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Pilot Study of Dapansutrile Capsules in Schnitzler's Syndrome

Primary Purpose

Schnitzler Syndrome

Status
Unknown status
Phase
Phase 2
Locations
Netherlands
Study Type
Interventional
Intervention
dapansutrile
Sponsored by
Olatec Therapeutics LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Schnitzler Syndrome focused on measuring Schnitzler

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • RadboudumcRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

dapansutrile capsules

Arm Description

Hard gelatin capsules containing 100 mg of dapansutrile (API)

Outcomes

Primary Outcome Measures

Schnitzler's Syndrome Symptom Index
Proportion of subjects with Grade 0 or 1 SchS symptoms at end of treatment. The Schnitzler's Syndrome Symptom Index (SchS Index) is a composite index incorporating the investigator global assessment of disease activity and levels of plasma CRP. The SchS Index is graded on a 0 - 3 scale with 0 being the best score and indicating "no symptoms of Schnitzler's syndrome" and 3 being the worst score and indicating "severe symptoms of Schnitzler's syndrome".

Secondary Outcome Measures

Physical Examination
A full or targeted physical examination of the patient's major body systems
Vital signs - pulse
Pulse will be recorded and analyzed for changes.
Vital signs - temperature
Body temperature will be recorded and analyzed for changes.
Vital signs - respirations
Respiration rate will be recorded and analyzed for changes.
Vital signs - blood pressure
Systolic and diastolic blood pressure will be recorded and analyzed for changes.
Safety laboratory measures - chemistry markers
Blood samples will be drawn and analyzed for chemistry blood markers.
Safety laboratory measures - hematology/complete blood count
Blood samples will be drawn and a complete blood count will be performed.
Safety laboratory measures - urinalysis
Urine samples will be collected and analyzed.
Adverse events
Adverse events will be described with respect to seriousness, intensity, relationship to treatment, action taken and outcome of the event.
Photographs of posterior torso
Photographs of posterior torso or other non-identifying areas of the body displaying urticarial rash.
Investigator Global Assessment of Disease Activity
One general question the Investigator is asked to answer about the overall perceived status of the subject's symptoms.
Subject Global Assessment of Disease Activity
Overall assessment of disease activity in response to the question: Please note on this scale (0=bad; 10=outstanding) how you are feeling.
Subject Skin Assessment
Extent of urticaria on subject's body.
Time to relapse of SchS Symptoms after cessation of dapansutrile
Time to the emergence of Grade 2 or higher SchS symptoms
Subject Global Evaluation of Treatment
Two general questions the subject is asked to answer about the overall perceived quality of the investigational product.

Full Information

First Posted
June 11, 2018
Last Updated
July 1, 2021
Sponsor
Olatec Therapeutics LLC
Collaborators
Radboud University Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT03595371
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Unknown status
Study Start Date
May 15, 2018 (Actual)
Primary Completion Date
December 30, 2022 (Anticipated)
Study Completion Date
February 28, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Olatec Therapeutics LLC
Collaborators
Radboud University Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Schnitzler Syndrome
Keywords
Schnitzler

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
dapansutrile capsules
Arm Type
Experimental
Arm Description
Hard gelatin capsules containing 100 mg of dapansutrile (API)
Intervention Type
Drug
Intervention Name(s)
dapansutrile
Other Intervention Name(s)
OLT1177 capsules
Intervention Description
500 mg dapansutrile administered twice daily (with a potential to increase the dosage to 2 g dapansutrile daily) for a duration of up to 14 consecutive days.
Primary Outcome Measure Information:
Title
Schnitzler's Syndrome Symptom Index
Description
Proportion of subjects with Grade 0 or 1 SchS symptoms at end of treatment. The Schnitzler's Syndrome Symptom Index (SchS Index) is a composite index incorporating the investigator global assessment of disease activity and levels of plasma CRP. The SchS Index is graded on a 0 - 3 scale with 0 being the best score and indicating "no symptoms of Schnitzler's syndrome" and 3 being the worst score and indicating "severe symptoms of Schnitzler's syndrome".
Time Frame
Day 14
Secondary Outcome Measure Information:
Title
Physical Examination
Description
A full or targeted physical examination of the patient's major body systems
Time Frame
Day 14
Title
Vital signs - pulse
Description
Pulse will be recorded and analyzed for changes.
Time Frame
Day 14
Title
Vital signs - temperature
Description
Body temperature will be recorded and analyzed for changes.
Time Frame
Day 14
Title
Vital signs - respirations
Description
Respiration rate will be recorded and analyzed for changes.
Time Frame
Day 14
Title
Vital signs - blood pressure
Description
Systolic and diastolic blood pressure will be recorded and analyzed for changes.
Time Frame
Day 14
Title
Safety laboratory measures - chemistry markers
Description
Blood samples will be drawn and analyzed for chemistry blood markers.
Time Frame
Day 14
Title
Safety laboratory measures - hematology/complete blood count
Description
Blood samples will be drawn and a complete blood count will be performed.
Time Frame
Day 14
Title
Safety laboratory measures - urinalysis
Description
Urine samples will be collected and analyzed.
Time Frame
Day 14
Title
Adverse events
Description
Adverse events will be described with respect to seriousness, intensity, relationship to treatment, action taken and outcome of the event.
Time Frame
Up to 42 days
Title
Photographs of posterior torso
Description
Photographs of posterior torso or other non-identifying areas of the body displaying urticarial rash.
Time Frame
Day 14
Title
Investigator Global Assessment of Disease Activity
Description
One general question the Investigator is asked to answer about the overall perceived status of the subject's symptoms.
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)
Title
Subject Global Assessment of Disease Activity
Description
Overall assessment of disease activity in response to the question: Please note on this scale (0=bad; 10=outstanding) how you are feeling.
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)
Title
Subject Skin Assessment
Description
Extent of urticaria on subject's body.
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)
Title
Time to relapse of SchS Symptoms after cessation of dapansutrile
Description
Time to the emergence of Grade 2 or higher SchS symptoms
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)
Title
Subject Global Evaluation of Treatment
Description
Two general questions the subject is asked to answer about the overall perceived quality of the investigational product.
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)
Other Pre-specified Outcome Measures:
Title
Plasma concentrations of dapansutrile
Description
Blood samples analyzed for levels of dapansutrile.
Time Frame
Later of Day 14 or symptom relapse (expected no later than Day 28)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male and female subjects 18 years old or older Prior diagnosis of Schnitzler's syndrome 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 Grade 0 SchS symptoms at the Screening/Baseline visit 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 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: Pregnant, nursing or intent to become pregnant during the study Not responsive or well controlled by anakinra therapy for at least 6 weeks prior to the Screening/Baseline visit 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) Active infection within 3 days prior to the Screening/Baseline visit History of or known positive for HIV, Hepatitis B surface antigen (HBsAg) or antibodies to Hepatitis C Virus (HCV) 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 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 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Robert Barrow
Phone
+1 833-652-8321
Email
inquiries@olatec.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Curt Scribner, MD
Organizational Affiliation
Olatec Therapeutics LLC
Official's Role
Study Chair
Facility Information:
Facility Name
Radboudumc
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ruud Raijmakers, MD
First Name & Middle Initial & Last Name & Degree
Anna Simon, MD, PhD
First Name & Middle Initial & Last Name & Degree
Ruud Raijmakers, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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Pilot Study of Dapansutrile Capsules in Schnitzler's Syndrome

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