What is the mechanism of action of this treatment?

Common treatments for Ichthyosis, such as TMB-001 0.05% ointment, primarily focus on moisturizing and exfoliating the skin to reduce scaling and improve skin barrier function. These treatments often contain keratolytic agents like urea, lactic acid, or salicylic acid, which help to break down the excessive keratin in the skin, promoting exfoliation and reducing the thickness of the scales. Emollients and moisturizers are also crucial as they hydrate the skin, making it more pliable and less prone to cracking. For Ichthyosis patients, these mechanisms are vital as they directly address the primary symptoms of dryness and scaling, improving skin appearance and comfort, and preventing secondary complications such as infections.

What side effects are associated with this type of intervention?

Common side effects of topical treatments for congenital ichthyosis, such as TMB-001 0.05% ointment, include skin irritation, redness, and dryness. Some patients may experience itching or a burning sensation upon application. In rare cases, allergic reactions can occur, leading to more severe skin reactions. It is important to monitor for these side effects and consult a healthcare provider if they persist or worsen.

What prior FDA approvals exist?

There is limited specific information on prior FDA approvals for topical treatments of congenital ichthyosis similar to TMB-001 0.05% ointment. Generally, treatments for ichthyosis include topical keratolytics, emollients, and retinoids to manage symptoms. Commonly used topical agents include urea, lactic acid, and salicylic acid, which help to exfoliate and moisturize the skin. Systemic retinoids like acitretin have also been used for severe cases. Further research and clinical trials, such as the one for TMB-001, are ongoing to find more effective treatments.

What other types of research exist?

Promising novel alternatives to TMB-001 0.05% ointment for treating congenital ichthyosis include: 1) Genetically modified autologous cultured skin grafts, which have shown stable and complete epidermal regeneration in patients with generalized junctional epidermolysis bullosa. 2) Beremagene geperpavec (B-VEC), a topical gene therapy delivering a functional version of the COL7A1 gene, which has demonstrated significant wound healing in dystrophic epidermolysis bullosa. 3) Protein replacement therapy using recombinant collagen type VII, which has shown potential in preclinical studies to restore the dermoepidermal junction and reduce blistering in animal models. These therapies represent advanced approaches targeting the genetic and protein deficiencies underlying ichthyosis.

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TMB-001 Ointment for Ichthyosis (ASCEND Trial)

Phase 3

Waitlist Available

Research Sponsored by Timber Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Documented history of moderate to severe disease at Screening

Subject has clinical diagnosis of CI and has a genetic confirmation of either ARCI or RXLI subtypes of CI

Must not have

Subject is immunosuppressed or receives systemic immunotherapy

Subject has lesions suspicious for skin cancer or untreated skin cancers within the Treatment Areas

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 weeks

Awards & highlights

Pivotal Trial

Summary

This trial tests a skin ointment called TMB-001 for people with a chronic skin condition called Congenital Ichthyosis. The ointment helps by reducing excess skin production and promoting shedding. The study aims to see if TMB-001 is safe and effective for these patients.

Who is the study for?

This trial is for males and females aged 6 and older with a clinical diagnosis of Congenital Ichthyosis (CI), specifically RXLI or ARCI subtypes, confirmed by genetics. Participants must have significant skin scaling and be in good health. Women who can bear children must use two forms of birth control. Those pregnant, lactating, or planning pregnancy; using certain treatments like UV therapy or systemic immunosuppressants; with untreated infections; drug/alcohol abuse history; on other trials; failed retinoid therapies; liver/kidney issues are excluded.

What is being tested?

The study tests TMB-001 ointment's safety and effectiveness against CI compared to a placebo (vehicle). It has three phases over 24 weeks: an initial induction period with once-daily treatment, increased to twice daily during the main treatment phase, followed by maintenance where dosing frequency may vary based on response. An optional arm studies systemic exposure in those with extensive affected skin.

What are the potential side effects?

Potential side effects aren't specified but could include reactions at the application site such as redness, itching, or irritation given it's a topical medication. Systemic side effects might occur if absorption through the skin happens but would depend on individual patient factors.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My condition was moderate to severe at my last check-up.

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I have been diagnosed with congenital ichthyosis and confirmed to have ARCI or RXLI.

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I am a woman who is either postmenopausal, cannot have children, or I use two forms of birth control.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am on immunotherapy or have a weakened immune system.

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I have suspicious or untreated skin lesions in the areas to be treated.

Select...

I have been taking immunosuppressive drugs, including steroids, within the last 2 weeks.

Select...

I have infections that haven't been treated yet.

Select...

I have tried retinoid therapy for my condition without success.

Select...

I am able to communicate and cooperate with my doctor without any issues.

Select...

I have a genetic condition related to a specific type of severe skin disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in Investigator global assessment (IGA) score

Secondary study objectives

Change in Visual Index of Ichthyosis Severity score

To investigate the proportion of subjects experiencing local skin reactions with topically applied TMB-001 0.05% ointment.

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: TMB-001 0.05%Experimental Treatment1 Intervention

TMB-001 0.05% ointment: Induction phase QD for 3 weeks, followed by BID for 9 weeks. Maintenance therapy for additional 12 weeks randomized QD vs BID

Group II: Maximal useExperimental Treatment1 Intervention

Optional Parallel Arm to evaluate the systemic exposure and safety of TMB-001 0.05% under conditions of maximal use.

Group III: VehiclePlacebo Group1 Intervention

Matching vehicle ointment with no action isotretinoin: Induction phase QD for 3 weeks, followed by BID for 9 weeks. Cross over to 12 weeks TMB-001 0.05% BID.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

TMB-001

2022

Completed Phase 3

~210

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Ichthyosis, such as TMB-001 0.05% ointment, primarily focus on moisturizing and exfoliating the skin to reduce scaling and improve skin barrier function. These treatments often contain keratolytic agents like urea, lactic acid, or salicylic acid, which help to break down the excessive keratin in the skin, promoting exfoliation and reducing the thickness of the scales. Emollients and moisturizers are also crucial as they hydrate the skin, making it more pliable and less prone to cracking. For Ichthyosis patients, these mechanisms are vital as they directly address the primary symptoms of dryness and scaling, improving skin appearance and comfort, and preventing secondary complications such as infections.