HL2351 CAPS Phase II Study

Phase 2

Terminated

• Conditions: Cryopyrin-Associated Periodic Syndromes (CAPS)
• Interventions: Drug: HL2351

• Registration Number: NCT02853084
• Lead Sponsor: Handok Inc.

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.

Study Timeline

• Study Start: 2015-10
• Status Verified: 2016-05
• Study First Submitted: 2016-05-10
• Study First Submitted QC: 2016-07-28
• Study First Posted: 2016-08-02
• Last Update Submitted: 2017-01-03
• Last Update Posted: 2017-01-04
• Primary Completion: 2017-05
• Study Completion: 2019-01

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HL2351	HL2351	-

Primary Outcome Measures

Name	Time	Method
Pharmacokinetic profile(Tmax)	0,12,24,36,48,72,96,144,168 hr
Change in Diary Symptom Sum Score (DSSS)	from baseline to Month 6
Pharmacokinetic profile(AUCinf)	0,12,24,36,48,72,96,144,168 hr
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(t1/2)	0,12,24,36,48,72,96,144,168 hr
Pharmacokinetic profile(Cmax)	0,12,24,36,48,72,96,144,168 hr
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
Pharmacokinetic profile(AUClast)	0,12,24,36,48,72,96,144,168 hr

Trial Locations

Locations (1)

Handok Inc.; 🇰🇷 Seoul, Korea, Republic of