Back to resultsHL2351 CAPS Phase II Study
Primary Purpose
Cryopyrin-Associated Periodic Syndromes (CAPS)
Status
Terminated
Phase
Phase 2
Locations
Korea, Republic of
Study Type
Interventional
Intervention
HL2351
Sponsored by
About this trial
This is an interventional treatment trial for Cryopyrin-Associated Periodic Syndromes (CAPS) focused on measuring 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)
Eligibility Criteria
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
Sites / Locations
- Handok Inc.
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HL2351
Arm Description
Outcomes
Primary Outcome Measures
Change in Diary Symptom Sum Score (DSSS)
Change in SAA, CRP, ESR levels
Change in physician's global assessment of autoinflammatory using 100mm VAS score disease
Change in patient's(parents or legal guardians) global assessment of autoinflammatory using 100mm VAS score disease
Pharmacokinetic profile(Cmax)
Pharmacokinetic profile(AUClast)
Pharmacokinetic profile(AUCinf)
Pharmacokinetic profile(Tmax)
Pharmacokinetic profile(t1/2)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02853084
Brief Title
HL2351 CAPS Phase II Study
Official Title
An Open Label, Single Arm Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of HL2351 in Patients With Cryopyrin Associated Periodic Syndromes
Study Type
Interventional
2. Study Status
Record Verification Date
May 2016
Overall Recruitment Status
Terminated
Why Stopped
difficult of recruiting
Study Start Date
October 2015 (undefined)
Primary Completion Date
May 2017 (Anticipated)
Study Completion Date
January 2019 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Handok Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
HL2351
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
HL2351
Intervention Description
The dose will be adjusted based on disease activities
Primary Outcome Measure Information:
Title
Change in Diary Symptom Sum Score (DSSS)
Time Frame
from baseline to Month 6
Title
Change in SAA, CRP, ESR levels
Time Frame
from baseline to Month 6, from baseline to each visit up to Month 24
Title
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
Title
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
Title
Pharmacokinetic profile(Cmax)
Time Frame
0,12,24,36,48,72,96,144,168 hr
Title
Pharmacokinetic profile(AUClast)
Time Frame
0,12,24,36,48,72,96,144,168 hr
Title
Pharmacokinetic profile(AUCinf)
Time Frame
0,12,24,36,48,72,96,144,168 hr
Title
Pharmacokinetic profile(Tmax)
Time Frame
0,12,24,36,48,72,96,144,168 hr
Title
Pharmacokinetic profile(t1/2)
Time Frame
0,12,24,36,48,72,96,144,168 hr
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
JOONG GON KIM
Organizational Affiliation
Seoul National University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Handok Inc.
City
Seoul
Country
Korea, Republic of
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
HL2351 CAPS Phase II Study
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