Familial Mediterranean FeverPhase 2NCT05190991
Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever
A Phase 2 study testing RPH-104 in people with Familial Mediterranean Fever and FMF, run by R-Pharm International, LLC at 8 sites in 3 countries.
Trial summary
The purpose of this study is to assess safety and efficacy of the long-term treatment with RPH-104 at doses of 80 or 160 mg once every 2 weeks (q2w) in patients with familial Mediterranean fever (FMF) with colchicine resistance or intolerance (i.e. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Familial Mediterranean Fever, FMF
- What is being tested?
- RPH-104
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 8 sitesin 3 countries
- Study dates
- 5 Oct 2021to Jan 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The patient with Familial Mediterranean Fever (FMF) with resistance to or intolerance of colchicine, who completed the core study, during which he/she received at least one dose of RPH-104.
- Voluntarily signed and dated Patient Informed Consent Form (ICF) for participation in this study.
- The patient's ability and desire, according to the Investigator's discretion, to follow the schedule of visits, follow the study procedures and follow the Protocol requirements, including the following:
- to visit the study site every 2 weeks for RPH-104 administration by qualified study site personnel
- or
- to learn the subcutaneous (SC) injection technique and self-administer RPH-104 at his/her accommodation as per the study Protocol
- or
- to agree with the qualified medical personnel visits to his/her accommodation for RPH-104 administration.
Who cannot join
- Any medically significant event that was observed in a patient during his/her participation in the core study, as well as any other medical conditions (including psychiatric disorders) or laboratory abnormalities, which may increase the potential risk associated with participation in the study and treatment with RPH-104, or may affect the interpretation of the study results, and which, according to the Investigator's opinion, may lead to the patient's non-compliance with the study inclusion criteria.
- Pregnant and/or lactating women or women planning pregnancy during the study or within 2 months after the last RPH-104 dose.
- Women of childbearing potential, i.e. all females with physiological ability to conceive except for those with final cessation of menses, which should be determined retrospectively after 12 months of natural amenorrhea, i.e. amenorrhea with an appropriate clinical status, for example, at respective age, who do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose or Men who are sexually active and do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose.
- Highly effective contraception methods include:
- complete abstinence: if it corresponds to the preferred and conventional lifestyle of the subject. Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation method) and interrupted coitus are not considered acceptable contraceptive methods;
- female sterilization: surgical bilateral ovariectomy (with/without hysterectomy) or tubal ligation at least 6 weeks before the start of the core study. In a case of ovariectomy only, the female reproductive status should be verified by further hormonal test;
Show all 23Show fewer
- male sterilization (with documented absence of sperm in ejaculate post vasectomy) at least 6 months before the start of the core study. Vasectomized male partner should be the only partner of the participating female subject;
- combination of any two of the following methods (a+b or a+c or b+c):
- use of oral, injectable or implanted hormonal contraceptives; in a case of oral contraceptives, the woman should constantly use the same product as was used during the core study;
- installation of an intrauterine device or contraceptive system;
- use of barrier contraceptives: condom or occlusive cap (diaphragm or cervical cap/contraceptive vaginal ring) with spermicidal foam/gel/film/cream/vaginal suppository
- The need for a therapy with any of the following products from the moment of signing the ICF till the study treatment period completion:
- systemic glucocorticoids at a dose exceeding 0,2 mg/kg/day of prednisolone (or 0,16 mg/kg/day of methylprednisolone, or an equivalent dose of another glucocorticoid) orally;
- rilonacept, tocilizumab, rituximab, canakinumab, tumor necrosis factor alpha (TNF-a) inhibitors (TNFi) and other biological products (except for RPH-104);
- immunosuppressants (cyclosporine, methotrexate, leflunomide, thalidomide, azathioprine, 6-mercaptopurine, cyclophosphamide, etc.);
- methylprednisolone (or an equivalent) at a dose of more than 40 mg/day parenterally;
- intramuscular, intra-articular or peri-articular administration of glucocorticoids;
- anakinra;
- tofacitinib, baricitinib;
- any experimental drugs (except for RPH-104)
- The need to use a live (attenuated) vaccine during the study or within 3 months after the last RPH-104 dose. Live attenuated vaccines include vaccines against viruses: measles, rubella, mumps, chickenpox, rotavirus, flu (as a nasal spray), yellow fever, polio (oral polio vaccine); vaccines against tuberculosis (BCG), typhoid fever (oral typhoid vaccine) and typhus (typhus vaccine). Immunocompetent family members of the patient should not be vaccinated with the oral polio vaccine during the patient's participation in the study
- Positive results of tuberculosis screening performed at Visit 10 of the core study (QuantiFERON-Tuberculosis(TB)/T-Spot.TB test, chest X-ray).
- Participation in other experimental studies (except for the core study).
Trial locations and contacts
8 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
8 sites in 3 countries
Russia4 sites, 4 recruiting
- Recruiting
FSBEI HE First Moscow State Medical University named after I.M. Sechenov
Moscow, 119048
- Recruiting
Moscow Multidisciplinary Scientific and Clinical Center named after S.P. Botkin
Moscow, 125284
- Recruiting
Medical Technologies Ltd.
Saint Petersburg, 191025
- Recruiting
Terafarm, Llc
Stavropol, 355000
Turkey (Türkiye)3 sites, 3 recruiting
- Recruiting
Hacettepe University Faculty of Medicine
Ankara, 06230
- Recruiting
Istanbul University Istanbul Faculty of Medicine
Istanbul, 34093
- Recruiting
Istanbul University Cerrahpasa Faculty of Medicine
Istanbul, 34098
Armenia1 site, 1 recruiting
- Recruiting
Center of Medical Genetics and Primary Health Care LLC
Yerevan, 0001
About this study
Official title: An International Multicenter Open-label Clinical Study of the Safety and Efficacy of RPH-104 for Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine
The purpose of this study is to assess safety and efficacy of the long-term treatment with RPH-104 at doses of 80 or 160 mg once every 2 weeks (q2w) in patients with familial Mediterranean fever (FMF) with colchicine resistance or intolerance (i.e. colchicine resistant, crFMF), who completed the core study, during which they received at least one dose of RPH-104 (i.e. study patient population).
Read the detailed description
This study is a long-term open-label extension (OLE) of the core double blind randomized placebo-controlled study CL04018065. This OLE study will have the following periods:
- Screening period - 2 weeks (from signing an Informed Consent Form [ICF] at Visit 10 of the core study to Visit 1, Day 0 of this OLE study) - includes unblinding of the treatment groups determined in the core study (unless was unblinded previously) and determination of the eligibility criteria compliance for this study (all other screening procedures are performed after the completion of Visit 11 of the core study, which is also a Day 0 of this OLE study); All patients meeting the study inclusion/exclusion criteria according to the screening results will be transferred to the treatment period at the same day (Day 0) - the beginning of this period will be considered Visit 1 of this study
- Treatment period - All patients will be receiving RPH-104 treatment for 198 weeks in the open-label regimen (both patient and the Investigator, along with all study team will know which treatment is used) at the doses determined for them in the core study. Thus, the possible RPH-104 dose will be:
- 80 mg q2w subcutaneously (SC);
- 160 mg q2w SC The first RPH-104 administration in this study will be performed at Visit 1. To maintain the q2w dosage regimen, the screening and the first open-label injection of RPH-104 (if the patient meets the study eligibility criteria) must be performed on the same day - Day 0 (which is also the day of Visit 11 of the core study) - i.e. the first administration in this study will be performed 2 weeks (±3 days) after the last administration in the core study. Further, RPH-104 will be administered to patients q2w both during each scheduled visit to the study site (with safety and efficacy assessments according to the visits schedule), and during Drug Administration Visits (DAVs) at the study site or at the patient's accommodation An injection of the study drug to patients is performed by qualified medical personnel every 2 weeks when the patient visits the study site; it is also possible for the patients to self-administer the drug at home (for which patients will be appropriately trained and provided with the necessary quantity of the drug, materials for the injection (including special containers for their disposal) and proper drug transportation). Safety and efficacy assessments are performed at Visit 1, Visit 2 (in 2 weeks), Visit 3 (in 4 weeks), Visit 4 (in 4 weeks), Visit 5 (in 8 weeks) and thereafter every 12 weeks (Visit 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19 and 20) according to the visits schedule. In case of RPH-104 administration in patients' accommodation, telephone contacts with a patient will be conducted by the Investigator every 4 weeks in between the visits starting from the Visit 4.In case of FMF attack development, patients who receive 80 mg of the drug may be switched to the increased maximum drug dose160 mg based at the discretion of the investigator. The drug is administered only at scheduled visits. Dose reduction of RPH-104 is not allowed in this study. After patients receive the last dose of RPH-104 at Week 198 of the study, the treatment period will be considered completed and an 8-week safety follow-up will start
- Safety follow-up period - 8 weeks. During this period the patients will have to visit the study site twice in 4 weeks (Visit 21) and 8 weeks (Visit 22) after the last RPH-104 dose for safety assessments.
The last visit of the safety follow-up period (Visit 22) is the End of Study Visit; after completion of all procedures of this visit, patients will be considered to have completed the study.
- Conditions
- Familial Mediterranean Fever, FMF
- Being tested
- RPH-104
- Keywords
- RPH-104, colchicine inefficacy, colchicine intolerance, colchicine resistance, subcutaneous, FMF, interleukin-1 inhibitor
- Medical subject headings
- Familial Mediterranean Fever
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RPH-104Biological
solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial
| Study group | Role | What they receive |
|---|---|---|
| RPH-104 q2w | Experimental | RPH-104 80 mg once every 2 weeks subcutaneously or RPH-104 160 mg once every 2 weeks subcutaneously Receives: Biological: RPH-104 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (AEs), by System Organ Class and Preferred Term
Measured: Up to 62 weeks
Incidence of Treatment-Emergent Serious Adverse Events (SAEs), by System Organ Class and Preferred Term
Measured: Up to 62 weeks
Incidence of Treatment-Emergent Adverse Events of Special Interest (AESI), by System Organ Class and Preferred Term
Measured: Up to 62 weeks
Incidence rate for serious adverse events (SAEs)
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs
Measured: Up to 62 weeks
Incidence rate for adverse events of special Interest (AESI)
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for AESI
Measured: Up to 62 weeks
Secondary outcomes (16)
Percentage of patients with physician global assessment of disease activity scale (PGA) <2
PGA of disease activity involves a 5-point system: from 0 = no clinical signs and symptoms associated with the disease to 4 = severe clinical signs and symptoms associated with the disease.
Measured: Up to 62 weeks
Percentage of patients with serological remission
Serological remission is defined as C-reactive (CRP) level ≤10 mg/L.
Measured: Up to 62 weeks
Percentage of patients whose Serum amyloid A (SAA) levels returned to normal values
SAA normal levels are defined as SAA <10 mg/L
Measured: Up to 62 weeks
Percentage of patients who have experienced ≥ 1 attacks per month (since Day 0)
Criteria for the diagnosis of an attack are defined as the simultaneous development of clinical and serological signs of an attack, including:
- the score on the scale of global physician's assessment of disease activity (PGA) ≥ 2, suggesting "mild", "moderate" or "severe" activity of the disease (i.e., clinical signs), AND
- CRP level ≥ 30 mg/L (i.e. serological signs)
Measured: Up to 54 weeks
Percentage of patients who have not had a single attack
Criteria for the diagnosis of an attack are defined as the simultaneous development of clinical and serological signs of an attack, including:
- the score on the scale of global physician's assessment of disease activity (PGA) ≥ 2, suggesting "mild", "moderate" or "severe" activity of the disease (i.e., clinical signs), AND
- CRP level ≥ 30 mg/L (i.e. serological signs)
Measured: Up to 54 weeks
Percentage of patients escalated to RPH-104 160 mg q2w dose
The dose may be increased if a new attack is confirmed
Measured: Up to 54 weeks
Percentage of patients receiving additional symptomatic therapy with NSAIDs, paracetamol or glucocorticoids due to FMF
Symptomatic therapy was prescribed by the decision of the investigator upon confirmation of a new attack
Measured: Up to 62 weeks
Change in the inflammatory markers CRP compared to baseline
CRP level mg/l at each visit
Measured: Up to 62 weeks
Change in the inflammatory markers SAA compared to baseline
SAA level mg/l at each visit
Measured: Up to 62 weeks
Changes in the PGA score over time
PGA of disease activity involves a 5-point system: from 0 = no clinical signs and symptoms associated with the disease to 4 = severe clinical signs and symptoms associated with the disease.
Measured: Up to 54 weeks
Changes in the severity of chest pain based on the main disease symptoms scale.
An assessment of severity of the main symptoms of the disease will be based on a 5-point scale: 0 = no symptom, 1 = minimal, 2 = mild, 3 = moderate, 4 = severe
Measured: Up to 54 weeks
Changes in the severity of abdominal pain based on the main disease symptoms scale.
An assessment of severity of the main symptoms of the disease will be based on a 5-point scale: 0 = no symptom, 1 = minimal, 2 = mild, 3 = moderate, 4 = severe
Measured: Up to 54 weeks
Changes in the severity of arthralgia/arthritis based on the main disease symptoms scale.
An assessment of severity of the main symptoms of the disease will be based on a 5-point scale: 0 = no symptom, 1 = minimal, 2 = mild, 3 = moderate, 4 = severe
Measured: Up to 54 weeks
Changes in the severity of skin rash based on the main disease symptoms scale.
An assessment of severity of the main symptoms of the disease will be based on a 5-point scale: 0 = no symptom, 1 = minimal, 2 = mild, 3 = moderate, 4 = severe
Measured: Up to 54 weeks
Changes in the renal function in patients with impaired renal function at the screening
Impaired renal function is defined as creatinine clearance(ClCr) <90 ml/min [calculated using the Cockcroft-Gault formula] at the screening
Measured: Up to 62 weeks
Changes in proteinuria in patients with baseline proteinuria
Presence of protein in urine according to urinalysis results
Measured: Up to 62 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2022
- Study start5 Oct 2021
- Primary completionDec 2028 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Mediterranean FeverStudies 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
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- R-Pharm International, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Atlant Clinical LLC, Unimed Laboratories, Data Management 365, R-Pharm JSC, TRPharm İlaç Sanayi Ticaret Anonim Şirketi and ASCOT SCIENCE Education and Consulting Ltd., Exacte Labs LLC, Key Stat LLC
- Responsible party
- Sponsor
- Investigators
- Mikhail Samsonov, Study director, R-Pharm
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05190991
- Sponsor's protocol ID
- CL04018071
- Status last verified
- Dec 2024
- First submitted
- 3 Nov 2021
- Last updated
- 16 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International Multicenter Open-label Clinical Study of the Safety and Efficacy of RPH-104 for Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine
- ClinicalTrials.gov ID
- NCT05190991
- Acronym / sponsor ID
- CL04018071
- Conditions
- Familial Mediterranean Fever, FMF
- Keywords
- RPH-104, colchicine inefficacy, colchicine intolerance, colchicine resistance, subcutaneous, FMF, interleukin-1 inhibitor
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- R-Pharm International, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Atlant Clinical LLC, Unimed Laboratories, Data Management 365, R-Pharm JSC, TRPharm İlaç Sanayi Ticaret Anonim Şirketi and ASCOT SCIENCE Education and Consulting Ltd., Exacte Labs LLC, Key Stat LLC
- Study start
- 5 Oct 2021 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 13 Jan 2022 (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 purpose of this study is to assess safety and efficacy of the long-term treatment with RPH-104 at doses of 80 or 160 mg once every 2 weeks (q2w) in patients with familial Mediterranean fever (FMF) with colchicine resistance or intolerance (i.e. colchicine resistant, crFMF), who completed the core study, during which they received at least one dose of RPH-104 (i.e. study patient population).
Detailed description
This study is a long-term open-label extension (OLE) of the core double blind randomized placebo-controlled study CL04018065. This OLE study will have the following periods:
- Screening period - 2 weeks (from signing an Informed Consent Form [ICF] at Visit 10 of the core study to Visit 1, Day 0 of this OLE study) - includes unblinding of the treatment groups determined in the core study (unless was unblinded previously) and determination of the eligibility criteria compliance for this study (all other screening procedures are performed after the completion of Visit 11 of the core study, which is also a Day 0 of this OLE study); All patients meeting the study inclusion/exclusion criteria according to the screening results will be transferred to the treatment period at the same day (Day 0) - the beginning of this period will be considered Visit 1 of this study
- Treatment period - All patients will be receiving RPH-104 treatment for 198 weeks in the open-label regimen (both patient and the Investigator, along with all study team will know which treatment is used) at the doses determined for them in the core study. Thus, the possible RPH-104 dose will be:
- 80 mg q2w subcutaneously (SC);
- 160 mg q2w SC The first RPH-104 administration in this study will be performed at Visit 1. To maintain the q2w dosage regimen, the screening and the first open-label injection of RPH-104 (if the patient meets the study eligibility criteria) must be performed on the same day - Day 0 (which is also the day of Visit 11 of the core study) - i.e. the first administration in this study will be performed 2 weeks (±3 days) after the last administration in the core study. Further, RPH-104 will be administered to patients q2w both during each scheduled visit to the study site (with safety and efficacy assessments according to the visits schedule), and during Drug Administration Visits (DAVs) at the study site or at the patient's accommodation An injection of the study drug to patients is performed by qualified medical personnel every 2 weeks when the patient visits the study site; it is also possible for the patients to self-administer the drug at home (for which patients will be appropriately trained and provided with the necessary quantity of the drug, materials for the injection (including special containers for their disposal) and proper drug transportation). Safety and efficacy assessments are performed at Visit 1, Visit 2 (in 2 weeks), Visit 3 (in 4 weeks), Visit 4 (in 4 weeks), Visit 5 (in 8 weeks) and thereafter every 12 weeks (Visit 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19 and 20) according to the visits schedule. In case of RPH-104 administration in patients' accommodation, telephone contacts with a patient will be conducted by the Investigator every 4 weeks in between the visits starting from the Visit 4.In case of FMF attack development, patients who receive 80 mg of the drug may be switched to the increased maximum drug dose160 mg based at the discretion of the investigator. The drug is administered only at scheduled visits. Dose reduction of RPH-104 is not allowed in this study. After patients receive the last dose of RPH-104 at Week 198 of the study, the treatment period will be considered completed and an 8-week safety follow-up will start
- Safety follow-up period - 8 weeks. During this period the patients will have to visit the study site twice in 4 weeks (Visit 21) and 8 weeks (Visit 22) after the last RPH-104 dose for safety assessments.
The last visit of the safety follow-up period (Visit 22) is the End of Study Visit; after completion of all procedures of this visit, patients will be considered to have completed the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RPH-104 q2w | Experimental | Biological: RPH-104RPH-104 80 mg once every 2 weeks subcutaneously or RPH-104 160 mg once every 2 weeks subcutaneously |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- The patient with Familial Mediterranean Fever (FMF) with resistance to or intolerance of colchicine, who completed the core study, during which he/she received at least one dose of RPH-104.
- Voluntarily signed and dated Patient Informed Consent Form (ICF) for participation in this study.
- The patient's ability and desire, according to the Investigator's discretion, to follow the schedule of visits, follow the study procedures and follow the Protocol requirements, including the following:
- or
- to learn the subcutaneous (SC) injection technique and self-administer RPH-104 at his/her accommodation as per the study Protocol
Key exclusion criteria (23 listed)
- Any medically significant event that was observed in a patient during his/her participation in the core study, as well as any other medical conditions (including psychiatric disorders) or laboratory abnormalities, which may increase the potential risk associated with participation in the study and treatment with RPH-104, or may affect the interpretation of the study results, and which, according to the Investigator's opinion, may lead to the patient's non-compliance with the study inclusion criteria.
- Pregnant and/or lactating women or women planning pregnancy during the study or within 2 months after the last RPH-104 dose.
- Women of childbearing potential, i.e. all females with physiological ability to conceive except for those with final cessation of menses, which should be determined retrospectively after 12 months of natural amenorrhea, i.e. amenorrhea with an appropriate clinical status, for example, at respective age, who do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose or Men who are sexually active and do not agree to use highly effective contraceptives throughout the study, starting from the moment of signing the ICF and for at least 8 weeks after the last RPH-104 dose.
- The need for a therapy with any of the following products from the moment of signing the ICF till the study treatment period completion:
- The need to use a live (attenuated) vaccine during the study or within 3 months after the last RPH-104 dose. Live attenuated vaccines include vaccines against viruses: measles, rubella, mumps, chickenpox, rotavirus, flu (as a nasal spray), yellow fever, polio (oral polio vaccine); vaccines against tuberculosis (BCG), typhoid fever (oral typhoid vaccine) and typhus (typhus vaccine). Immunocompetent family members of the patient should not be vaccinated with the oral polio vaccine during the patient's participation in the study
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 |
|---|---|---|
| RPH-104solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial | Biological | RPH-104 q2w |
Primary outcome
Incidence of Treatment-Emergent Adverse Events (AEs), by System Organ Class and Preferred Term
Time frame: Up to 62 weeks
Incidence of Treatment-Emergent Serious Adverse Events (SAEs), by System Organ Class and Preferred Term
Time frame: Up to 62 weeks
Incidence of Treatment-Emergent Adverse Events of Special Interest (AESI), by System Organ Class and Preferred Term
Time frame: Up to 62 weeks
Incidence rate for serious adverse events (SAEs)
Time frame: Up to 62 weeks
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs
Incidence rate for adverse events of special Interest (AESI)
Time frame: Up to 62 weeks
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for AESI
16 secondary outcomes
Percentage of patients with physician global assessment of disease activity scale (PGA) <2
Time frame: Up to 62 weeks
Percentage of patients with serological remission
Time frame: Up to 62 weeks
Percentage of patients whose Serum amyloid A (SAA) levels returned to normal values
Time frame: Up to 62 weeks
Percentage of patients who have experienced ≥ 1 attacks per month (since Day 0)
Time frame: Up to 54 weeks
Percentage of patients who have not had a single attack
Time frame: Up to 54 weeks
Percentage of patients escalated to RPH-104 160 mg q2w dose
Time frame: Up to 54 weeks
Percentage of patients receiving additional symptomatic therapy with NSAIDs, paracetamol or glucocorticoids due to FMF
Time frame: Up to 62 weeks
Change in the inflammatory markers CRP compared to baseline
Time frame: Up to 62 weeks
Change in the inflammatory markers SAA compared to baseline
Time frame: Up to 62 weeks
Changes in the PGA score over time
Time frame: Up to 54 weeks
Changes in the severity of chest pain based on the main disease symptoms scale.
Time frame: Up to 54 weeks
Changes in the severity of abdominal pain based on the main disease symptoms scale.
Time frame: Up to 54 weeks
Changes in the severity of arthralgia/arthritis based on the main disease symptoms scale.
Time frame: Up to 54 weeks
Changes in the severity of skin rash based on the main disease symptoms scale.
Time frame: Up to 54 weeks
Changes in the renal function in patients with impaired renal function at the screening
Time frame: Up to 62 weeks
Changes in proteinuria in patients with baseline proteinuria
Time frame: Up to 62 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Dec 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.
Mikhail Samsonov study director
R-Pharm
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RPH-104 in people with Familial Mediterranean Fever and FMF, run by R-Pharm International, LLC at 8 sites in 3 countries.
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 8 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: looking for participants now, as of the sponsor's last update on 16 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Russia, Turkey (Türkiye), Armenia. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yulia Karpova (+7 495 956 79 37 ext. 1197, jn.karpova@inbox.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (AEs), by System Organ Class and Preferred Term (time frame: Up to 62 weeks). The study lists 5 primary outcomes in all. 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 16 Dec 2024.