Actively Recruiting

Phase 2
Age: 18Years - 80Years
All Genders
ID05190991

Safety and Efficacy of RPH-104 to Prevent Recurring Attacks in Adults With Colchicine Resistant or Intolerant Familial Mediterranean Fever

Led by R-Pharm International, LLC · Updated on 2024-12-16

60

Participants Needed

8

Research Sites

4 weeks

Total Duration

On this page

Sponsors

R

R-Pharm International, LLC

Lead Sponsor

A

Atlant Clinical LLC

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the long-term safety and effectiveness of RPH-104 in adults with familial Mediterranean fever (FMF) who do not respond well to or cannot tolerate colchicine treatment. This study focuses on patients who have already participated in an earlier core trial and received at least one dose of RPH-104. The goal is to understand how well RPH-104 works to prevent recurrent fever attacks in this specific patient group over an extended period. The study includes a screening phase lasting 2 weeks to confirm eligibility and unblind previous treatment assignments. Following this, participants receive open-label RPH-104 injections every two weeks for up to 198 weeks. The doses studied are either 80 mg or 160 mg administered subcutaneously. Patients may receive their injections at the study site or at home if trained to self-administer. Safety and effectiveness are regularly assessed through scheduled visits and phone contacts. If patients experience attacks on the lower dose, an increase to 160 mg may occur. After completing treatment, an 8-week safety follow-up with two visits monitors participants. Participants will visit the study site frequently for drug administration and evaluations, including assessments of side effects and disease activity. Researchers collect data on adverse events, inflammatory markers, symptom severity, and kidney function. Telephone follow-ups also support patient monitoring. The primary outcomes focus on tracking side effects, while secondary outcomes assess disease control and symptom changes. Overall, the study lasts nearly four years, combining treatment and follow-up to provide detailed long-term information on RPH-104 use in colchicine-resistant or intolerant FMF patients.

CONDITIONS

Brief Title

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever

Who Can Participate

Age: 18Years - 80Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 18 to 80 years with familial Mediterranean fever resistant to or intolerant of colchicine
  • Completed the core study and received at least one dose of RPH-104
  • Voluntarily signed informed consent for this study
  • Able and willing to follow study visit schedules and procedures
  • Willing to receive RPH-104 injections every two weeks at the study site or learn to self-administer at home
  • Agree to qualified medical personnel visits for RPH-104 administration if applicable
Not Eligible

You will not qualify if you...

  • Medically significant events or conditions during the core study or other medical problems that increase risk or affect study results
  • Pregnant, breastfeeding, or planning pregnancy during the study or within 2 months after last RPH-104 dose
  • Women of childbearing potential not agreeing to use highly effective contraception during and for 8 weeks after the study
  • Men sexually active not agreeing to use highly effective contraception during and for 8 weeks after the study
  • Need for systemic glucocorticoids over specified doses or certain immunosuppressants and biological products excluding RPH-104
  • Use of live vaccines during the study or within 3 months after last RPH-104 dose
  • Positive tuberculosis screening at core study visit 10
  • Participation in other experimental studies except the core study prior to this trial

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 weeks

Participants are screened for eligibility to participate in the trial.

1 screening and enrollment visit (in-person)

Treatment

Duration - 198 weeks

Participants receive RPH-104 injections every two weeks to prevent recurrent fever attacks, with safety and efficacy assessments conducted regularly.

Visits every 2 weeks initially and then every 12 weeks with additional telephone contacts every 4 weeks starting from Visit 4; injections administered either at the study site or by the participant at home after training

Safety Follow-up

Duration - 8 weeks

Participants visit the study site for safety assessments after completing the treatment period.

2 visits (in-person) at 4 and 8 weeks after last dose

Trial Site Locations

Total: 8 locations

1

Center of Medical Genetics and Primary Health Care LLC

Yerevan, Armenia, 0001

Actively Recruiting

2

FSBEI HE First Moscow State Medical University named after I.M. Sechenov

Moscow, Russia, 119048

Actively Recruiting

3

Moscow Multidisciplinary Scientific and Clinical Center named after S.P. Botkin

Moscow, Russia, 125284

Actively Recruiting

4

Medical Technologies Ltd.

Saint Petersburg, Russia, 191025

Actively Recruiting

5

Terafarm, Llc

Stavropol, Russia, 355000

Actively Recruiting

6

Hacettepe University Faculty of Medicine

Ankara, Turkey (Türkiye), 06230

Actively Recruiting

7

Istanbul University Istanbul Faculty of Medicine

Istanbul, Turkey (Türkiye), 34093

Actively Recruiting

8

Istanbul University Cerrahpasa Faculty of Medicine

Istanbul, Turkey (Türkiye), 34098

Actively Recruiting

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Research Team

Y

Yulia Karpova

M

Marina Beloukhova

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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