NCT01981720

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2014

Longer than P75 for phase_1

Geographic Reach
4 countries

8 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 5, 2013

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 11, 2013

Completed
2 months until next milestone

Study Start

First participant enrolled

January 16, 2014

Completed
6.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 26, 2020

Completed
1.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

November 9, 2021

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

February 8, 2023

Completed
Last Updated

September 13, 2023

Status Verified

September 1, 2023

Enrollment Period

6.6 years

First QC Date

November 5, 2013

Results QC Date

January 15, 2023

Last Update Submit

September 10, 2023

Conditions

Fabry Disease

Keywords

PRX-102pegunigalsidase alfaFabry Disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    Every two weeks up to 60 months

Other Outcomes (2)

  • Plasma Lyso-Gb3 Concentration

    Baseline and month 60

  • Estimated Glomerular Filtration Rate (eGFR)

    Baseline and Month 60

Study Arms (1)

PRX-102 (pegunigalsidase alfa)

EXPERIMENTAL

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

Biological: PRX-102 (pegunigalsidase alfa)

Interventions

PRX-102 (pegunigalsidase alfa)BIOLOGICAL

PRX-102 1 mg/kg every 2 weeks

Also known as: pegunigalsidase alfa, Recombinant human alpha galactosidase-A
PRX-102 (pegunigalsidase alfa)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

University of Florida

Gainesville, Florida, 32611, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

Institute of Metabolic Disease

Dallas, Texas, 75226, United States

Location

O & O Alpan

Fairfax, Virginia, 22030, United States

Location

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)

Asunción, Paraguay

Location

Hospital de Dia Quiron Zaragoza

Zaragoza, 50012, Spain

Location

The Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCerebral Small Vessel DiseasesCerebrovascular DisordersVascular DiseasesCardiovascular DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Limitations and Caveats

Small sample size.

Results Point of Contact

Title
Sari Alon MSc
Organization
Protalix Ltd.
sari@protalix.com
+972-4-9028100

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 5, 2013

First Posted

November 11, 2013

Study Start

January 16, 2014

Primary Completion

August 26, 2020

Study Completion

November 9, 2021

Last Updated

September 13, 2023

Results First Posted

February 8, 2023

Record last verified: 2023-09

Data Sharing

IPD Sharing
Will not share

Locations

ES(1)PA(1)US(5)GB(1)