Fabry DiseasePhase 3NCT03614234
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
A Phase 3 study testing pegunigalsidase alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 14 sites in 7 countries.
Trial summary
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- pegunigalsidase alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 14 sitesin 7 countries
- Study dates
- 13 Nov 2018to 13 Apr 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completion of study PB-102-F50.
- The patient signs informed consent.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include 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 (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.
Who cannot join
- 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.
Trial locations and contacts
14 sites in 7 countries.
14 sites in 7 countries
United States7 sites
Emory University School of Medicine
Atlanta, Georgia, 30307
UAB Medicine
Birmingham, Alabama, 35233
Renal Disease Research Institute, LLC
Dallas, Texas, 75235
O & O Alpan
Fairfax, Virginia, 22030
Infusion Associates
Grand Rapids, Michigan, 49525
University of Iowa Hospitals and Clinica
Iowa City, Iowa, 52242
University of Utah Hospitals & Clinics
Salt Lake City, Utah, 84112
United Kingdom2 sites
Addenbrooke's Hospital
Cambridge, CB2 0QQ
The Royal Free Hospital
London
Czechia1 site
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
Prague, 128 08
Italy1 site
Azienda Ospedaliera Universitaria "Federico II"
Naples, Via Pansini, 80131
About this study
Official title: Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
Read the detailed description
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
- Conditions
- Fabry Disease
- Being tested
- pegunigalsidase alfa
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease
- Medical subject headings
- Fabry Disease, Proteinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pegunigalsidase alfaDrug
Also called PRX-102
Recombinant human alpha galactosidase A
| Study group | Role | What they receive |
|---|---|---|
| Experimental open label | Experimental | Pegunigalsidase alfa Receives: Drug: pegunigalsidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of treatment-related adverse events
CTCAE v4.03
Measured: Throughout the study, 364 weeks
Secondary outcomes (8)
Kidney function 1
Estimated glomerular filtration rate (eGFRCKD-EPI)
Measured: Every 6 months throughout the duration of the study, 364 weeks
Cardiac assessment
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Measured: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Biomarkers for Fabry disease
Plasma Lyso-Gb3 and Gb3
Measured: Every 6 months throughout the duration of the study, 364 weeks
Kidney function 2
Protein/Creatinine ratio (UPCR), spot urine test
Measured: Every 6 months throughout the duration of the study, 364 weeks
Clinical assessment
Record of pain medication and pre-medication use
Measured: Every four weeks throughout the duration of the study, 364 weeks
Pain assessment
Short form Brief Pain Inventory (BPI)
Measured: Every 6 months throughout the duration of the study, 364 weeks
Symptom assessment
Mainz Severity Score Index (MSSI)
Measured: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Quality of life assessment
Quality of life (EQ-5D-5L)
Measured: Every 6 months throughout the duration of the study, 364 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2018
- Study start13 Nov 2018
- Primary completion13 Apr 2026
- Study completion13 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label extension study
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03614234
- Sponsor's protocol ID
- CLI-06657AA1-03
- Status last verified
- Jan 2026
- First submitted
- 30 Jul 2018
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Holida M, Linhart A, Longo N, Wallace E, Tondel C, Hughes D, Warnock DG, Pisani A, Eyskens F, Deegan P, Feldt-Rasmussen U, Goker-Alpan O, Mehta A, Piotti G, Fichera V, Wang M, Chertkoff R, Waldek S, Wilcox WR, Bernat JA. Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study. Orphanet J Rare Dis. 2026 Mar 20;21(1):185. doi: 10.1186/s13023-026-04303-8.
PubMed 41862950
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT03614234
- Acronym / sponsor ID
- CLI-06657AA1-03
- Conditions
- Fabry Disease
- Keywords
- Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- 13 Nov 2018 (actual)
- Primary completion
- 13 Apr 2026 (actual)
- Study completion
- 13 Apr 2026 (actual)
- First posted
- 3 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
Detailed description
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label extension study
| Arm | Type | Receives |
|---|---|---|
| Experimental open label | Experimental | Drug: pegunigalsidase alfaPegunigalsidase alfa |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Completion of study PB-102-F50.
- The patient signs informed consent.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include 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 (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.
Key exclusion criteria (1 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| pegunigalsidase alfaAlso: PRX-102Recombinant human alpha galactosidase A | Drug | Experimental open label |
Primary outcome
Evaluation of treatment-related adverse events
Time frame: Throughout the study, 364 weeks
CTCAE v4.03
8 secondary outcomes
Kidney function 1
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Cardiac assessment
Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Biomarkers for Fabry disease
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Kidney function 2
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Clinical assessment
Time frame: Every four weeks throughout the duration of the study, 364 weeks
Pain assessment
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Symptom assessment
Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Quality of life assessment
Time frame: Every 6 months throughout the duration of the study, 364 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Derived from this study: PubMed 41862950Holida M, Linhart A, Longo N, Wallace E, Tondel C, Hughes D, Warnock DG, Pisani A, Eyskens F, Deegan P, Feldt-Rasmussen U, Goker-Alpan O, Mehta A, Piotti G, Fichera V, Wang M, Chertkoff R, Waldek S, Wilcox WR, Bernat JA. Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study. Orphanet J Rare Dis. 2026 Mar 20;21(1):185. doi: 10.1186/s13023-026-04303-8.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing pegunigalsidase alfa in people with Fabry Disease, run by Chiesi Farmaceutici S.p.A. at 14 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, United Kingdom, Belgium, Czechia, Denmark and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of treatment-related adverse events (time frame: Throughout the study, 364 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03614234, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.