Schnitzler SyndromePhase 2NCT04213274
Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome
A Phase 2 study testing 80 mg RPH-104 in people with Schnitzler Syndrome and Urticarial Vasculitis With Monoclonal Immunoglobulin M Component, Schnitzler (Disorder), run by R-Pharm Overseas, Inc. at 4 sites in United States.
Trial summary
The primary goal of the study is to assess the efficacy and safety of RPH-104 in subjects with Schnitzler Syndrome using Schnitzler Disease Activity Score (SDAS), which includes the Physician's Global Assessment (PGA) and the local laboratory C-reactive protein (CRP) result Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Schnitzler Syndrome, Urticarial Vasculitis With Monoclonal Immunoglobulin M Component, Schnitzler (Disorder)
- What is being tested?
- 80 mg RPH-104
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 4 sitesUnited States
- Study dates
- May 2023to Jan 2024
- 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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Able to read, understand and willing to sign the ICF and abide with study procedures. The ICF must be signed and dated prior to performing any Screening assessment.
- 2. Confirmed diagnosis of SchS based on diagnostic criteria adapted by Lipsker as an urticarial skin rash (chronic), monoclonal IgM component (ie, IgM < 10 g/L), or IgG (variant type), and at least of 2 of the following:
- Fever (intermittent)
- Arthralgia or arthritis
- Bone pain
- Lymphadenopathy
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- Hepato and/or splenomegaly
- Elevated erythrocyte sedimentation rate (ESR) and/or leukocytosis
- Bone abnormalities (on radiological or histological investigation)
- 3. Subjects with symptomatic SchS (as defined by SDAS with a score of 2 or more with a CRP [local laboratory] levels > 10 mg/L) on the day of randomization.
- 4. Willing, committed, and able to return for all clinic visits and complete all study related procedures, including willingness to have SC injections administered by a qualified person.
- 5. Males, female partners of sexually active male subjects, and women of childbearing potential (WOCBP) (defined as all female subjects with physiological potential to conceive) must agree to use highly effective contraceptive methods throughout the study starting from Screening (signing informed consent) through at least 8 weeks after the final dose of study drug.
- Highly effective contraceptive method is defined as follows:
- Complete abstinence: if it corresponds to the preferred and conventional lifestyle of the female subject. Periodic abstinence (eg, calendar, ovulation, symptothermal, postovulation method) and interrupted coitus are not considered acceptable contraceptive methods.
- Sterilization: surgical bilateral ovariectomy (with/without hysterectomy) or tubal ligation at least 6 weeks prior to the study therapy initiation. In case of ovariectomy only the female reproductive status should be verified by further hormonal test.
- Sterilization of male partner with documented absence of sperm in ejaculate post vasectomy for at least 6 months (vasectomized male partner should be the only partner of the participating female subject).
- Combination of 2 of the following methods (i + ii, i + iii, or ii + iii):
- Oral, injectable, or implanted hormonal contraceptives; in case of oral contraceptives the female subjects should administer the same product for at least 3 months prior to the study therapy.
- Intrauterine device or contraceptive system.
- Barrier methods: condom or occlusive cap (diaphragm or cervical cap/vaginal fornix cap) with spermicidal foam/gel/film/cream/vaginal suppository.
- 6. WOCBP must have negative pregnancy tests at Screening (serum chorionic gonadotropin test), and all subsequent visits (urine).
Who cannot join
- 1. Hypersensitivity to the study drug (RPH 104) and/or its components/excipients and/or the products of the same chemical class.
- 2. Concurrent/on going treatment with anakinra (Kineret) or recent treatment within 5 days prior to Day 0.
- 3. Concurrent/on going treatment with other biologics or recent treatment within 4 weeks or 5 × t½ prior to Day 0, whichever is longer.
- 4. Concurrent/on going treatment with immunosuppressive agents (eg, cyclosporine, methotrexate, dapsone, colchicine, or others) within 4 weeks or 5 × t½ prior to Day 0, whichever is longer.
- 5. Concurrent/on going treatment with high doses of systemic steroids (> 0.2 mg/kg/day prednisolone equivalent). Intra articular, peri articular, intravenous, or intramuscular corticosteroid injections within 4 weeks prior to the Day 0 visit.
- 6. Administration of live (attenuated) vaccine within 3 months prior to Day 0 and necessity of live vaccine administration for 3 months after Day 70.
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- 7.
- History of active tuberculosis (TB), evidence of active or latent M. tuberculosis infection as defined by local guidelines/local medical practice at Screening.
- Positive QuantiFERON Gold Plus (TB) test at Screening.
- Chest X ray findings confirming pulmonary TB at Screening.
- 8. Active bacterial, fungal, or viral infection(s) within 4 weeks prior to Day 0.
- 9. A history of persistent chronic bacterial, fungal, or viral infection(s) requiring treatment with parenteral antibiotics, parenteral antivirals, or parenteral antifungals within 4 weeks prior to Day 0.
- 10. Opportunistic infections and/or Kaposi's sarcoma at the time of Screening.
- 11. Any other relevant concomitant diseases (infectious, cardiovascular, nervous, endocrine, urinary, gastrointestinal, hepatic disorders, coagulation disorders, and other autoimmune/autoinflammatory diseases, etc) or conditions which, according to the investigator's judgment, may affect the subject's participation or well being in the study and/or distort assessment of the study results.
- 12. History of malignancies within 5 years prior to screening other than successfully treated non metastatic, basal, or squamous cell carcinoma of the skin and/or in situ cancer.
- 13. Evidence of lymphoproliferative diseases (except SchS associated monoclonal gammopathy).
- 14. Presence of any of the following laboratory abnormalities at screening visit: white blood cell count (WBC) < 3000/µL; platelet count < 75,000/µL; alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3.0 × upper limit of normal (ULN), TBil > 2 × ULN unless due to Gilbert's syndrome.
- 15. Severe renal failure: Cockcroft Gault creatinine clearance < 30 mL/min.
- 16. Women who are either pregnant or lactating.
- 17. Subjects for whom there is concern about compliance with the protocol procedures.
- 18. Psychiatric disorders which, according to the investigator's judgment, may affect the subject participation in the study and his/her ability to follow the protocol procedures.
- 19. History of organ transplantation or transplantation is anticipated during the study.
- 20. Concomitant participation in other clinical studies at the start of Screening or administration of any unauthorized (investigational) products less than 4 weeks or 5 × t½ periods (whichever is longer) before Day 0 (treatment initiation).
Trial locations and contacts
4 sites in 1 country.
United States4 sites
National Jewish Health
Denver, Colorado, 80206
Penn State Health Hersey Medical Center
Hershey, Pennsylvania, 17033
Oregon Health & Science University
Portland, Oregon, 97239-3098
Mayo Clinic
Rochester, Minnesota, 55905
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2 Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome
The primary goal of the study is to assess the efficacy and safety of RPH-104 in subjects with Schnitzler Syndrome using Schnitzler Disease Activity Score (SDAS), which includes the Physician's Global Assessment (PGA) and the local laboratory C-reactive protein (CRP) result
Read the detailed description
The study will include a screening period (up to 28 days), a double blind, placebo controlled treatment period (14 days), followed by an 8 week safety follow up period after the second dose of study drug.
Subjects with an established diagnosis of Schnitzler Syndrome (SchS) will participate in a Screening period of up to 28 days after signing an informed consent form (ICF). Subjects will be advised to schedule the Day 0 visit with the study staff as soon as the symptoms of SchS flare occur during the Screening period.
If the subject does not meet the inclusion/exclusion criteria at the site visit during the screening period, other screening visits are allowed within this period until the subject meets the study criteria.
A total of 14 subjects will be randomly assigned to 1 of 2 treatment groups (in a 1:1 ratio) to receive a double blind, single subcutaneous (SC) injection of study drug (80 mg RPH-104 or matching placebo) on Day 0. On Day 14, subjects will receive a second dose of randomized treatment as well as an additional dose of 80 mg RPH-104 or placebo based on response to treatment, such that responders (SDAS = 0) will receive a dose of placebo, and non responders defined as no response (no change in SDAS) or partial responders (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) will receive an 80 mg RPH 104 dose. (Schnitzler Disease Activity Score includes the PGA score and C-reactive protein (CRP) result: If the PGA and CRP grades are not of the same severity, the higher severity level of either PGA or CRP will be used to determine the SDAS)
The study will be stopped after Data and Safety Monitoring Board (DSMB) review if:
- Death is reported in 2 subjects assigned to the RPH 104 treatment arm or after receiving RPH 104 on Day 14 (i.e., non responder to the Day 0 study drug dose).
- Two subjects assigned to the RPH 104 treatment arm or after receiving RPH 104 on Day 14 (ie, non responder to the Day 0 study drug dose) report liver function test abnormalities which meet Hy's Law criteria (ie, increased ALT and/or AST 3 fold or greater from upper limit of normal (ULN) combined with jaundice or weakness or hypochondrial pain/severity or increased Total Bilirubin (TBil) level 2 fold or greater from ULN, [not explained by any other causative factors like virus]).
The severity of SchS symptoms will be assessed daily from Day 0 through Day 28 using the PR SchS Scale, at clinic visits on Day 0, Day 14, and Day 28 using the Patient Global Impression of Severity (PGIS), at clinic visits on Day 14 and Day 28 using the Patient Global Impression of Change (PGIC), and at each clinic visit using the PGA, CRP, SAA, and SDAS. Subjects will be followed through Day 70 for safety (A Data Safety Monitoring Board (DSMB) will periodically review and evaluate the accumulated study data for subject safety).
- Conditions
- Schnitzler Syndrome, Urticarial Vasculitis With Monoclonal Immunoglobulin M Component, Schnitzler (Disorder)
- Being tested
- 80 mg RPH-104, Placebo
- Keywords
- neutrophilic urticarial dermatosis, autoinflammatory syndrome, monoclonal gammopathy, neutrophilic dermatosis, interleukin-1, Schnitzler syndrome
- Medical subject headings
- Schnitzler Syndrome, Paraproteinemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
80 mg RPH-104Biological
solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial
PlaceboDrug
Normal Saline (0.9% Sodium Chloride solution for Injection), 2 mL in the 4 mL-glass vial
| Study group | Role | What they receive |
|---|---|---|
| Placebo - placebo | Placebo comparator | Subject randomized to receive subcutaneous single injection of placebo on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the positive response to treatment (SDAS = 0) one more dose of placebo Receives: Drug: Placebo |
| Placebo - 80 mg RPH-104 | Other | Subject randomized to receive subcutaneous single injection of placebo on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the lack of response (no change in SDAS) or partial response to treatment (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) subcutaneous single injection of 80 mg RPH-104 Receives: Biological: 80 mg RPH-104; Drug: Placebo |
| 80 mg RPH-104 - 80 mg RPH-104 | Experimental | Subject randomized to receive subcutaneous single injection of 80 mg RPH-104 on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the positive response to the treatment (SDAS = 0) one subcutaneous injection of placebo Receives: Biological: 80 mg RPH-104; Drug: Placebo |
| 80 mg RPH-104 - 160 mg RPH-104 | Experimental | Subject randomized to receive subcutaneous single injection of 80 mg RPH-104 on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the lack of response (no change in SDAS) or partial response to treatment (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) one more subcutaneous injection of 80 mg RPH-104 Receives: Biological: 80 mg RPH-104 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects with complete response (Schnitzler Disease Activity Score (SDAS = 0)) to therapy on Day 14 in the RPH-104 group as compared to the placebo group
- Proportion of subjects with complete response (SDAS = 0) to therapy on Day 14 in the RPH 104 treated group as compared to the placebo group based on SDAS using the PGA and the local laboratory CRP result
Measured: Day 14
Secondary outcomes (5)
Change from baseline to Day 14 in subject-reported symptom severity of SchS: Patient-reported Severity of Schnitzler Syndrome Scale (PR-SchS Scale)
Change from baseline to Day 14 in subject-reported symptom severity of SchS using the Patient-reported Severity of Schnitzler Syndrome Scale (PR-SchS Scale). The PR-SchS Scale includes 5 features that are the most common and important SchS symptoms. These include rash, fever, tiredness, joint pain, and bone/muscle pain.
Measured: Baseline and Day 14
Proportion of subjects with normalized serum amyloid A (SAA) and C-reactive protein (CRP) at Days 14 and 28
Proportion of subjects with normalized SAA (< 10 mg/L) and CRP (≤ 10 mg/L) at Days 14 and 28
Measured: Day 14 and Day 28
Change from baseline to Day 14 and Day 28 in CRP and SAA
Change from baseline to Day 14 and Day 28 in C-reactive protein and serum amyloid A
Measured: Baseline, Day 28 and Day 14
Proportion of subjects with complete response (SDAS = 0) to therapy on Day 28 by treatment sequence based on SDAS using the Physician's Global Assessment (PGA) and the CRP result
Proportion of subjects with complete response (SDAS = 0) to therapy on Day 28 by treatment sequence (placebo - placebo, placebo - 80 mg RPH 104, 80 mg RPH 104 80 mg RPH 104, and 80 mg RPH 104 - 160 mg RPH 104) based on SDAS using the PGA and the CRP result.
Measured: Baseline and Day 28
Proportion of subjects with partial response by treatment sequence on Days 14 and 28
Proportion of subjects with partial response (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) by treatment sequence (placebo - placebo, placebo 80 mg RPH 104, 80 mg RPH 104 80 mg RPH 104, and 80 mg RPH 104 160 mg RPH 104) on Days 14 and 28
Measured: Baseline, Day 14 and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2019
- Study startMay 2023 (estimated)
- Primary completionAug 2023 (estimated)
- Study completionJan 2024 (estimated)
Why it stopped: due to the decision of company Top management
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- R-Pharm Overseas, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Responsible party
- Sponsor
- Investigators
- Yan Lavrovsky, Study director, R-Pharm Overseas, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04213274
- Sponsor's protocol ID
- CL04018066
- Status last verified
- Nov 2024
- First submitted
- 22 Oct 2019
- Last updated
- 15 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2 Study of the Efficacy and Safety of RPH-104 in Adult Subjects With Schnitzler Syndrome
- ClinicalTrials.gov ID
- NCT04213274
- Acronym / sponsor ID
- CL04018066
- Conditions
- Schnitzler Syndrome, Urticarial Vasculitis With Monoclonal Immunoglobulin M Component, Schnitzler (Disorder)
- Keywords
- neutrophilic urticarial dermatosis, autoinflammatory syndrome, monoclonal gammopathy, neutrophilic dermatosis, interleukin-1, Schnitzler syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: due to the decision of company Top management
- Enrolment
- 0 participants (actual)
- Lead sponsor
- R-Pharm Overseas, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Study start
- May 2023 (estimated)
- Primary completion
- Aug 2023 (estimated)
- Study completion
- Jan 2024 (estimated)
- First posted
- 30 Dec 2019 (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.
The primary goal of the study is to assess the efficacy and safety of RPH-104 in subjects with Schnitzler Syndrome using Schnitzler Disease Activity Score (SDAS), which includes the Physician's Global Assessment (PGA) and the local laboratory C-reactive protein (CRP) result
Detailed description
The study will include a screening period (up to 28 days), a double blind, placebo controlled treatment period (14 days), followed by an 8 week safety follow up period after the second dose of study drug.
Subjects with an established diagnosis of Schnitzler Syndrome (SchS) will participate in a Screening period of up to 28 days after signing an informed consent form (ICF). Subjects will be advised to schedule the Day 0 visit with the study staff as soon as the symptoms of SchS flare occur during the Screening period.
If the subject does not meet the inclusion/exclusion criteria at the site visit during the screening period, other screening visits are allowed within this period until the subject meets the study criteria.
A total of 14 subjects will be randomly assigned to 1 of 2 treatment groups (in a 1:1 ratio) to receive a double blind, single subcutaneous (SC) injection of study drug (80 mg RPH-104 or matching placebo) on Day 0. On Day 14, subjects will receive a second dose of randomized treatment as well as an additional dose of 80 mg RPH-104 or placebo based on response to treatment, such that responders (SDAS = 0) will receive a dose of placebo, and non responders defined as no response (no change in SDAS) or partial responders (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) will receive an 80 mg RPH 104 dose. (Schnitzler Disease Activity Score includes the PGA score and C-reactive protein (CRP) result: If the PGA and CRP grades are not of the same severity, the higher severity level of either PGA or CRP will be used to determine the SDAS)
The study will be stopped after Data and Safety Monitoring Board (DSMB) review if:
- Death is reported in 2 subjects assigned to the RPH 104 treatment arm or after receiving RPH 104 on Day 14 (i.e., non responder to the Day 0 study drug dose).
- Two subjects assigned to the RPH 104 treatment arm or after receiving RPH 104 on Day 14 (ie, non responder to the Day 0 study drug dose) report liver function test abnormalities which meet Hy's Law criteria (ie, increased ALT and/or AST 3 fold or greater from upper limit of normal (ULN) combined with jaundice or weakness or hypochondrial pain/severity or increased Total Bilirubin (TBil) level 2 fold or greater from ULN, [not explained by any other causative factors like virus]).
The severity of SchS symptoms will be assessed daily from Day 0 through Day 28 using the PR SchS Scale, at clinic visits on Day 0, Day 14, and Day 28 using the Patient Global Impression of Severity (PGIS), at clinic visits on Day 14 and Day 28 using the Patient Global Impression of Change (PGIC), and at each clinic visit using the PGA, CRP, SAA, and SDAS. Subjects will be followed through Day 70 for safety (A Data Safety Monitoring Board (DSMB) will periodically review and evaluate the accumulated study data for subject safety).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo - placebo | Placebo comparator | Drug: PlaceboSubject randomized to receive subcutaneous single injection of placebo on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the positive response to treatment (SDAS = 0) one more dose of placebo |
| Placebo - 80 mg RPH-104 | Other | Biological: 80 mg RPH-104, Drug: PlaceboSubject randomized to receive subcutaneous single injection of placebo on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the lack of response (no change in SDAS) or partial response to treatment (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) subcutaneous single injection of 80 mg RPH-104 |
| 80 mg RPH-104 - 80 mg RPH-104 | Experimental | Biological: 80 mg RPH-104, Drug: PlaceboSubject randomized to receive subcutaneous single injection of 80 mg RPH-104 on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the positive response to the treatment (SDAS = 0) one subcutaneous injection of placebo |
| 80 mg RPH-104 - 160 mg RPH-104 | Experimental | Biological: 80 mg RPH-104Subject randomized to receive subcutaneous single injection of 80 mg RPH-104 on Day 0,and then, on Day 14 - second dose of randomized treatment + based on the lack of response (no change in SDAS) or partial response to treatment (SDAS ≥ 1, and activity reduction by 1 or more points of SDAS compared to baseline) one more subcutaneous injection of 80 mg RPH-104 |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- 1. Able to read, understand and willing to sign the ICF and abide with study procedures. The ICF must be signed and dated prior to performing any Screening assessment.
- 2. Confirmed diagnosis of SchS based on diagnostic criteria adapted by Lipsker as an urticarial skin rash (chronic), monoclonal IgM component (ie, IgM < 10 g/L), or IgG (variant type), and at least of 2 of the following:
- 3. Subjects with symptomatic SchS (as defined by SDAS with a score of 2 or more with a CRP [local laboratory] levels > 10 mg/L) on the day of randomization.
- 4. Willing, committed, and able to return for all clinic visits and complete all study related procedures, including willingness to have SC injections administered by a qualified person.
- 5. Males, female partners of sexually active male subjects, and women of childbearing potential (WOCBP) (defined as all female subjects with physiological potential to conceive) must agree to use highly effective contraceptive methods throughout the study starting from Screening (signing informed consent) through at least 8 weeks after the final dose of study drug.
Key exclusion criteria (23 listed)
- 1. Hypersensitivity to the study drug (RPH 104) and/or its components/excipients and/or the products of the same chemical class.
- 2. Concurrent/on going treatment with anakinra (Kineret) or recent treatment within 5 days prior to Day 0.
- 3. Concurrent/on going treatment with other biologics or recent treatment within 4 weeks or 5 × t½ prior to Day 0, whichever is longer.
- 4. Concurrent/on going treatment with immunosuppressive agents (eg, cyclosporine, methotrexate, dapsone, colchicine, or others) within 4 weeks or 5 × t½ prior to Day 0, whichever is longer.
- 5. Concurrent/on going treatment with high doses of systemic steroids (> 0.2 mg/kg/day prednisolone equivalent). Intra articular, peri articular, intravenous, or intramuscular corticosteroid injections within 4 weeks prior to the Day 0 visit.
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 |
|---|---|---|
| 80 mg RPH-104solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial | Biological | 80 mg RPH-104 - 160 mg RPH-104, 80 mg RPH-104 - 80 mg RPH-104, Placebo - 80 mg RPH-104 |
| PlaceboNormal Saline (0.9% Sodium Chloride solution for Injection), 2 mL in the 4 mL-glass vial | Drug | 80 mg RPH-104 - 80 mg RPH-104, Placebo - 80 mg RPH-104, Placebo - placebo |
Primary outcome
Proportion of subjects with complete response (Schnitzler Disease Activity Score (SDAS = 0)) to therapy on Day 14 in the RPH-104 group as compared to the placebo group
Time frame: Day 14
• Proportion of subjects with complete response (SDAS = 0) to therapy on Day 14 in the RPH 104 treated group as compared to the placebo group based on SDAS using the PGA and the local laboratory CRP result
5 secondary outcomes
Change from baseline to Day 14 in subject-reported symptom severity of SchS: Patient-reported Severity of Schnitzler Syndrome Scale (PR-SchS Scale)
Time frame: Baseline and Day 14
Proportion of subjects with normalized serum amyloid A (SAA) and C-reactive protein (CRP) at Days 14 and 28
Time frame: Day 14 and Day 28
Change from baseline to Day 14 and Day 28 in CRP and SAA
Time frame: Baseline, Day 28 and Day 14
Proportion of subjects with complete response (SDAS = 0) to therapy on Day 28 by treatment sequence based on SDAS using the Physician's Global Assessment (PGA) and the CRP result
Time frame: Baseline and Day 28
Proportion of subjects with partial response by treatment sequence on Days 14 and 28
Time frame: Baseline, Day 14 and Day 28
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Nov 2024.
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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.
Yan Lavrovsky study director
R-Pharm Overseas, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 80 mg RPH-104 in people with Schnitzler Syndrome and Urticarial Vasculitis With Monoclonal Immunoglobulin M Component, Schnitzler (Disorder), run by R-Pharm Overseas, Inc. at 4 sites in United States.
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 21 inclusion and 23 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: withdrawn, as of the sponsor's last update on 15 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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. Some participants in this study may receive a placebo. 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 withdrawn before enrolling participants, so there are no results.
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?
Proportion of subjects with complete response (Schnitzler Disease Activity Score (SDAS = 0)) to therapy on Day 14 in the RPH-104 group as compared to the placebo group (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 15 Nov 2024.