Phase 3
N=97
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT03566017 ↗Enrolled (actual)
97
Serious AEs
46.4%
Results posted
Mar 2026
Primary outcome: Primary: Number of Participants With Treatment-related Adverse Events — 46; 8; 6; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- pegunigalsidase alfa (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Chiesi Farmaceutici S.p.A.
- Primary completion
- Jan 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-related Adverse Events |
46; 8; 6; 5; 4; 3 | — |
| SECONDARY Change From Baseline to the Last Observation for the eGFR |
-10.5 | — |
| SECONDARY Annualized eGFR Slope |
-2.2 | — |
| SECONDARY Shift From Baseline to the Last Scheduled Visit in UPCR Category |
45; 9; 0; 0; 8; 8 | — |
| SECONDARY Change From Baseline to the Last Observation in Plasma Lyso-Gb3 and Globotriaosylceramide (Gb3) Concentrations |
-1.7; -776.9 | — |
| SECONDARY Change From Baseline to the Last Observation for Pain Severity Items of the Brief Pain Inventory (BPI) |
-0.3; 0.2; 0.1; -0.1 | — |
| SECONDARY Change From Baseline to the Last Observation in Overall Score for the Mainz Severity Score Index (MSSI) |
0.6 | — |
| SECONDARY Change in Use of Pain Medication During Treatment |
9; 11; 1; 0; 7; 10 | — |
| SECONDARY Change From Baseline to the Last Observation in the EuroQoL Visual Analog Scale (EQ VAS) Score |
0.9 | — |
| SECONDARY Change From Baseline to the Last Observation in Left Ventricular Mass Index |
7.5; 9.0 | — |
| SECONDARY Change in Infusion Premedication Use From Baseline to 12 Months |
61; 1; 2; 1; 6; 3 | — |
| SECONDARY Change in Infusion Premedication Use From Baseline to 24 Months |
55; 4; 1; 1; 6; 3 | — |
| SECONDARY Change in Infusion Premedication Use From Baseline to 48 Months |
45; 3; 0; 1; 5; 1 | — |
| SECONDARY Change in Infusion Premedication Use From Baseline to 72 Months |
25; 1; 0; 1; 2; 0 | — |
| SECONDARY Number of Participants Who Had ADA at Any Post-baseline Visit |
47 | — |
| SECONDARY Number of Participants With Treatment-emergent ADA Who Had Titer-boosted or Treatment-induced ADA |
7; 24 | — |
| SECONDARY Infusion-related Reactions (IRRs) Occurring During the Infusion or Within 2 Hours After Its Completion (IRR-2H) in More Than One Participant Overall by MedDRA Preferred Term (PT) |
23; 7; 4; 3; 2; 2 | — |
| SECONDARY Infusion-related Reactions Occurring During the Infusion or Within 24H After Its Completion (IRR-24H) in More Than One Participant Overall by MedDRA PT |
29; 8; 4; 4; 3; 3 | — |
Summary
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT03566017). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.