The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis
Status:
Not yet recruiting
Trial end date:
2024-05-01
Target enrollment:
Participant gender:
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 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 (Figure 1):
• 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 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.