ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 13 of 16 for:    "Muckle-Wells syndrome"

HL2351 CAPS Phase II Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02853084
Recruitment Status : Terminated (difficult of recruiting)
First Posted : August 2, 2016
Last Update Posted : January 4, 2017
Sponsor:
Information provided by (Responsible Party):
Handok Pharmaceuticals Co., Ltd.

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).

Condition or disease Intervention/treatment Phase
Cryopyrin‑Associated Periodic Syndromes (CAPS) Drug: HL2351 Phase 2

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : October 2015
Estimated Primary Completion Date : May 2017
Estimated Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: HL2351 Drug: HL2351
The dose will be adjusted based on disease activities




Primary Outcome Measures :
  1. Change in Diary Symptom Sum Score (DSSS) [ Time Frame: from baseline to Month 6 ]
  2. Change in SAA, CRP, ESR levels [ Time Frame: from baseline to Month 6, from baseline to each visit up to Month 24 ]
  3. 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 ]
  4. 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 ]
  5. Pharmacokinetic profile(Cmax) [ Time Frame: 0,12,24,36,48,72,96,144,168 hr ]
  6. Pharmacokinetic profile(AUClast) [ Time Frame: 0,12,24,36,48,72,96,144,168 hr ]
  7. Pharmacokinetic profile(AUCinf) [ Time Frame: 0,12,24,36,48,72,96,144,168 hr ]
  8. Pharmacokinetic profile(Tmax) [ Time Frame: 0,12,24,36,48,72,96,144,168 hr ]
  9. Pharmacokinetic profile(t1/2) [ Time Frame: 0,12,24,36,48,72,96,144,168 hr ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02853084


Locations
Korea, Republic of
Handok Inc.
Seoul, Korea, Republic of
Sponsors and Collaborators
Handok Pharmaceuticals Co., Ltd.
Investigators
Principal Investigator: JOONG GON KIM Seoul National University Hospital

Responsible Party: Handok Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT02853084     History of Changes
Other Study ID Numbers: HL_C201
First Posted: August 2, 2016    Key Record Dates
Last Update Posted: January 4, 2017
Last Verified: May 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Handok Pharmaceuticals Co., Ltd.:
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)

Additional relevant MeSH terms:
Syndrome
Cryopyrin-Associated Periodic Syndromes
Disease
Pathologic Processes
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases