Fabry DiseaseGoPEGNCT06095713
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
An observational study of Fabry Disease, run by Universität Münster at 8 sites in Germany.
Trial summary
Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 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
- How many people
- 60 participants
- Where
- 8 sitesGermany
- Study dates
- 1 Oct 2023to 30 Sep 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
Study population: Patient with Fabry Disease
Who may be able to join
- Males and females, ≥18 years, diagnosed with Fabry disease.
- ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months).
- Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening.
- Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening.
- Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study.
Who cannot join
- Patient is unwilling to give informed consent.
- Patient is unable to comply with the clinical protocol.
- Patients on dialysis.
- Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial.
- Patients with a history of organ transplantation.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
Germany8 sites
Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin
Fabry disease center Cologne, Universitätsklinikum Köln
Cologne
Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg
Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover
Fabry disease center Mainz, Universitätsmedizin Mainz
Mainz
Fachinternistische Gemeinschaftspraxis, Müllheim
Müllheim
Fabry disease center Münster, Universitätsklinikum Münster
Münster
Fabry disease center Würzburg, Universitätsklinikum Würzburg
Würzburg
About this study
Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.
Read the detailed description
Pegunigalsidase-alfa, a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD), was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy compared with available products.
The rationale of the current project is that disease progression of patients with FD can be stabilized comparable to patients under current ERT, leading to a validation of the clinical phase 3-studies and a transfer of these previous outcomes to a nationwide "real world" designed study in Germany.
- Conditions
- Fabry Disease
- Being tested
- Pegunigalsidase-alfa
- Keywords
- Pegunigalsidase-alfa
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pegunigalsidase-alfaDrug
Standard of care
| Study group | Role | What they receive |
|---|---|---|
| Standard of Care | Patient treated with Pegunigalsidase-alfa according to standard of care Receives: Drug: Pegunigalsidase-alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
eGFR
eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.
Measured: yearly
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2023
- Study start1 Oct 2023
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universität Münster
- Sponsor type
- University, hospital or other
- Collaborators
- Chiesi GmbH
- Responsible party
- Sponsor
- Investigators
- Eva Brand, MD, PhD, Principal investigator, Universitätsklinikum Münster
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06095713
- Sponsor's protocol ID
- 11_0020WWU
- Acronym
- GoPEG
- Status last verified
- Apr 2025
- First submitted
- 10 Oct 2023
- Last updated
- 30 Apr 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
- ClinicalTrials.gov ID
- NCT06095713
- Acronym / sponsor ID
- GoPEG / 11_0020WWU
- Conditions
- Fabry Disease
- Keywords
- Pegunigalsidase-alfa
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Universität MünsterUniversity, hospital or other
- Collaborators
- Chiesi GmbH
- Study start
- 1 Oct 2023 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 23 Oct 2023 (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.
Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.
Detailed description
Pegunigalsidase-alfa, a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD), was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy compared with available products.
The rationale of the current project is that disease progression of patients with FD can be stabilized comparable to patients under current ERT, leading to a validation of the clinical phase 3-studies and a transfer of these previous outcomes to a nationwide "real world" designed study in Germany.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Standard of Care | – | Drug: Pegunigalsidase-alfaPatient treated with Pegunigalsidase-alfa according to standard of care |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patient with Fabry Disease
Key inclusion criteria (5 listed)
- Males and females, ≥18 years, diagnosed with Fabry disease.
- ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months).
- Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening.
- Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening.
- Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study.
Key exclusion criteria (5 listed)
- Patient is unwilling to give informed consent.
- Patient is unable to comply with the clinical protocol.
- Patients on dialysis.
- Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial.
- Patients with a history of organ transplantation.
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-alfaStandard of care | Drug | Standard of Care |
Primary outcome
eGFR
Time frame: yearly
eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2026.
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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.
Eva Brand, MD, PhD principal investigator
Universitätsklinikum Münster
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Universität Münster at 8 sites in Germany.
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 5 inclusion and 5 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: under way, not looking for participants, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Germany. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
eGFR (time frame: yearly). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Apr 2026.