HL2351 CAPS Phase II Study

Study Overview
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Brief Summary
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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
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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.

Official Title
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An Open Label, Single Arm Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of HL2351 in Patients With Cryopyrin Associated Periodic Syndromes

Conditions
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Cryopyrin-Associated Periodic Syndromes (CAPS)

Intervention / Treatment
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* Drug: HL2351


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: HL2351<br> | Drug: HL2351<br>* The dose will be adjusted based on disease activities<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Diary Symptom Sum Score (DSSS) |  | from baseline to Month 6 | 
| Change in SAA, CRP, ESR levels |  | 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 |  | 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 |  | from baseline to Month 6, from baseline to each visit up to Month 24 | 
| Pharmacokinetic profile(Cmax) |  | 0,12,24,36,48,72,96,144,168 hr | 
| Pharmacokinetic profile(AUClast) |  | 0,12,24,36,48,72,96,144,168 hr | 
| Pharmacokinetic profile(AUCinf) |  | 0,12,24,36,48,72,96,144,168 hr | 
| Pharmacokinetic profile(Tmax) |  | 0,12,24,36,48,72,96,144,168 hr | 
| Pharmacokinetic profile(t1/2) |  | 0,12,24,36,48,72,96,144,168 hr | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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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)