Table of contents
- Clinical trial overview
- Who the study is for
- Study treatment and comparison groups
- What the trial measures
- Trial phase and size
- Key trial details
Clinical trial overview
This clinical trial is studying TOP-N53, which is the study name used for 2-[1-(3-{6-[(1E)-(Hydroxyimino)Methyl]-5-Methyl-4-Oxo-7-Propyl-3H,4H-Pyrrolo[2,1-F][1,2,4]Triazin-2-Yl}-4-Propoxybenzenesulfonyl)Piperidin-4-Yl]Ethyl Nitrate in this trial.[1] The study is focused on digital ulcers in people with systemic sclerosis, which are open sores on the fingers in a long-term disease that can affect the skin and blood vessels.[1]
Who the study is for
The trial is designed for patients with systemic sclerosis who have active, ischemic digital ulcers at the fingertips.[1] Ischemic means there is too little blood flow, which can make wounds harder to heal and may increase pain.[1]
- Target population: people with systemic sclerosis and fingertip digital ulcers.[1]
- Study size: the trial plans to enroll 15 patients.[1]
Study treatment and comparison groups
The investigational product is applied directly on the wound by the topical route, meaning it is placed on the ulcer instead of being taken by mouth.[1] The brief summary says the study tests escalating dose levels of 0 micrograms as vehicle, 2 micrograms, 4 micrograms, and 8 micrograms of TOP-N53 in a hydrogel formulation per wound.[1]
- Vehicle: the hydrogel base without the active study drug.[1]
- TOP-N53 topical treatment: tested at 2 micrograms, 4 micrograms, and 8 micrograms per wound.[1]
- Oral comparator in the study design: sildenafil Teva 20 mg film-coated tablets are listed as an oral intervention.[1]
What the trial measures
The main goal is to learn about local safety and tolerability, which means how well the treatment is handled at the wound site and whether it causes unwanted local problems.[1] The primary outcome includes local treatment-emergent adverse events, meaning side effects or medical events that start after treatment begins.[1]
The study also measures patient-reported pain at the digital ulcer.[1] Pain is recorded with a numeric rating scale from 0 to 10, where 0 means no pain and 10 means the worst pain imaginable.[1]
- Local treatment-emergent adverse events: any new local problems seen after treatment starts.[1]
- Pain score: the patient’s own report of pain at the ulcer site using a 0-10 scale.[1]
Trial phase and size
This is a Phase 1/2 trial.[1] Early-phase studies like this usually look first at safety and tolerability, and they may also give early signs about how a treatment behaves in the target group.[1]
The study is currently listed as Authorised.[1] It is a small interventional study with 15 planned participants.[1]
Key trial details
The trial is an interventional study, which means researchers assign the study treatment rather than only observing patients.[1] The brief summary says the treatment is given over a 3-hour exposure time for any dose, and over 24 hours for the 8 microgram dose.[1]
In simple terms, the study is checking whether topical TOP-N53 can be used on fingertip ulcers in systemic sclerosis without causing important local problems, while also tracking pain at the wound site.[1]
