Trial Review

The ANZCTR website is back online for trial registration and updates. We apologise for any inconvenience caused while the site was inactive.


With activity expected to increase on the ANZCTR again, there may be extended wait times while we process pending studies, with priority being given to those trials submitted in February. Thank you for your patience.


Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements.
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04154293




Registration number
NCT04154293
Ethics application status
Date submitted
1/11/2019
Date registered
6/11/2019
Date last updated
25/08/2022

Titles & IDs
Public title
A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
Scientific title
A Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital Ichthyosis
Secondary ID [1] 0 0
1R01FD006079-01A1
Secondary ID [2] 0 0
235-9051-202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Ichthyosis 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Isotretinoin
Other interventions - Vehicle

Placebo comparator: Vehicle Ointment (Control) - Topical, BID (Twice daily)

Experimental: TMB-001 Ointment, 0.05% - Topical, BID ( twice daily)

Experimental: TMB-001 Ointment, 0.1% - Topical, BID (Twice daily)


Treatment: Drugs: Isotretinoin
Topical Isotretinoin ointment

Other interventions: Vehicle
Topical Vehicle Ointment
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
Assessment method [1] 0 0
Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment
Assessment method [1] 0 0
Number of subjects with Investigator Global Assessment decrease in disease severity by at least 2 grades
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12
Assessment method [2] 0 0
Mean numeric Change from baseline in Itch-Numeric Rating Scale (I-NRS) at Week 12 for entire population. I-NRS is an 11-point visual analogue scale scored based upon 0 being no itch and 10 being worst itch imaginable. Subjects were asked to grade their itch on such a scale at each visit where I-NRS was administered. A 4-point change from baseline is considered clinically significant
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12
Assessment method [3] 0 0
Change from Baseline in Dermatology Life Quality Index (DLQI) at Week 12 in all adult subjects. The DLQI is validated in all subjects over age 16 years and is a 10-question questionnaire which requests information on how the dermatological condition affects different aspects of their life. Each question is scored on a 0-3 scale (0=no impact, 3=major impact) for a total possible maximal score of 30. Scores over 11 in adults are considered to indicate moderate impairment of quality of life due to the skin disease. A 4-point improvement from baseline is considered clinically significant
Timepoint [3] 0 0
12 weeks

Eligibility
Key inclusion criteria
* Subject is male or female, 9 years of age or older at Visit 2 (Baseline)
* Subject has provided written informed consent
* Females of child bearing potential must be surgically sterile or agree to 2 forms of birth control
* Subject has clinical diagnosis of Congenital Ichthyosis with genetic confirmation of subtype
* Subject has between 10% and 90% total BSA affected by Congenital Ichthyosis
* Subject has at least 2 VIIS assessment areas with a scaling score of 3 or greater
Minimum age
9 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Subject has inflammatory skin diseases unrelated to ichthyosis
* Subject has used other prohibited topical treatments in the assessment areas within certain days from baseline
* Subject has used systemic retinoids within12 weeks of baseline
* Subject has untreated secondary infections
* Subject has lesions suspicious for skin cancer or untreated skin cancers

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/08/2021
Sample size
Target
Accrual to date
Final
34
Recruitment in Australia
Recruitment state(s)
QLD,SydneyVIC
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [2] 0 0
Premier Specialists, The church - Kogarah
Recruitment hospital [3] 0 0
Murdoch Children's Research Institute, RCH - Melbourne
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
NSW 2217 - Kogarah
Recruitment postcode(s) [3] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Louisiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Timber Pharmaceuticals Inc.
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Alan M Mendelsohn, MD
Address 0 0
Timber Pharmaceuticals
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT04154293