JTE-162 for Cryopyrin-Associated Periodic Syndromes

Overseen By Kala Patel, R.Ph., RAC

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Akros Pharma Inc.

Must not be taking: Anti-IL-1 treatments

Disqualifiers: CINCA/NOMID, Amyloidosis, Progressive hearing loss, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 2 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called JTE-162 for individuals with cryopyrin-associated periodic syndromes (CAPS), a condition causing recurring fevers and inflammation. The study aims to evaluate the effectiveness and safety of JTE-162 when taken once daily for two weeks. Candidates may qualify if diagnosed with familial cold autoinflammatory syndrome (FCAS) or Muckle-Wells syndrome (MWS), experience symptoms like skin rash and joint pain, and possess a specific genetic marker. Participants must be willing to discontinue their current treatment for the trial's duration. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

You may need to stop taking your current anti-interleukin (IL)-1 treatment if you are on it, as the trial requires participants to discontinue this medication.

Is there any evidence suggesting that JTE-162 is likely to be safe for humans?

Research has shown that JTE-162 remains in the early testing stages, so limited safety information is available. This trial is in Phase 1, the initial step in testing a new treatment in people. At this stage, the primary goal is to assess the treatment's safety and tolerability.

Since JTE-162 is not approved for use outside of trials, no data from other studies or approved uses demonstrate its safety. Participants in this study will help determine how well people tolerate JTE-162 and identify any side effects.

Phase 1 trials are typically carefully controlled and monitored to ensure participant safety. Those considering joining will be in a setting where researchers closely monitor any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Cryopyrin-Associated Periodic Syndromes, which often include biologics that target specific inflammatory proteins, JTE-162 is a new oral medication being tested. Researchers are excited about JTE-162 because it offers a simpler, more convenient pill form, potentially improving patient compliance compared to injectable treatments. Additionally, JTE-162 may have a unique mechanism of action that targets different pathways involved in inflammation, offering hope for better symptom control and fewer side effects.

What evidence suggests that JTE-162 might be an effective treatment for CAPS?

Research shows that JTE-162 is under study as a treatment for cryopyrin-associated periodic syndromes (CAPS), a rare condition causing inflammation due to immune system issues. In this trial, JTE-162 is in the early testing stages, with participants receiving JTE-162 tablets once daily for 2 weeks. Researchers are examining its ability to reduce inflammation by targeting specific parts of the immune system. This method resembles canakinumab, a treatment already used for CAPS, which has proven effective in up to 94.7% of patients in some studies. The success of canakinumab suggests that JTE-162 might also be effective, but further research is needed to confirm its benefits. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals with Cryopyrin-Associated Periodic Syndrome (CAPS), which includes conditions like Familial Cold Autoinflammatory Syndrome. Participants should meet specific health criteria set by the study.

Inclusion Criteria

I have FCAS or MWS, confirmed by symptoms and a genetic test.

I am willing to stop my current IL-1 treatment if needed.

Demonstrates de novo flaring of CAPS during the Screening Period

Exclusion Criteria

I have conditions like amyloidosis or hearing loss that may not allow for anti-IL-1 treatment.

I haven't had serious infections or recurring infectious diseases recently.

I have CINCA/NOMID.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive JTE-162 tablets once daily for 2 weeks

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• JTE-162

Trial Overview The trial is testing JTE-162, a potential treatment for CAPS. It's given once daily for two weeks to assess its effectiveness and how the body processes it.

How Is the Trial Designed?

Group I: JTE-162 Tablets Experimental Treatment 1 Intervention

Dose 1 once daily for 2 Weeks

Who Is Running the Clinical Trial?

Akros Pharma Inc.

Lead Sponsor

Trials

24

Recruited

2,800+

PPD Development, LP

Industry Sponsor

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07247266

NCT07247266 | Phase 1b, Open-label Study to Evaluate ... Phase 1b, Open-label Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/26667214/

Real-life effectiveness of canakinumab in cryopyrin- ... Overall, complete response (CR) was seen in 72% of CAPS patients, significantly less often in severe (14%) than in mild CAPS phenotypes (79%). Only 53% attained ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/23380020/

Safety and efficacy of canakinumab in Japanese patients ... Results: A complete response was achieved in 18 (94.7%) patients with some requiring a dose and/or a frequency adjustment to attain full ...

4. clinexprheumatol.org clinexprheumatol.org/article.asp?a=15800

Real-world safety and effectiveness of canakinumab in ... Treatment with canakinumab has suggested sustained remission in the majority of patients in the real-world setting. Key words canakinumab, cryopyrin-associated ...

5. jheor.org jheor.org/article/9860-a-retrospective-patient-chart-review-and-survey-in-patients-with-cryopyrin-associated-periodic-syndromes-treated-with-anakinra.pdf

A Retrospective Patient Chart Review and Survey in ... Cryopyrin-associated periodic syndrome (CAPS) is a rare autoinflammatory disease which encompasses a disease severity spectrum from familial cold ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/41263512/

final results from a post-marketing surveillance in Japan Safety and effectiveness of canakinumab in Japanese patients with cryopyrin-associated periodic syndrome: final results from a post-marketing ...

7. clinexprheumatol.org clinexprheumatol.org/article.asp?a=10184

Long-term safety and efficacy of canakinumab in cryopyrin- ... To assess the long-term safety and efficacy of canakinumab in. Japanese patients with cryopyrin-asso- ciated periodic syndrome (CAPS). Methods. In this open- ...

8. aiarthritis.org aiarthritis.org/understanding-cryopyrin-associated-periodic-syndromes-caps

Understanding Cryopyrin-Associated Periodic Syndromes ... Cryopyrin-Associated Periodic Syndromes (CAPS) are a group of rare, autoinflammatory disorders characterized by systemic inflammation due to mutations in the ...