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NCT Number: NCT03614234

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

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.

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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pathological Conditions, Signs and Symptoms Proteinuria Signs and Symptoms Sphingolipidoses Urination Disorders Urogenital Diseases Urologic Diseases Urological Manifestations Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

UZ Antwerpen, Edegem, Belgium

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

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.

Treatment and study plan

pegunigalsidase alfa

Drug

Recombinant human alpha galactosidase A

Other names: PRX-102

Primary outcomes

  1. Evaluation of treatment-related adverse events

    Time frame: Throughout the study, 364 weeks

    CTCAE v4.03

Secondary outcomes

  1. Kidney function 1

    Time frame: Every 6 months throughout the duration of the study, 364 weeks

    Estimated glomerular filtration rate (eGFRCKD-EPI)

  2. Cardiac assessment

    Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364

    Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test

  3. Biomarkers for Fabry disease

    Time frame: Every 6 months throughout the duration of the study, 364 weeks

    Plasma Lyso-Gb3 and Gb3

  4. Kidney function 2

    Time frame: Every 6 months throughout the duration of the study, 364 weeks

    Protein/Creatinine ratio (UPCR), spot urine test

  5. Clinical assessment

    Time frame: Every four weeks throughout the duration of the study, 364 weeks

    Record of pain medication and pre-medication use

  6. Pain assessment

    Time frame: Every 6 months throughout the duration of the study, 364 weeks

    Short form Brief Pain Inventory (BPI)

  7. Symptom assessment

    Time frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364

    Mainz Severity Score Index (MSSI)

  8. Quality of life assessment

    Time frame: Every 6 months throughout the duration of the study, 364 weeks

    Quality of life (EQ-5D-5L)

Sponsors and collaborators

Lead sponsor

Chiesi Farmaceutici S.p.A.

Industry

Registry information

Official study 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

Important dates

Study start
2018
Primary completion
2026
Study completion
2026
First posted
Aug 3, 2018
Registry last updated
May 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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