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New enzyme therapy shows promise for Long-Term fabry disease management

NCT ID NCT03566017

Disease control Sponsor: Chiesi Farmaceutici S.p.A. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked at the long-term safety of pegunigalsidase alfa, an enzyme replacement therapy, in 97 adults with Fabry disease. Participants received the drug intravenously every two weeks for up to several years. The main goal was to track side effects and see how well the drug was tolerated over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pegunigalsidase alfa (a lab-made enzyme replacement therapy)
What this could lead to
If successful, this could confirm that pegunigalsidase alfa is a safe long-term treatment option for managing Fabry disease and slowing kidney damage.
What could go wrong
This is an open-label extension study with no placebo group, so results are less definitive. Side effects are possible, and the drug may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

97 people

The number who actually took part.

Started

Sep 2018

Finished

Jan 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 60 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03. 2. The participant signed informed consent. 3. 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.

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Conditions

The condition(s) this trial relates to.

Fabry disease proteinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Academisch Medisch Centrum

    Amsterdam, 1105 AZ, Netherlands

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ, United Kingdom

  • Azienda Ospedaliera Universitaria "Federico II"

    Naples, Via Pansini, 80131, Italy

  • Capital District Health Authority

    Halifax, Nova Scotia, B3H 1V8, Canada

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • General Hospital Slovenj Gradec

    Slovenj Gradec, 2380, Slovenia

  • Haukeland University Hospital Klinisk Forskningspost

    Bergen, 5021, Norway

  • Hospital Raymond-Poincaré

    Garches, 92380, France

  • Hospital de Dia Quiron Zaragoza

    Zaragoza, 50012, Spain

  • Infusion Associates

    Grand Rapids, Michigan, 49525, United States

  • Institute of Metabolic Disease

    Dallas, Texas, 75246, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • O+O Alpan LLC

    Fairfax, Virginia, 22030, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Renal Disease Research Institute, LLC - Dallas

    Dallas, Texas, 75235, United States

  • Royal Melbourne Hospital

    Parkville, Victoria, 3050, Australia

  • Salford Royal

    Salford, Greater Manchester, M6 8HD, United Kingdom

  • Semmelweis Egyetem

    Budapest, 1083, Hungary

  • The Royal Free Hospital

    London, NW3 2QG, United Kingdom

  • Turku University Central Hospital

    Turku, FI-20521, Finland

  • UAB Medicine

    Birmingham, Alabama, 35233, United States

  • University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

    Edgbaston, Birmingham, B152TH, United Kingdom

  • University of California Irvine Center

    Orange, California, 92868, United States

  • University of California San Diego

    La Jolla, California, 92037, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Utah Hospitals & Clinics

    Salt Lake City, Utah, 84108, United States

  • Vseobecna fakultni nemocnice v Praze

    Prague, Czech Republic, 12808, Czechia

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