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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04154293
Ethics application status
Date submitted
1/11/2019
Date registered
6/11/2019
Date last updated
27/10/2020

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
Reduction in targeted Ichthyosis severity - Proportion 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
Reduction in overall Ichthyosis severity - Investigator Global Assessment decrease in disease severity by at least 2 grades
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Patient Reported Outcomes - Change in Itch-Numeric Rating Scale
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Patient Reported Outcomes - Change in Dermatology Life Quality Index
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
Gender
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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 Pharmceuticals LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to investigate the efficacy and safety of two concentrations of
topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1%
isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis
(CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital
ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.

Funding Source FDA-OOPD
Trial website
https://clinicaltrials.gov/show/NCT04154293
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Jessica Raiz, MPH
Address 0 0
Country 0 0
Phone 0 0
+19732149208
Fax 0 0
Email 0 0
jraiz@timberpharma.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04154293