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 16 male and female Fabry disease patients between the ages of 13 and 70 years to be part of the study. The study is conducted in Japan.
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 stage II of main study and in the optional extension study, 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 6 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
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
PRX-102 1 mg/kg every 2 weeks
PRX-102 2 mg/kg every 4 weeks
Fukuoka University Chikushi Hospital
Chikushino-shi, Fukuoka, Japan
WITHDRAWNTohoku University Hospital
Sendai, Miyagi, Japan
RECRUITINGIncidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Incidence of Infusion Related Reactions (IRRs)
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Incidence of Injection site reactions (ISRs)
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change of laboratory tests' results
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change in in body weight in kilograms
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change in height in centimeters
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change in Tanner stage
Tanner Staging of Sexual Development will be used to assess sexual development (i.e. breast development (B1 to B5) and pubic hair development (Ph-1 to Ph-5) in females and pubic hair and genetical development (G1-G5) in males.
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change from baseline of 12-lead ECG quantitative parameters: Mean Heart Rate, PR Interval, QRS Duration, QT Interval, QTc Interval, and ST Segment
Quantitative ECG parameters will be summarized by cohort and overall
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs)
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University of the Ryukyu Hospital
Nishihara, Okinawa, Japan
Osaka University Hospital
Suita, Osaka, Japan
RECRUITINGJuntendo University Hospital, 3-1-3 Hongo, Bunkyo-ku, Tokyo
Bunkyo-ku, Tokyo, Japan
RECRUITINGTokyo Jikei University Hospital
Minato-ku, Tokyo, Japan
RECRUITINGKeio University Hospital
Shinjuku-ku, Tokyo, Japan
RECRUITINGAsahikawa Medical University Hospital
Asahikawa, Japan
RECRUITINGNiigata University Medical & Dental Hospital
Niigata, Japan
RECRUITINGNational Hospital Organization Okayama Medical Center
Okayama, Japan
NOT_YET_RECRUITINGTime frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
ADA status change from baseline
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Incidence of premedication use at each visit and change of infusion premedications from baseline
Time frame: 12 Months, 24 Months and through study completion (an average of 4.5 years)
Change from baseline of Maximum plasma concentration (Cmax), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Time to maximum plasma concentration (tmax), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the plasma concentration-time curve from time 0 to time t (AUC0 t), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve from time 0 to 2 weeks (AUC0-2wk), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve from time 0 to infinity (AUC0-∞), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Terminal half-life (t1/2), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Area under the curve over a dosing interval (AUCτ), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Observed drug concentration at the end of the dosing interval (Cτ), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Clearance (Cl), pharmacokinetic parameter
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)
Change from baseline of Volume of distribution (Vz), pharmacokinetic parameters
Time frame: Assessments will be done over three two-week periods, starting at Baseline (Week 0), V7 (Week 12), and V27 (Week 52)