TMB-001 Ointment for Ichthyosis

(ASCEND Trial)

Recruiting at 34 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Timber Pharmaceuticals Inc.

Must not be taking: Retinoids, Immunosuppressants

Disqualifiers: Pregnancy, Inflammatory skin diseases, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 9 weeks

209 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that you stop using certain topical treatments and therapies in the treatment areas before starting the study. Specifically, you must not use topical prescription or over-the-counter therapies (except emollients, keratolytics, and topical steroids) within 2 weeks before the study begins, and you must avoid keratolytics or topical corticosteroids within 5 days before starting. Additionally, you should not have used oral isotretinoin in the past 12 months or ultraviolet treatment within 4 weeks before the study.

What data supports the effectiveness of the drug TMB-001 Ointment for Ichthyosis?

The Phase 2b CONTROL study suggests that TMB-001, a topical form of isotretinoin, did not cause significant lab changes in patients with congenital ichthyosis, indicating it may be a safer alternative to oral retinoids, which are known to be effective but can have serious side effects. ^{1 2 3 4 5}

Is TMB-001 ointment safe for humans?

In a study, TMB-001 ointment was used for 12 weeks in people with congenital ichthyosis and did not cause any significant changes in lab tests, suggesting it is generally safe. ^{1 2 4 6 7}

Eligibility Criteria

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.

Inclusion Criteria

I am 6 years old or older.

Subject and parent/guardian (if applicable) are willing and able to apply the study treatment(s) as directed, comply with study instructions, and commit to all follow-up visits for the duration of the study

Your baseline assessment must show that specific areas have a score of 3 or higher.

Exclusion Criteria

I have a skin or physical condition that could affect the study's safety or results.

Your liver or kidney function tests are significantly higher than normal.

I am on immunotherapy or have a weakened immune system.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction

Eligible subjects are randomized to TMB-001 0.05% or Vehicle treatment once daily for 3 weeks with mandatory use of a bland emollient

3 weeks

Treatment

Subjects receive TMB-001 0.05% or Vehicle treatment twice daily for 9 weeks

9 weeks

Maintenance

Eligible subjects in the TMB-001 group are randomized to open-label treatment with TMB-001 0.05% BID or QD for 12 weeks

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

Treatment Details

Interventions

• TMB-001

Trial Overview 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.

Participant Groups

Group I: TMB-001 0.05% Experimental Treatment 1 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 use Experimental Treatment 1 Intervention

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

Group III: Vehicle Placebo Group 1 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.

Who Is Running the Clinical Trial?

Timber Pharmaceuticals Inc.

Lead Sponsor

Trials

2

Recruited

240+

Timber Pharmceuticals LLC

Lead Sponsor

Trials

2

Recruited

240+

LEO Pharma

Industry Sponsor

Trials

275

Recruited

189,000+

Christophe Bourdon

LEO Pharma

Chief Executive Officer since 2022

Engineering degree from Université de Technologie de Compiègne

Dr. Siri Torhaug

LEO Pharma

Chief Medical Officer since 2022

MD, PhD

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

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. [2023]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Ophthalmic findings in patients with autosomal recessive lamellar ichthyosis due to TGM1 mutations in an isolated population. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial. [2021]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Compound Heterozygous Mutations in TGM1 Causing a Severe Form of Lamellar Ichthyosis: A Case Report. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Management of congenital ichthyoses: European guidelines of care, part one. [2020]

6. Germany pubmed.ncbi.nlm.nih.gov

Pharmacological treatments for cutaneous manifestations of inherited ichthyoses. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Bathing Suit Variant of Autosomal Recessive Congenital Ichthyosis (ARCI) in Two Indian Patients. [2020]