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The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis

NCT05295732 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 153

Last updated 2026-05-22

Study results available
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Summary

This is a randomized, double-blind and vehicle-controlled Phase III study to evaluate the safety and efficacy of topical TMB-001 0.05% ointment for the treatment of congenital ichthyosis (CI) in subjects with either the RXLI or ARCI subtypes.

In addition, a subset of preselected centers will recruit subjects in parallel with either the RXLI or ARCI subtypes for enrollment into an Optional Maximal Use arm for evaluation of the systemic exposure and safety of topical TMB-001 0.05% ointment for the treatment of CI.

The Phase III Study is designed in three periods:

\- Period 1 - Induction (3 weeks):

At the beginning of the 3-week Induction Period, eligible subjects will be randomized (2:1 ratio) to either TMB-001 0.05% once-a-day (QD) or Vehicle QD treatment, with use of mandatory standardized bland emollient (Cetaphil™) provided by the Sponsor.

\- Period 2 - Treatment (9 weeks):

The dosing frequency in the 9-week treatment period will be increased in each treatment group to TMB-001 0.05% BID or Vehicle BID. Mandatory bland emollient will be discontinued.

\- Period 3 - Maintenance (12 weeks):

At Week 12, eligible subjects in the TMB-001 treatment group will be randomized (1:1 ratio) to an open-label treatment with TMB-001 0.05% BID or TMB-001 0.05% QD. To be eligible, subjects must have achieved a ≥1-point reduction in IGA score from Baseline. Subjects with less than a 1-point reduction in IGA score from Baseline will be discontinued from the study.

Vehicle-treated subjects who achieved \<1-point reduction in IGA score from Baseline are eligible to cross over to the TMB-001 0.05% BID treatment group. Subjects with a ≥1-point reduction in IGA score from Baseline on vehicle will be discontinued from the study.

Subjects at the end of the study or subjects discontinued from the study at any time will be followed-up for additional 2 weeks for AEs.

Conditions

  • Ichthyosis

Interventions

DRUG

TMB-001

Topical TMB-001 0.05% QD/BID

DRUG

Matching Vehicle

Topical Vehicle

Sponsors & Collaborators

  • Timber Pharmaceuticals Inc.

    collaborator INDUSTRY

  • LEO Pharma

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-06-21
Primary Completion
2024-06-17
Completion
2024-09-23
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France
  • Germany
  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05295732 on ClinicalTrials.gov