JTE-162 treatment for people with cryopyrin‑associated periodic syndrome (CAPS)

A Phase 1b, Open-label, Single-arm Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)

This will test whether taking JTE-162 once daily for two weeks is safe and helps people with CAPS.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Akros Pharma Inc. (Industry-sponsored)
• Locations: 1 site (North York, Ontario)
• Trial ID: NCT07247266 on ClinicalTrials.gov

What this trial studies

This is a Phase 1 interventional study giving JTE-162 once daily for two weeks to people with CAPS to collect safety, tolerability, pharmacokinetics and early efficacy data. Eligible participants must have FCAS or Muckle‑Wells syndrome with a confirmed NLRP3 mutation, demonstrate a de novo CAPS flare during screening, and be willing to stop anti‑IL‑1 therapy if applicable. The study will include clinical monitoring and PK sampling during and after the dosing period to characterize drug levels and adverse events. The trial is sponsored by Akros Pharma with a single listed site in North York, Ontario.

Who should consider this trial

Good fit: People with FCAS or Muckle‑Wells syndrome who have a confirmed NLRP3 mutation, are experiencing a CAPS flare during screening, and can discontinue anti‑IL‑1 treatment for the washout period.

Not a fit: Patients with CINCA/NOMID, a history of amyloidosis, progressive hearing loss or organ damage, active systemic infections, or who cannot safely stop anti‑IL‑1 therapy are unlikely to benefit or be eligible for this study.

Why it matters

Potential benefit: If successful, JTE-162 could offer a short-course therapeutic option that reduces CAPS flares while demonstrating an acceptable safety and pharmacokinetic profile.

How similar studies have performed: Other therapies that target the IL‑1 pathway have shown clear benefit in CAPS, so the general therapeutic approach has precedent, but JTE‑162 itself appears to be a novel investigational agent without published CAPS results.

Eligibility criteria

Inclusion Criteria:

* Diagnosed with familial cold autoinflammatory syndrome (FCAS) or Muckle-Wells syndrome (MWS) confirmed by:
* Clinical History: At least 2 typical clinical symptoms (e.g., urticarial skin rash, myalgia, arthralgia, recurrent fever, fatigue/malaise, conjunctivitis or other autoinflammatory symptoms) prior to the Screening Visit; AND
* Genetic Confirmation: Confirmed nucleotide-binding and oligomerization domain (NOD)-like receptor family pyrin domain containing 3 (NLRP3) mutation;
* Willing to discontinue current anti-interleukin (IL)-1 treatment, if applicable;
* Demonstrates de novo flaring of CAPS during the Screening Period.

Exclusion Criteria:

* Has chronic infantile neurologic cutaneous articular syndrome (CINCA)/neonatal-onset multisystem inflammatory disease (NOMID);
* Has a history or presence of amyloidosis, progressive hearing loss, organ damage or any symptom contraindicating anti-IL-1 treatment washout;
* Has active systemic bacterial, fungal or viral infection(s) within 14 days prior to Day 1 or a history of clinically significant recurrent infectious diseases

Where this trial is running

North York, Ontario

• Gordon Sussman Clinical Research Inc. — North York, Ontario, Canada (Recruiting)

Study contacts

• Study coordinator: Takanori Nemoto, M.S.

Conditions Cryopyrin-associated Periodic Syndromes; JTE-162; Efficacy; Safety; Tolerability; Cryopyrin-associated periodic syndromes; CAPS; Pharmacokinetics