Pilot Study of Dapansutrile Capsules in Schnitzler’s Syndrome

Brief Title

Pilot Study of Dapansutrile Capsules in Schnitzler's Syndrome

Official Title

A Pilot, Open-Label, Phase 2, Single-Center, Repeat Dose, Proof- Of-Concept Safety, Pharmacodynamics and Efficacy Study of Orally Administered Dapansutrile Capsules in Subjects With Schnitzler's Syndrome

Brief Summary

      This is a pilot, open-label Phase 2, single-center, repeat dose, single cohort,
      proof-of-concept, safety, pharmacodynamics and efficacy study of dapansutrile capsules to be
      conducted in subjects with Schnitzler's syndrome (SchS) currently well controlled by anakinra
      therapy. At least 5 but no more than 10 subjects will be enrolled.
    

Detailed Description

      Subjects who are currently taking and responsive to anakinra (Kineret®) for at least 6 weeks
      will be screened for eligibility at the Screening / Baseline (Day 1) visit. Following
      confirmation of eligibility, subjects will be enrolled, the first dose of dapansutrile will
      be administered at the clinical site and safety and efficacy assessments will be completed.
      Subjects will self-administer dapansutrile twice a day by mouth for 14 consecutive days.
      Subjects will continue their standard dose of anakinra for Days 1, 2 and 3 of the 14-day
      Treatment Period and will then cease taking anakinra. At the end of the 14-day Treatment
      Period subjects will remain off all medication for Schnitzler's syndrome and at the first
      signs of a relapse or worsening of SchS symptoms, subjects will visit the study clinic for
      the Symptom Onset visit (SOV) to determine with the Investigator when injections of anakinra
      should be resumed. In addition, subjects will return to the study clinic on Days 5, 9, 14,
      15,16, 18 and 21 for follow-up visits1 and will be contacted by telephone on Day 42 (± 3
      Days) for additional follow-up. The Day 15 (± 1 day), Day 16 (± 1 day) and Day 18 (± 1 day)
      visits will only occur if anakinra therapy has not yet been resumed.

      Subjects will be given the option to remain in the Nijmegen area after the Day 14 visit and
      return to the study clinic for the Day 15, 16 and 18 follow-up visits. Alternatively,
      subjects will be given the option to have these visits conducted at their home by a trained
      study nurse.

      Safety assessments will be conducted at each visit and subjects will capture the frequency
      and intensity of symptoms, including body temperature, using a paper diary. Safety and
      tolerability will be evaluated by monitoring the occurrence of adverse events (AEs) and
      changes in abbreviated physical examination findings, vital signs and clinical safety
      laboratory test results (chemistry, hematology and urinalysis) and inflammatory biomarkers.
      Clinical activity will be evaluated by: Subject Diary (completed daily), Subject Global
      Assessment of Disease Activity, Investigator Global Assessment of Disease Activity, and
      analysis of biomarkers of inflammation, including changes in C-reactive protein (CRP). Daily
      diary assessments will be captured starting at the Screening / Baseline (Day 1) visit and
      will continue until Symptom Onset visit or Day 21 visit (whichever occurs latest).
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Schnitzler's Syndrome Symptom Index

Secondary Outcome

 Physical Examination

Condition

Schnitzler Syndrome

Intervention

dapansutrile

Study Arms / Comparison Groups

 dapansutrile capsules
Description:  Hard gelatin capsules containing 100 mg of dapansutrile (API)

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

10

Start Date

May 15, 2018

Completion Date

August 31, 2020

Primary Completion Date

June 30, 2020

Eligibility Criteria

        Inclusion Criteria:

          1. Male and female subjects 18 years old or older

          2. Prior diagnosis of Schnitzler's syndrome

          3. Presence of Schnitzler's syndrome that is well controlled by and responsive to
             anakinra for at least 6 weeks prior to the Screening/Baseline visit

          4. Grade 0 SchS symptoms at the Screening/Baseline visit

          5. Acceptable overall medical condition to be safely enrolled in and to complete the
             study (with specific regard to cardiovascular, renal and hepatic conditions) in the
             opinion of the Investigator

          6. Ability to provide written informed consent prior to initiation of any study-related
             procedures, and ability, in the opinion of the Investigator, to understand and comply
             with all the requirements of the study as outlined in the protocol.

        Exclusion Criteria:

          1. Pregnant, nursing or intent to become pregnant during the study

          2. Not responsive or well controlled by anakinra therapy for at least 6 weeks prior to
             the Screening/Baseline visit

          3. Use or planned use of any prohibited concomitant medications/therapies such as
             immunotherapies or corticosteroids during the study (until relapse and resumption of
             anakinra injections)

          4. Active infection within 3 days prior to the Screening/Baseline visit

          5. History of or known positive for HIV, Hepatitis B surface antigen (HBsAg) or
             antibodies to Hepatitis C Virus (HCV)

          6. Any other concomitant medical or psychiatric conditions, including alcohol or
             substance abuse, diseases or prior surgeries that in the opinion of the Investigator
             would impair the subject from safely participating in the trial and/or completing
             protocol requirements

          7. Enrollment in any trial and/or use of any investigational product or device within the
             immediate 30-day period prior to the Screening/Baseline visit

          8. Enrollment in any study previously sponsored by Olatec Therapeutics LLC, specifically
             Study OLT1177-01, Study OLT1177-02, Study OLT1177-03, Study OLT1177-04 or Study
             OLT1177-05
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Curt Scribner, MD, +1 833-652-8321, [email protected]

Location Countries

Netherlands

Location Countries

Netherlands

Administrative Informations


NCT ID

NCT03595371

Organization ID

OLT1177-07


Responsible Party

Sponsor

Study Sponsor

Olatec Therapeutics LLC

Collaborators

 Radboud University

Study Sponsor

Curt Scribner, MD, Study Chair, Olatec Therapeutics LLC


Verification Date

August 2019