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Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

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 - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Completion of study PB-102-F20, PB-102-F03, or PB-102-F30
  • The patient signs informed consent
  • Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 1 month after treatment termination.

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
  • Phoenix Children's Hospital
  • University of California San Diego
  • University of California Irvine Center
  • University of Florida
  • Emory University School of Medicine
  • University of Iowa Hospitals and Clinics
  • Infusion Associates
  • Cincinnati Children's Hospital Medical Center
  • University of Pittsburgh
  • Renal Disease Research Institute, LLC - Dallas
  • Eccles Primary Children's Outpatient Services Building
  • O+O Alpan LLC
  • Medical College of Wisconsin
  • Royal Melbourne Hospital
  • Capital District Health Authority
  • Vseobecna fakultni nemocnice v Praze
  • Turku University Central Hospital
  • Hospital Raymond-Poincaré
  • Semmelweis Egyetem
  • Azienda Ospedaliera Universitaria "Federico II"
  • Academisch Medisch Centrum
  • Haukeland University Hospital Klinisk Forskningspost
  • General Hospital Slovenj Gradec
  • Hospital de Dia Quiron Zaragoza
  • University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
  • Salford Royal
  • 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 magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Biomarkers for Fabry disease
plasma Lyso-Gb3 and Gb3
Record of pain medication use
Frequency of pain medication use, or pre-infusion medication
Kidney function 2
Protein/Creatinine ratio, spot urine test (UPCR)
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
May 24, 2018
Last Updated
April 20, 2023
Sponsor
Chiesi Farmaceutici S.p.A.
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1. Study Identification

Unique Protocol Identification Number
NCT03566017
Brief Title
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Official Title
Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 16, 2018 (Actual)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
April 2025 (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-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.
Detailed Description
This is an open-label study. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment is until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.

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
97 (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 trial, 364 weeks
Title
Cardiac assessment
Description
Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)
Time Frame
Every 12 months to end of the study, 7 years
Title
Biomarkers for Fabry disease
Description
plasma Lyso-Gb3 and Gb3
Time Frame
Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
Title
Record of pain medication use
Description
Frequency of pain medication use, or pre-infusion medication
Time Frame
Every two weeks for 7 years
Title
Kidney function 2
Description
Protein/Creatinine ratio, spot urine test (UPCR)
Time Frame
Every 6 months to the end of the study, 7 years
Title
Pain assessment
Description
short form Brief Pain Inventory (BPI)
Time Frame
Every 6 months up to the end of the study, 7 years
Title
Symptom assessment
Description
Mainz Severity Score Index (MSSI)
Time Frame
Every 12 months up to the end of the study, 7 years
Title
Quality of life assessment
Description
quality of life (EQ-5D-5L)
Time Frame
Every 6 months up to the end of the study, 7 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completion of study PB-102-F20, PB-102-F03, or PB-102-F30 The patient signs informed consent Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception. 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
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
University of California San Diego
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
University of California Irvine Center
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
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
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Renal Disease Research Institute, LLC - Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Eccles Primary Children's Outpatient Services Building
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
O+O Alpan LLC
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Capital District Health Authority
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3H 1V8
Country
Canada
Facility Name
Vseobecna fakultni nemocnice v Praze
City
Prague
State/Province
Czech Republic
ZIP/Postal Code
12808
Country
Czechia
Facility Name
Turku University Central Hospital
City
Turku
ZIP/Postal Code
FI-20521
Country
Finland
Facility Name
Hospital Raymond-Poincaré
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
Semmelweis Egyetem
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Azienda Ospedaliera Universitaria "Federico II"
City
Napoli
State/Province
Via Pansini
ZIP/Postal Code
80131
Country
Italy
Facility Name
Academisch Medisch Centrum
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Haukeland University Hospital Klinisk Forskningspost
City
Bergen
ZIP/Postal Code
5021
Country
Norway
Facility Name
General Hospital Slovenj Gradec
City
Slovenj Gradec
ZIP/Postal Code
2380
Country
Slovenia
Facility Name
Hospital de Dia Quiron Zaragoza
City
Zaragoza
ZIP/Postal Code
50012
Country
Spain
Facility Name
University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
City
Edgbaston
State/Province
Birmingham
ZIP/Postal Code
B152TH
Country
United Kingdom
Facility Name
Salford Royal
City
Salford
State/Province
Greater Manchester
ZIP/Postal Code
M6 8HD
Country
United Kingdom
Facility Name
Addenbrooke's Hospital
City
Cambridge
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
The Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom

12. IPD Sharing Statement

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Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

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