HL2351 CAPS Phase II Study

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

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02853084

Enrollment

Registered

2016-08-02

Start date

2015-10-31

Completion date

2019-01-31

Last updated

2017-01-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cryopyrin-Associated Periodic Syndromes (CAPS)

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)

Brief 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).

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.

Interventions

DRUGHL2351

The dose will be adjusted based on disease activities

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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

Design outcomes

Primary

Measure	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

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026