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Extension Study of PRX-102 for up to 60 Months

Primary Purpose

Fabry Disease

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
PRX-102 (pegunigalsidase alfa)
Sponsored by
Protalix
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring PRX-102, pegunigalsidase alfa, Fabry Disease

Eligibility Criteria

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

Inclusion Criteria:

  • Completion of study PB-102-F02
  • The patient signs informed consent
  • Female patients 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 throughout the duration of the study and for 3 months after termination of treatment.

Exclusion Criteria:

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

Sites / Locations

  • University of Florida
  • University of Iowa Hospitals and Clinics
  • Johns Hopkins University School of Medicine
  • Institute of Metabolic Disease
  • O & O Alpan
  • Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
  • Hospital de Dia Quiron Zaragoza
  • The Royal Free Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PRX-102 (pegunigalsidase alfa)

Arm Description

PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)

Outcomes

Primary Outcome Measures

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.

Secondary Outcome Measures

Full Information

First Posted
November 5, 2013
Last Updated
September 10, 2023
Sponsor
Protalix
Collaborators
Chiesi Farmaceutici S.p.A.
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1. Study Identification

Unique Protocol Identification Number
NCT01981720
Brief Title
Extension Study of PRX-102 for up to 60 Months
Official Title
A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for up to 60 Months to Adult Fabry Patients
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
January 16, 2014 (Actual)
Primary Completion Date
August 26, 2020 (Actual)
Study Completion Date
November 9, 2021 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

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

5. Study Description

Brief Summary
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
Detailed Description
An open-label study to evaluate the ongoing safety, tolerability and efficacy parameters of PRX-102 in adult Fabry patients (≥18 years of age). Patients enrolled received 1.0 mg/kg of PRX-102 as an intravenous infusion every 2 weeks (+/- 3 days) for up to 60 months and no less than 36 months.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PRX-102 (pegunigalsidase alfa)
Arm Type
Experimental
Arm Description
PRX-102 (pegunigalsidase alfa) 1.0 mg/kg IV every 2 weeks (+/- 3 days)
Intervention Type
Biological
Intervention Name(s)
PRX-102 (pegunigalsidase alfa)
Other Intervention Name(s)
pegunigalsidase alfa, Recombinant human alpha galactosidase-A
Intervention Description
PRX-102 1 mg/kg every 2 weeks
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.03
Description
Results represent the number of treatment-emergent adverse events (TEAE) that were considered definitely, probably or possibly related to study treatment.
Time Frame
Every two weeks up to 60 months
Other Pre-specified Outcome Measures:
Title
Plasma Lyso-Gb3 Concentration
Description
Globotriaosylsphingosine (Lyso-Gb3) is Fabry disease specific biomarker, which was measured at Baseline, every 3 months up to 24 months and then every 6 months up to the end of the study. Baseline and Month 60 and change from Baseline reported.
Time Frame
Baseline and month 60
Title
Estimated Glomerular Filtration Rate (eGFR)
Description
eGFR was calculated based on the serum creatinine values according to the CKD-EPI equation. The absolute change in eGFR from baseline measurement at visit 1 to Month 60 was summarized using descriptive statistics. Baseline and month 60 reported.
Time Frame
Baseline and Month 60

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Completion of study PB-102-F02 The patient signs informed consent Female patients and male patients whose co-partners were 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 throughout the duration of the study and for 3 months after termination of treatment. 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 the patient's compliance with the requirements of the study
Facility Information:
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32611
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
Johns Hopkins University School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Institute of Metabolic Disease
City
Dallas
State/Province
Texas
ZIP/Postal Code
75226
Country
United States
Facility Name
O & O Alpan
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)
City
Asunción
Country
Paraguay
Facility Name
Hospital de Dia Quiron Zaragoza
City
Zaragoza
ZIP/Postal Code
50012
Country
Spain
Facility Name
The Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Extension Study of PRX-102 for up to 60 Months

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