A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
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
2 other identifiers
interventional
34
2 countries
11
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 Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Dec 2019
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 1, 2019
CompletedFirst Posted
Study publicly available on registry
November 6, 2019
CompletedStudy Start
First participant enrolled
December 3, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 20, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
August 30, 2021
CompletedResults Posted
Study results publicly available
August 25, 2022
CompletedAugust 25, 2022
August 1, 2022
1.7 years
November 1, 2019
July 10, 2022
August 3, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
12 weeks
Secondary Outcomes (3)
Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment
12 weeks
Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12
12 weeks
Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12
12 weeks
Study Arms (3)
Vehicle Ointment (Control)
PLACEBO COMPARATORTopical, BID (Twice daily)
TMB-001 Ointment, 0.05%
EXPERIMENTALTopical, BID ( twice daily)
TMB-001 Ointment, 0.1%
EXPERIMENTALTopical, BID (Twice daily)
Interventions
Topical Isotretinoin ointment
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Medical Dermatology Specialists
Phoenix, Arizona, 85006, United States
Stanford University School of Medicine
Palo Alto, California, 94304, United States
Yale Center for Clinical Investigation
New Haven, Connecticut, 06510, United States
The Indiana Clinical Trials Center
Plainfield, Indiana, 46168, United States
Austin Institute for Clinical Research, Inc
Metairie, Louisiana, 70005, United States
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19140, United States
Queensland Children's Hospital
Brisbane, Queensland, 4101, Australia
Premier Specialists, The church
Kogarah, Sydney, NSW 2217, Australia
Murdoch Children's Research Institute, RCH
Melbourne, Victoria, 3052, Australia
Related Publications (1)
Marathe K, Teng JMC, Guenthner S, Bunick CG, Kempers S, Eads K, Castelo-Soccio L, Mendelsohn AM, Raiz J, Murrell DF. Topical Isotretinoin (TMB-001) Treatment for 12 Weeks Did Not Result in Clinically Relevant Laboratory Abnormalities in Participants with Congenital Ichthyosis in the Phase 2b CONTROL Study. Dermatol Ther (Heidelb). 2023 Jun;13(6):1255-1264. doi: 10.1007/s13555-023-00923-1. Epub 2023 May 11.
PMID: 37170057DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
small sample size. study not powered for statistical significance.
Results Point of Contact
- Title
- Alan Mendelsohn, MD
- Organization
- Timber Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Alan M Mendelsohn, MD
Timber Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- Double-blind
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 2019
First Posted
November 6, 2019
Study Start
December 3, 2019
Primary Completion
August 20, 2021
Study Completion
August 30, 2021
Last Updated
August 25, 2022
Results First Posted
August 25, 2022
Record last verified: 2022-08