About the CONTROL Study
The purpose of this Phase 2b study is to investigate the efficacy and safety of two concentrations of TMB-001 (topical isotretinoin) for the treatment of congenital ichthyosis (CI).
What is TMB-001?
Many dermatologists are familiar with oral isotretinoin. By formulating isotretinoin into a proprietary topical formulation it may be possible to reduce systemic absorption, potentially allowing for chronic use over larger areas of the body. TMB-001 is being developed for the treatment of moderate to severe subtypes of CI. In 2018, the U.S. Food & Drug Administration (FDA) awarded an Orphan Products Grant to support Phase 2a and Phase 2b clinical trials evaluating TMB-001.
Participation in this study will last approximately 12 weeks weeks and require 6 visits. Due to the global pandemic, the study has been modified to allow for most study visits to occur at a participant’s home, avoiding unnecessary travel to study centers.
This is a randomized, parallel, double-blind, vehicle-controlled study.
What does that mean? A randomized trial means that the drug received is assigned by chance (like flipping a coin). During the 12-week treatment period, eligible subjects will be randomized to one of three treatment groups. This is a double-blind trial, which means neither the participant nor study doctor will know which of the three treatment groups you are in.
This is also a vehicle-controlled study, which means one of the treatment groups is an ointment containing most of the same ingredients as the other ointments being studied, but without isotretinoin. The chances of being assigned to any of the three treatment groups are equal. This means any participant has a one in three chance of receiving either a higher dose of isotretinoin, lower dose of isotretinoin or vehicle control ointment.
If you qualify for the study, you may be eligible to receive reimbursement if you travel to and from the study center, as well as being compensated for your time.
Study participants must be 9 years of age or older and have either autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) or recessive X-linked ichthyosis (RXLI) subtypes of CI. If your subtype is not genetically confirmed, genetic testing will be provided by Timber. Participants must have a minimum of 10% of their total body surface area affected by CI at baseline and be in good general health. Women who are pregnant or planning to become pregnant may not participate in the study.