HL2351 CAPS Phase II Study

Overview

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).

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

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: May 2017

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

  • Drug: HL2351
    • The dose will be adjusted based on disease activities

Arms, Groups and Cohorts

  • Experimental: HL2351

Clinical Trial Outcome Measures

Primary Measures

  • 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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Handok Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • JOONG GON KIM, Principal Investigator, Seoul National University Hospital

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