Cryopyrin-associated Periodic Syndromes (CAPS)Phase 1NCT07247266
Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of JTE-162 in Subjects With Cryopyrin-Associated Periodic Syndrome (CAPS)
A Phase 1 study testing JTE-162 in people with Cryopyrin-associated Periodic Syndromes (CAPS), run by Shionogi Inc. at 1 site in Canada.
Trial summary
This study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS) Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Cryopyrin-associated Periodic Syndromes (CAPS)
- What is being tested?
- JTE-162
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteCanada
- Study dates
- 2 Feb 2026to 10 Sep 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Canada1 site
Gordon Sussman Clinical Research Inc.
North York, Ontario, M3B 3S6
About this study
Official title: 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 study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS)
- Being tested
- JTE-162
- Keywords
- JTE-162, Efficacy, Safety, Tolerability, Cryopyrin-associated periodic syndromes, CAPS, Pharmacokinetics
- Medical subject headings
- Cryopyrin-Associated Periodic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JTE-162Drug
Tablets containing JTE-162
| Study group | Role | What they receive |
|---|---|---|
| JTE-162 Tablets | Experimental | Dose 1 once daily for 2 Weeks Receives: Drug: JTE-162 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP)
Measured: 2 Weeks
Change and percent change from baseline to EOT in Serum amyloid A (SAA)
Measured: 2 Weeks
Change and percent change from baseline to EOT in Interleukin (IL)-6
Measured: 2 Weeks
Change and percent change from baseline to EOT in Key symptom score (KSS)
Measured: 2 Weeks
Change and percent change from baseline to EOT in Individual symptom score on Daily Health Assessment Form, Second Generation (DHAF2)
Measured: 2 Weeks
Change and percent change from baseline to EOT in Global assessments of disease activity on DHAF2
Measured: 2 Weeks
Change and percent change from baseline to EOT in Global CAPS symptom assessment by the Investigator
Measured: 2 Weeks
Change and percent change from baseline to EOT in Individual CAPS symptom assessment by the Investigator
Measured: 2 Weeks
Number of adverse events
Measured: 4 Weeks
JTE-162 post-dose plasma concentrations on Day 1
Measured: 1 Day
JTE-162 trough plasma concentrations on Days 2, 3 and 4, and at the Week 2 Visit (Day 15±2)
Measured: Day 2, Day 3, Day 4 and Day 15±2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2025
- Study start2 Feb 2026
- Primary completion9 Sep 2026
- Study completion10 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cryopyrin-associated Periodic Syndromes (CAPS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shionogi Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07247266
- Sponsor's protocol ID
- AE162-X-24-003
- Status last verified
- Oct 2026
- First submitted
- 18 Nov 2025
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07247266
- Acronym / sponsor ID
- AE162-X-24-003
- Keywords
- JTE-162, Efficacy, Safety, Tolerability, Cryopyrin-associated periodic syndromes, CAPS, Pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Shionogi Inc.Industry (pharma, biotech, devices)
- Collaborators
- PPD Development, LP
- Study start
- 2 Feb 2026 (actual)
- Primary completion
- 9 Sep 2026 (actual)
- Study completion
- 10 Sep 2026 (actual)
- First posted
- 25 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-162 administered once daily for 2 weeks in subjects with cryopyrin-associated periodic syndrome (CAPS)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| JTE-162 Tablets | Experimental | Drug: JTE-162Dose 1 once daily for 2 Weeks |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| JTE-162Tablets containing JTE-162 | Drug | JTE-162 Tablets |
Primary outcome
Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP)
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Serum amyloid A (SAA)
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Interleukin (IL)-6
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Key symptom score (KSS)
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Individual symptom score on Daily Health Assessment Form, Second Generation (DHAF2)
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Global assessments of disease activity on DHAF2
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Global CAPS symptom assessment by the Investigator
Time frame: 2 Weeks
Change and percent change from baseline to EOT in Individual CAPS symptom assessment by the Investigator
Time frame: 2 Weeks
Number of adverse events
Time frame: 4 Weeks
JTE-162 post-dose plasma concentrations on Day 1
Time frame: 1 Day
JTE-162 trough plasma concentrations on Days 2, 3 and 4, and at the Week 2 Visit (Day 15±2)
Time frame: Day 2, Day 3, Day 4 and Day 15±2
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Cryopyrin-associated Periodic Syndromes (CAPS)
RecruitingObservational study
Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases (NOMID/CAPS, DIRA, CANDLE, SAVI, NLRC4-MAS, Still'S-like Diseases, and Other Undifferentiated Autoinflammatory Diseases)
NOMID, DIRA
RecruitingPhase 1
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
Cryopyrin-Associated Periodic Syndromes, Amyloidosis
RecruitingObservational study
Ilaris NIS in Korea
Hereditary Periodic Fever Syndromes, Cryopyrin-associated Periodic Syndromes (CAPS)
Recruiting
Circadian Rhythm Deregulation in Patients With CAPS
Cryopyrin Associated Periodic Syndrome, Familial Cold Urticaria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing JTE-162 in people with Cryopyrin-associated Periodic Syndromes (CAPS), run by Shionogi Inc. at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change and percent change from baseline to end of treatment (EOT) in high-sensitivity C-reactive protein (hs-CRP) (time frame: 2 Weeks). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.