About the ASCEND Study
The purpose of this Phase 3 study is to investigate the efficacy and safety of TMB-001 (topical isotretinoin) for the treatment of congenital ichthyosis (CI). Furthermore, to investigate an optimal dosage for maintenance therapy with TMB-001.
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 the Phase 3 clinical trial evaluating TMB-001.
Participation in this study will last approximately 12-24 weeks and require 6-9 visits to the study center.
This is a randomized, parallel, double-blind, vehicle-controlled study.
What does that mean? A randomized trial means that the study drug received is assigned by chance (like flipping a coin). This is a double-blinded trial, which means neither the participant nor study doctor will know which study treatment group 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 any participant receiving TMB-001 vs vehicle is 2:1, meaning for every 2 participants that receive TMB-001 ointment, one will receive vehicle- control ointment in the first 12 weeks.
At the end of week 12, eligible participants will be randomized again to either once a day or twice a day TMB-001 treatment for an additional 12 weeks. Patients who were initially randomized to vehicle will be eligible to receive twice a dayTMB-001 treatment from Weeks 13-24.
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 compensation for your time
Study participants must be 6 years of age or older and have either autosomal recessive congenital ichthyosis (ARCI) 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.