Congenital Ichthyosis and Tuberous Sclerosis Complex Clinical Trials are Now Enrolling Patients

Timber Pharmaceuticals focuses on rare diseases of dermatologic origin. These include scleroderma, congenital ichthyosis, tuberous sclerosis complex, and facial angiofibromas. They are currently working on two clinical trials.

Trial 1

The company has just announced that they have opened 11 clinical trial sites in both the United States and Australia for their most recent trial. This is a Phase 2b clinical trial called CONTROL. It is investigating TMB-001, a topical isotretinoin for patients with congenital ichthyosis (CI). CI is a group of conditions, all rare, that lead to dry and scaly skin and pose a number of symptoms.

All of these trial sites are accurately enrolling patients with moderate to severe disease. Two of the subtypes included are X-linked and lamellar ichthyosis. They have a target enrollment of 45 patients. Individuals who are nine years of age or older are eligible.

This investigation is randomized and double-blind. It will work to evaluate both the safety and the efficacy of the treatment in two different concentrations.

This therapy already received an Orphan Products Grant back in 2018 to encourage these trials. Researchers are hopeful that this therapy could bring relief to those suffering from the impaired hearing and eyesight, itching, inability to sweat, limitations in range of motion, and the high risk of infections that come with CI.

Trial 2

A second trial by this company is also currently underway. It will investigate TMB-002, a topical rapamycin for tuberous sclerosis complex (TSC). Specifically, this trial will investigate the therapy as a treatment for facial angiofibromas (FAs) associated with the disease. 70% of these trial sites are currently enrolling patients. This is also a Phase 2b investigation.

This trial has a target enrollment of 120 TSC patients. There will be 16 trial locations across the world.

Clinical Trials During a Pandemic

Operating clinical trials is not an easy feat in normal times. Amidst a global pandemic, it poses extra challenges. Researchers want to be able to open as many different trial locations as possible as quickly as possible.

Participants are eager to join this investigation, and researchers are doing everything in their power to make the process safe and efficient.

You can read more about these trials here.

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