Pilot Study of Dapansutrile Capsules in Schnitzler's Syndrome

NCT03595371 | Terminated Phase 2 FDA Drug

• 1 other identifier: OLT1177-07
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Schnitzler Syndrome

Keywords

Schnitzler

Outcome Measures

Primary Outcomes (1)

• 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".

  Day 14

Secondary Outcomes (15)

• Physical Examination

  Day 14

• Vital signs - pulse

  Day 14

• Vital signs - temperature

  Day 14

• Vital signs - respirations

  Day 14

• Vital signs - blood pressure

  Day 14

Other Outcomes (1)

• Plasma concentrations of dapansutrile

  Later of Day 14 or symptom relapse (expected no later than Day 28)

Study Arms (1)

dapansutrile capsules

EXPERIMENTAL

Hard gelatin capsules containing 100 mg of dapansutrile (API)

Drug: dapansutrile

Interventions

dapansutrile DRUG

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.

Also known as: OLT1177 capsules

dapansutrile capsules

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Olatec Therapeutics LLC: lead
• Radboud University Medical Center: collaborator

Study Sites (1)

Radboudumc

Nijmegen, 6525 GA, Netherlands

Location

MeSH Terms

Conditions

Schnitzler Syndrome

Interventions

dapansutrile

Condition Hierarchy (Ancestors)

Monoclonal Gammopathy of Undetermined Significance; Paraproteinemias; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Curt Scribner, MD

  Olatec Therapeutics LLC

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 11, 2018
• First Posted: July 23, 2018
• Study Start: May 15, 2018
• Primary Completion: May 29, 2018
• Study Completion: May 29, 2018
• Last Updated: February 2, 2024

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)