A Phase 2 interventional study of RPH-104 in Familial Mediterranean Fever and FMF, sponsored by R-Pharm International, LLC. Recruiting at 8 sites in 3 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2024-12-16.
Sponsored by R-Pharm International, LLC (part of R-Pharm) · Phase 2, Interventional, and Treatment
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).
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:
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:
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
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.
61 studies on the registry are indexed under Familial Mediterranean Fever; 27 are open to participants now.
This study's planned enrollment of 60 is above the median of 40 across 27 interventional studies indexed under Familial Mediterranean Fever.
Browse Familial Mediterranean Fever studies →R-Pharm International, LLC is the lead sponsor of 17 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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
or
Exclusion Criteria:
Women of , 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 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:
combination of any two of the following methods (a+b or a+c or b+c):
The need for a therapy with any of the following products from the moment of signing the ICF till the study treatment period completion:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RPH-104 80 mg once every 2 weeks subcutaneously or RPH-104 160 mg once every 2 weeks subcutaneously
Biological: RPH-104
solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL glass vial
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)
Incidence rate, expressed as the number of events per 100 patient-years of follow-up, for SAEs
Time frame: 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
Time frame: Up to 62 weeks
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.
Time frame: Up to 62 weeks
Percentage of patients with serological remission
Serological remission is defined as C-reactive (CRP) level ≤10 mg/L.
Time frame: 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
Time frame: 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)
Time frame: 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)
Time frame: 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
Time frame: 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
Time frame: Up to 62 weeks
Change in the inflammatory markers CRP compared to baseline
CRP level mg/l at each visit
Time frame: Up to 62 weeks
Change in the inflammatory markers SAA compared to baseline
SAA level mg/l at each visit
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Up to 62 weeks
Changes in proteinuria in patients with baseline proteinuria
Presence of protein in urine according to urinalysis results
Time frame: Up to 62 weeks
Started Oct 2021 (actual). Primary completion is estimated for Dec 2028, 86 months after the start.
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