Chiesi Farmaceutici S.p.A.
The aim of this study is to evaluate the safety and efficacy of pegunigalsidase alfa in Japanese patients (adults and adolescents) affected by Fabry disease. It is planned of a total of approximately 18-20 male and female Fabry disease patients between the ages of 13 and 60 years to be part of the study. The study is conducted in Japan.
Fabry Disease
PRX-102 1 mg/kg every 2 weeks
PRX-102 2 mg/kg every 4 weeks
Phase 2
Phase 3
Investigators are doing this study to find out if treatment with pegunigalsidase alfa will prevent or reduce the development of health problems caused by Fabry disease and thereby improve patients' health and quality of life. pegunigalsidase alfa (PRX-102) is a drug made using genetic engineering techniques and manufactured using cultured tobacco cells. It is given by intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight. The study consists of a main study that is divided into two stages, each of which will last one year, followed by an optional extension study. In the optional extension stage, the participants may receive PRX-102 intravenous infusion every 2 weeks, at a dosage of 1 milligram per kilogram (mg/kg) of body weight or every 4 weeks at a dosage of 2 milligrams per kilogram (mg/kg) of body weight. There are three groups (cohorts) in this study, with adults enrolled in either Cohort A or B and adolescents in Cohort C. Whether an adult is assigned to Cohort A or Cohort B depends on their kidney function and treatment history. This study will start with a screening visit of up to 4 weeks. It will be followed up by infusion visits every 2 weeks or 4 weeks. For subjects not continuing in the extension stage, a follow-up call is to be made 30 days after the last study drug infusion.}}
Study Type : | Interventional |
Estimated Enrollment : | 18 participants |
Masking : | None (Open Label) |
Primary Purpose : | Treatment |
Official Title : | A Multicenter Open-Label Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Pegunigalsidase Alfa (PRX-102) in Japanese Patients With Fabry Disease (RISE) |
Actual Study Start Date : | August 1, 2023 |
Estimated Primary Completion Date : | March 2026 |
Estimated Study Completion Date : | March 2028 |
Arm | Intervention/treatment |
---|---|
Experimental: PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks PRX-102 1 mg/kg every 2 weeks or PRX-102 2 mg/kg every 4 weeks (available only in the optional extension part) |
Drug: PRX-102 1 mg/kg every 2 weeks Drug: PRX-102 2 mg/kg every 4 weeks |
Ages Eligible for Study: | 18 Years to 60 Years |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
Recruiting
Fukuoka University Chikushi Hospital
Chikushino, Fukuoka, Japan, 818-8502
Recruiting
Tohoku University Hospital
Sendai, Miyagi, Japan, 980-8574
Recruiting
University of the Ryukyu Hospital
Nishihara, Okinawa, Japan, 903-0125
Recruiting
Osaka University Hospital
Suite, Osaka, Japan, 565-0871
Recruiting
Juntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
Bunkyo Ward, Tokyo, Japan, 113-0033
Recruiting
Tokyo Jikei University Hospital
Everyone talk, Tokyo, Japan, 105-8461
Recruiting
Keio University Hospital
Shinjuku-ku, Tokyo, Japan, 160-8582
Recruiting
Niigata University Medical & Dental Hospital
Niigata, Japan, 951-8520