical anti-inflammatory medications, corticosteroids, adapalene, a-hydroxy/glycolic acid on the face within 2 weeks (14 days) prior to the Baseline Visit.
-Subject has had photodynamic therapy within 8 weeks (56 days) prior to the Baseline Visit.
- Subject has used a tanning bed within 2 weeks (14 days) prior to the Baseline Visit.
- Subject has used home-based light treatment within 2 weeks (14 days) prior to the Baseline Visit.
-Subject 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.
-Subject has had excessive sun exposure (in the opinion of the investigator) within one week prior to the Baseline Visit and an unwillingness to refrain from excessive sun exposure during the study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by computer.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software (i.e. computerised sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
This study will be evaluated using 3 analysis sets: intent-to-treat (ITT), per protocol (PP), and safety. Efficacy conclusions will be drawn from the ITT analysis set. The PP analysis set will be used to support the efficacy findings in the ITT analyses. Safety conclusions will be drawn from the safety analysis set.
Demographics will be summarized using the safety analysis dataset by age, gender, race, ethnicity, height and weight. The primary efficacy analysis will be conducted on the ITT population using last observation carried forward (LOCF) for missing data. 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 summarized by proportions along with the 95% CI.
Vehicle QD and Vehicle BID groups may be combined for analyses.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
29/06/2018
Actual
29/06/2018
Date of last participant enrolment
Anticipated
30/04/2019
Actual
15/05/2019
Date of last data collection
Anticipated
28/07/2019
Actual
23/08/2019
Sample size
Target
360
Accrual to date
Final
386
Recruitment in Australia
Recruitment state(s)
SA,WA,VIC
Recruitment hospital [1] 10857 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment hospital [2] 10858 0
Sinclair Dermatology - East Melbourne
Recruitment hospital [3] 10859 0
Fremantle Dermatology - Fremantle
Recruitment hospital [4] 12336 0
Skin and Cancer Foundation - Carlton
Recruitment hospital [5] 12337 0
St George Dermatology & Skin Cancer Centre - Kograh
Recruitment hospital [6] 12338 0
Veracity Clinical Research - Woolloongabba
Recruitment hospital [7] 12339 0
The Skin Centre - Benowa
Recruitment hospital [8] 12340 0
North Eastern Health Specialists - Hectorville
Recruitment hospital [9] 12341 0
Woden Dermatology - Phillip
Recruitment hospital [10] 12342 0
Burswood Dermatology - Burswood
Recruitment hospital [11] 13091 0
Captain Stirling Medical Centre - Nedlands
Recruitment postcode(s) [1] 22601 0
5000 - Adelaide
Recruitment postcode(s) [2] 22602 0
3002 - East Melbourne
Recruitment postcode(s) [3] 22603 0
6160 - Fremantle
Recruitment postcode(s) [4] 24577 0
3053 - Carlton
Recruitment postcode(s) [5] 24578 0
2217 - Kograh
Recruitment postcode(s) [6] 24579 0
4102 - Woolloongabba
Recruitment postcode(s) [7] 24580 0
4217 - Benowa
Recruitment postcode(s) [8] 24581 0
5073 - Hectorville
Recruitment postcode(s) [9] 24582 0
2606 - Phillip
Recruitment postcode(s) [10] 24583 0
6100 - Burswood
Recruitment postcode(s) [11] 25601 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 10381 0
United States of America
State/province [1] 10381 0

Funding & Sponsors
Funding source category [1] 299413 0
Commercial sector/Industry
Name [1] 299413 0
Botanix Pharmaceuticals Limited
Country [1] 299413 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Botanix Pharmaceuticals Ltd
Address
68 Aberdeen Street
Northbridge WA 6003
Australia
Country
Australia
Secondary sponsor category [1] 298698 0
None
Name [1] 298698 0
Address [1] 298698 0
Country [1] 298698 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 300313 0
Bellberry HREC
Ethics committee address [1] 300313 0
123 Glen Osmond Road Eastwood South Australia 5063
Ethics committee country [1] 300313 0
Australia
Date submitted for ethics approval [1] 300313 0
24/04/2018
Approval date [1] 300313 0
12/06/2018
Ethics approval number [1] 300313 0

Summary
Brief summary
BTX 1503 contains the active pharmaceutical ingredient, cannabidiol in a topical liquid formulation, and is being developed for the treatment of acne vulgaris by Botanix Pharmaceuticals Limited. CBD is a member of a broader family of compounds known as cannabinoids, a class of compounds originally derived from the cannabis sativa plant. CBD is chemically synthesized under Good Manufacture Practices (GMP) for use in this study. The objective of this study is to assess safety and efficacy of various doses of BTX 1503
in subjects with moderate to severe acne vulgaris of the face.

This will be a multi-center, randomized, double-blinded, vehicle-controlled, parallel group, dose-finding study in pediatrics, adolescents and adults (aged 12 to 40 years). Study participants will be enrolled at sites in Australia and United States of America. Not all sites will enrol paediatric participants.

Participants will be randomised to one of five treatment arms: BTX 1503 5%, BID:BTX 1503 5%, QD:BTX 1503, 2.5% QD:Vehicle, BID:Vehicle QD) with 90 subjects in each BTX 1503 group and 45 subjects in each vehicle group for a total of 360 subjects. Participants will be provided with instructions for dose administration and will be asked to self-administer for 28 consecutive days. Safety and cutaneous tolerability will be assessed by the collection and review of AEs and application site review, laboratory parameters throughout the duration of the trial. Efficacy measurements will include change in inflammatory and non-inflammatory lesion counts from baseline. Photography of the face will be conducted for some participants..

Participants will remain in follow up until 84 days following the first application of investigational product, with outpatient visits at Days 1, 14, 28, 56 and 84.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 83258 0
Dr Catherine Reid
Address 83258 0
CMAX Clinical Research Pty Ltd
Level 5, 18a North Terrace
Adelaide SA 5000
Country 83258 0
Australia
Phone 83258 0
+61 8 7088 7900
Fax 83258 0
+61 8 7088 7999
Email 83258 0
[email protected]
Contact person for public queries
Name 83259 0
Michael Thurn
Address 83259 0
Botanix Pharmaceuticals Ltd
68 Aberdeen Street
Northbridge WA 6003
Australia
Country 83259 0
Australia
Phone 83259 0
+61 (0) 403 192 615
Fax 83259 0
Email 83259 0
[email protected]
Contact person for scientific queries
Name 83260 0
Mark Davis
Address 83260 0
Botanix Pharmaceuticals Limited
VP Clinical and Regulatory
610 W. Germantown Pike, Suite 400, Plymouth Meeting, PA 19462
Country 83260 0
United States of America
Phone 83260 0
+1 925 3361055
Fax 83260 0
Email 83260 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.