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

Primary Purpose

Fabry Disease

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
pegunigalsidase alfa
Sponsored by
Chiesi Farmaceutici S.p.A.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Completion of study PB-102-F50.
  2. The patient signs informed consent.
  3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence.

Exclusion Criteria:

Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.

Sites / Locations

  • UAB Medicine
  • Emory University School of Medicine
  • University of Iowa Hospitals and Clinica
  • Infusion Associates
  • Renal Disease Research Institute, LLC
  • University of Utah Hospitals & Clinics
  • O & O Alpan
  • UZ Antwerpen
  • Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
  • Medical Endocrinology PE 2132, Rigshospitalet
  • Azienda Ospedaliera Universitaria "Federico II"
  • Helse Bergen HF Haukeland Universitetssykehus
  • Addenbrooke's Hospital
  • The Royal Free Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental open label

Arm Description

Pegunigalsidase alfa

Outcomes

Primary Outcome Measures

Evaluation of treatment-related adverse events
CTCAE v4.03

Secondary Outcome Measures

Kidney function 1
Estimated glomerular filtration rate (eGFRCKD-EPI)
Cardiac assessment
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Biomarkers for Fabry disease
Plasma Lyso-Gb3 and Gb3
Kidney function 2
Protein/Creatinine ratio (UPCR), spot urine test
Clinical assessment
Record of pain medication and pre-medication use
Pain assessment
Short form Brief Pain Inventory (BPI)
Symptom assessment
Mainz Severity Score Index (MSSI)
Quality of life assessment
Quality of life (EQ-5D-5L)

Full Information

First Posted
July 30, 2018
Last Updated
April 20, 2023
Sponsor
Chiesi Farmaceutici S.p.A.
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1. Study Identification

Unique Protocol Identification Number
NCT03614234
Brief Title
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 13, 2018 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chiesi Farmaceutici S.p.A.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Model Description
Open-label extension study
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Experimental open label
Arm Type
Experimental
Arm Description
Pegunigalsidase alfa
Intervention Type
Drug
Intervention Name(s)
pegunigalsidase alfa
Other Intervention Name(s)
PRX-102
Intervention Description
Recombinant human alpha galactosidase A
Primary Outcome Measure Information:
Title
Evaluation of treatment-related adverse events
Description
CTCAE v4.03
Time Frame
Throughout the study, 364 weeks
Secondary Outcome Measure Information:
Title
Kidney function 1
Description
Estimated glomerular filtration rate (eGFRCKD-EPI)
Time Frame
Every 6 months throughout the duration of the study, 364 weeks
Title
Cardiac assessment
Description
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Time Frame
Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Title
Biomarkers for Fabry disease
Description
Plasma Lyso-Gb3 and Gb3
Time Frame
Every 6 months throughout the duration of the study, 364 weeks
Title
Kidney function 2
Description
Protein/Creatinine ratio (UPCR), spot urine test
Time Frame
Every 6 months throughout the duration of the study, 364 weeks
Title
Clinical assessment
Description
Record of pain medication and pre-medication use
Time Frame
Every four weeks throughout the duration of the study, 364 weeks
Title
Pain assessment
Description
Short form Brief Pain Inventory (BPI)
Time Frame
Every 6 months throughout the duration of the study, 364 weeks
Title
Symptom assessment
Description
Mainz Severity Score Index (MSSI)
Time Frame
Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Title
Quality of life assessment
Description
Quality of life (EQ-5D-5L)
Time Frame
Every 6 months throughout the duration of the study, 364 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Facility Information:
Facility Name
UAB Medicine
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30307
Country
United States
Facility Name
University of Iowa Hospitals and Clinica
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Infusion Associates
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49525
Country
United States
Facility Name
Renal Disease Research Institute, LLC
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
University of Utah Hospitals & Clinics
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
O & O Alpan
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
UZ Antwerpen
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
City
Praha
ZIP/Postal Code
128 08
Country
Czechia
Facility Name
Medical Endocrinology PE 2132, Rigshospitalet
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Azienda Ospedaliera Universitaria "Federico II"
City
Napoli
State/Province
Via Pansini
ZIP/Postal Code
80131
Country
Italy
Facility Name
Helse Bergen HF Haukeland Universitetssykehus
City
Bergen
ZIP/Postal Code
5021
Country
Norway
Facility Name
Addenbrooke's Hospital
City
Cambridge
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
The Royal Free Hospital
City
London
Country
United Kingdom

12. IPD Sharing Statement

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

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