Cryopyrin-Associated Periodic Syndromes (CAPS)Phase 2NCT02853084
HL2351 CAPS Phase II Study
A Phase 2 study testing HL2351 in people with Cryopyrin-Associated Periodic Syndromes (CAPS), run by Handok Inc. at 1 site in South Korea.
Trial summary
This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cryopyrin-Associated Periodic Syndromes (CAPS)
- What is being tested?
- HL2351
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteSouth Korea
- Study dates
- Oct 2015to Jan 2019
- Phase
- Phase 2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Documented molecular diagnosis of NALP3 mutation or diagnosis of CAPS based on sign and symptom
- Diagnosis of CAPS based on signs and symptoms
- Current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids
Who cannot join
- Patient been diagnosed with a hereditary periodic fever syndrome or autoinflammatory disease other than CAPS
- Patients whose disease is inadequately controlled on current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open Label, Single Arm Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of HL2351 in Patients With Cryopyrin Associated Periodic Syndromes
This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).
Read the detailed description
This trial enrolls patients with CAPS being on stable dose of anakinra daily and then switch over HL2351 given once weekly. It consists of 4 phases, 6-week screening, single dose PK, 6-month multiple dose, and 18-month extension phase.
- Being tested
- HL2351
- Keywords
- Auto-inflammatory diseases, IL-1 inhibitors, HL2351, Cryopyrin-Associated Periodic Syndromes (CAPS), Neonatal Onset Multisystem Inflammatory Disease (NOMID), Muckle-Wells Syndrome (MWS), Familial Cold Autoinflammatory Syndrome (FCAS)
- 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.
HL2351Drug
The dose will be adjusted based on disease activities
| Study group | Role | What they receive |
|---|---|---|
| HL2351 | Experimental | Not described Receives: Drug: HL2351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Diary Symptom Sum Score (DSSS)
Measured: from baseline to Month 6
Change in SAA, CRP, ESR levels
Measured: from baseline to Month 6, from baseline to each visit up to Month 24
Change in physician's global assessment of autoinflammatory using 100mm VAS score disease
Measured: from baseline to Month 6, from baseline to each visit up to Month 24
Change in patient's(parents or legal guardians) global assessment of autoinflammatory using 100mm VAS score disease
Measured: from baseline to Month 6, from baseline to each visit up to Month 24
Pharmacokinetic profile(Cmax)
Measured: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(AUClast)
Measured: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(AUCinf)
Measured: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(Tmax)
Measured: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(t1/2)
Measured: 0,12,24,36,48,72,96,144,168 hr
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2016 (estimated)
- Study startOct 2015
- Primary completionMay 2017 (estimated)
- Study completionJan 2019 (estimated)
Why it stopped: difficult of recruiting
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Handok Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- JOONG GON KIM, Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02853084
- Sponsor's protocol ID
- HL_C201
- Status last verified
- May 2016
- First submitted
- 10 May 2016
- Last updated
- 4 Jan 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 4 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Single Arm Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of HL2351 in Patients With Cryopyrin Associated Periodic Syndromes
- ClinicalTrials.gov ID
- NCT02853084
- Acronym / sponsor ID
- HL_C201
- Keywords
- Auto-inflammatory diseases, IL-1 inhibitors, HL2351, Cryopyrin-Associated Periodic Syndromes (CAPS), Neonatal Onset Multisystem Inflammatory Disease (NOMID), Muckle-Wells Syndrome (MWS), Familial Cold Autoinflammatory Syndrome (FCAS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: difficult of recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Handok Inc.Industry (pharma, biotech, devices)
- Study start
- Oct 2015
- Primary completion
- May 2017 (estimated)
- Study completion
- Jan 2019 (estimated)
- First posted
- 2 Aug 2016 (estimated)
- 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 is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).
Detailed description
This trial enrolls patients with CAPS being on stable dose of anakinra daily and then switch over HL2351 given once weekly. It consists of 4 phases, 6-week screening, single dose PK, 6-month multiple dose, and 18-month extension phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HL2351 | Experimental | Drug: HL2351 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Documented molecular diagnosis of NALP3 mutation or diagnosis of CAPS based on sign and symptom
- Diagnosis of CAPS based on signs and symptoms
- Current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids
Key exclusion criteria (2 listed)
- Patient been diagnosed with a hereditary periodic fever syndrome or autoinflammatory disease other than CAPS
- Patients whose disease is inadequately controlled on current stable doses of anakinra, alone or in combination with methotrexate or corticosteroids
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 |
|---|---|---|
| HL2351The dose will be adjusted based on disease activities | Drug | HL2351 |
Primary outcome
Change in Diary Symptom Sum Score (DSSS)
Time frame: from baseline to Month 6
Change in SAA, CRP, ESR levels
Time frame: from baseline to Month 6, from baseline to each visit up to Month 24
Change in physician's global assessment of autoinflammatory using 100mm VAS score disease
Time frame: from baseline to Month 6, from baseline to each visit up to Month 24
Change in patient's(parents or legal guardians) global assessment of autoinflammatory using 100mm VAS score disease
Time frame: from baseline to Month 6, from baseline to each visit up to Month 24
Pharmacokinetic profile(Cmax)
Time frame: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(AUClast)
Time frame: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(AUCinf)
Time frame: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(Tmax)
Time frame: 0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(t1/2)
Time frame: 0,12,24,36,48,72,96,144,168 hr
Results and safety
The study was stopped early (reason given: difficult of recruiting). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jan 2017.
- Individual participant dataSharing planned: Undecided
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
Not yet recruitingObservational study
Are Mast Cells Involved in Autoinflammatory Diseases
Autoinflammatory Disease, FMF
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.
JOONG GON KIM principal investigator
Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HL2351 in people with Cryopyrin-Associated Periodic Syndromes (CAPS), run by Handok Inc. at 1 site in South Korea.
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 3 inclusion and 2 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: stopped early, as of the sponsor's last update on 4 Jan 2017. 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 South Korea. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 was stopped early (reason given: difficult of recruiting). Results are not available in the source record.
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 in Diary Symptom Sum Score (DSSS) (time frame: from baseline to Month 6). The study lists 9 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 4 Jan 2017.