HL2351 CAPS Phase II Study

NCT02853084 | Terminated Phase 2 DMC

• 1 other identifier: HL_C201
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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).

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)

Outcome Measures

Primary Outcomes (9)

• 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

Study Arms (1)

HL2351

EXPERIMENTAL

Drug: HL2351

Interventions

HL2351 DRUG

The dose will be adjusted based on disease activities

HL2351

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Handok Inc.: lead

Study Sites (1)

Handok Inc.

Seoul, South Korea

Location

MeSH Terms

Conditions

Cryopyrin-Associated Periodic Syndromes

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Chronic Inducible Urticaria; Chronic Urticaria; Urticaria; Skin Diseases, Vascular; Cold Urticaria; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• JOONG GON KIM

  Seoul National University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2016
• First Posted: August 2, 2016
• Study Start: October 1, 2015
• Primary Completion: May 1, 2017
• Study Completion: January 1, 2019
• Last Updated: January 4, 2017

Record last verified: 2016-05

Locations

KR (1)