HL2351 CAPS Phase II Study

Cryopyrin-Associated Periodic Syndromes (CAPS) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02853084
• Other study ID #: HL_C201
• Status: Terminated
• Phase: Phase 2
• First received: May 10, 2016
• Last updated: January 3, 2017
• Start date: October 2015
• Est. completion date: January 2019

Study information

• Verified date: May 2016
• Source: Handok Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Ministry of Food and Drug Safety
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 8
• Est. completion date: January 2019
• Est. primary completion date: May 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cryopyrin-Associated Periodic Syndromes
• Cryopyrin-Associated Periodic Syndromes (CAPS)
• Syndrome

Intervention

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

Locations

Country	Name	City	State
Korea, Republic of	Handok Inc.	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Handok Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Diary Symptom Sum Score (DSSS)		from baseline to Month 6	No
Primary	Change in SAA, CRP, ESR levels		from baseline to Month 6, from baseline to each visit up to Month 24	No
Primary	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	No
Primary	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	No
Primary	Pharmacokinetic profile(Cmax)		0,12,24,36,48,72,96,144,168 hr	No
Primary	Pharmacokinetic profile(AUClast)		0,12,24,36,48,72,96,144,168 hr	No
Primary	Pharmacokinetic profile(AUCinf)		0,12,24,36,48,72,96,144,168 hr	No
Primary	Pharmacokinetic profile(Tmax)		0,12,24,36,48,72,96,144,168 hr	No
Primary	Pharmacokinetic profile(t1/2)		0,12,24,36,48,72,96,144,168 hr	No