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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
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
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12
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.
Address
Country

Ethics approval
Ethics application status

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

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




Results publications and other study-related documents

No documents have been uploaded by study researchers.

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