Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

Chiesi

Status and phase

Completed

Phase 3

Conditions

Fabry Disease

Treatments

Drug: pegunigalsidase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT03566017

CLI-06657AA1-04

Details and patient eligibility

About

The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.

Full description

This was an open-label study. Participants were enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment was until pegunigalsidase alfa was commercially available to the participant, or at the discretion of the Sponsor. Efficacy and safety analyses were summarized using descriptive statistics for continuous variables and counts and percentages for categorical variables. Data from the parent studies were also included in the analyses.

Enrollment

97 patients

Sex

All

Ages

18 to 60 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03.
The participant signed informed consent.
Female participants and male participants whose co-partners were of child-bearing potential agreed to use a medically acceptable method of contraception. These included combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or sexual abstinence. Contraception had to be used for 2 weeks after treatment termination.

Exclusion criteria

Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.