Schnitzler SyndromePhase 2NCT03595371
Pilot Study of Dapansutrile Capsules in Schnitzler's Syndrome
A Phase 2 study testing dapansutrile in people with Schnitzler Syndrome, run by Olatec Therapeutics, Inc. at 1 site in Netherlands.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Schnitzler Syndrome
- What is being tested?
- dapansutrile
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteNetherlands
- Study dates
- 15 May 2018to 29 May 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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).
- Conditions
- Schnitzler Syndrome
- Being tested
- dapansutrile
- Keywords
- Schnitzler
- Medical subject headings
- Schnitzler Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dapansutrileDrug
Also called OLT1177 capsules
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.
| Study group | Role | What they receive |
|---|---|---|
| dapansutrile capsules | Experimental | Hard gelatin capsules containing 100 mg of dapansutrile (API) Receives: Drug: dapansutrile |
What is being measured
The primary outcome is the main question the study is designed to answer.
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".
Measured: Day 14
Secondary outcomes (15)
Physical Examination
A full or targeted physical examination of the patient's major body systems
Measured: Day 14
Vital signs - pulse
Pulse will be recorded and analyzed for changes.
Measured: Day 14
Vital signs - temperature
Body temperature will be recorded and analyzed for changes.
Measured: Day 14
Vital signs - respirations
Respiration rate will be recorded and analyzed for changes.
Measured: Day 14
Vital signs - blood pressure
Systolic and diastolic blood pressure will be recorded and analyzed for changes.
Measured: Day 14
Safety laboratory measures - chemistry markers
Blood samples will be drawn and analyzed for chemistry blood markers.
Measured: Day 14
Safety laboratory measures - hematology/complete blood count
Blood samples will be drawn and a complete blood count will be performed.
Measured: Day 14
Safety laboratory measures - urinalysis
Urine samples will be collected and analyzed.
Measured: Day 14
Adverse events
Adverse events will be described with respect to seriousness, intensity, relationship to treatment, action taken and outcome of the event.
Measured: Up to 42 days
Photographs of posterior torso
Photographs of posterior torso or other non-identifying areas of the body displaying urticarial rash.
Measured: Day 14
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.
Measured: Later of Day 14 or symptom relapse (expected no later than Day 28)
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.
Measured: Later of Day 14 or symptom relapse (expected no later than Day 28)
Subject Skin Assessment
Extent of urticaria on subject's body.
Measured: Later of Day 14 or symptom relapse (expected no later than Day 28)
Time to relapse of SchS Symptoms after cessation of dapansutrile
Time to the emergence of Grade 2 or higher SchS symptoms
Measured: Later of Day 14 or symptom relapse (expected no later than Day 28)
Subject Global Evaluation of Treatment
Two general questions the subject is asked to answer about the overall perceived quality of the investigational product.
Measured: Later of Day 14 or symptom relapse (expected no later than Day 28)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2018
- Study start15 May 2018
- Primary completion29 May 2018
- Study completion29 May 2018
Why it stopped: The study was terminated due to study design issues that did not allow for determination of efficacy and safety in subjects with Schnitzler Syndrome who are currently well controlled on anakinra therapy.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Schnitzler SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Olatec Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Radboud University Medical Center
- Responsible party
- Sponsor
- Investigators
- Curt Scribner, MD, Study chair, Olatec Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03595371
- Sponsor's protocol ID
- OLT1177-07
- Status last verified
- Jan 2023
- First submitted
- 11 Jun 2018
- Last updated
- 2 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03595371
- Acronym / sponsor ID
- OLT1177-07
- Conditions
- Schnitzler Syndrome
- Keywords
- Schnitzler
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated due to study design issues that did not allow for determination of efficacy and safety in subjects with Schnitzler Syndrome who are currently well controlled on anakinra therapy.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Olatec Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Radboud University Medical Center
- Study start
- 15 May 2018 (actual)
- Primary completion
- 29 May 2018 (actual)
- Study completion
- 29 May 2018 (actual)
- First posted
- 23 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| dapansutrile capsules | Experimental | Drug: dapansutrileHard gelatin capsules containing 100 mg of dapansutrile (API) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (8 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| dapansutrileAlso: OLT1177 capsules500 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. | Drug | dapansutrile capsules |
Primary outcome
Schnitzler's Syndrome Symptom Index
Time frame: Day 14
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".
15 secondary outcomes
Physical Examination
Time frame: Day 14
Vital signs - pulse
Time frame: Day 14
Vital signs - temperature
Time frame: Day 14
Vital signs - respirations
Time frame: Day 14
Vital signs - blood pressure
Time frame: Day 14
Safety laboratory measures - chemistry markers
Time frame: Day 14
Safety laboratory measures - hematology/complete blood count
Time frame: Day 14
Safety laboratory measures - urinalysis
Time frame: Day 14
Adverse events
Time frame: Up to 42 days
Photographs of posterior torso
Time frame: Day 14
Investigator Global Assessment of Disease Activity
Time frame: Later of Day 14 or symptom relapse (expected no later than Day 28)
Subject Global Assessment of Disease Activity
Time frame: Later of Day 14 or symptom relapse (expected no later than Day 28)
Subject Skin Assessment
Time frame: Later of Day 14 or symptom relapse (expected no later than Day 28)
Time to relapse of SchS Symptoms after cessation of dapansutrile
Time frame: Later of Day 14 or symptom relapse (expected no later than Day 28)
Subject Global Evaluation of Treatment
Time frame: Later of Day 14 or symptom relapse (expected no later than Day 28)
Results and safety
The study was stopped early (reason given: The study was terminated due to study design issues that did not allow for determination of efficacy and safety in subjects with Schnitzler Syndrome who are currently well controlled on anakinra therapy.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Curt Scribner, MD study chair
Olatec Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing dapansutrile in people with Schnitzler Syndrome, run by Olatec Therapeutics, Inc. at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The study was terminated due to study design issues that did not allow for determination of efficacy and safety in subjects with Schnitzler Syndrome who are currently well controlled on anakinra therapy.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Schnitzler's Syndrome Symptom Index (time frame: Day 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2024.