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Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03018730
Recruitment Status : Active, not recruiting
First Posted : January 12, 2017
Last Update Posted : March 20, 2019
Sponsor:
Information provided by (Responsible Party):
Protalix

Tracking Information
First Submitted Date  ICMJE January 9, 2017
First Posted Date  ICMJE January 12, 2017
Last Update Posted Date March 20, 2019
Actual Study Start Date  ICMJE February 23, 2017
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 12, 2018)
Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Throughout the 12 months study ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 10, 2017)
Treatment-emergent anti-PRX-102 antibodies [ Time Frame: 12 months ]
Change History Complete list of historical versions of study NCT03018730 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 12, 2018)
  • Mean annualised change in eGFR [ Time Frame: Every 4 weeks for 12 months ]
    Mean annualised change in eGFR
  • Left Ventricular Mass Index (g/m2) preferably by MRI [ Time Frame: Every 6 months for 12 months ]
    Left Ventricular Mass Index (g/m2) preferably by MRI (ECG can be used as an alternative)
  • Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Plasma Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Urine Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Protein/Creatinine ratio [ Time Frame: Every 3 months for 12 months ]
    Protein/Creatinine ratio spot urine test
  • Frequency of pain medication use [ Time Frame: Every 2 weeks for 12 months ]
  • Exercise tolerance (Stress Test) [ Time Frame: Every 6 months for 12 months ]
  • Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 3 months for 12 months ]
  • Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 12 months ]
  • Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 12 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 10, 2017)
  • Mean annualised change in eGFR [ Time Frame: Every 2 weeks for 12 months ]
    Mean annualised change in eGFR
  • Left Ventricular Mass Index (g/m2) preferably by MRI [ Time Frame: Every 6 months for 12 months ]
    Left Ventricular Mass Index (g/m2) preferably by MRI (ECG can be used as an alternative)
  • Plasma Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Plasma Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Urine Lyso-Gb3 [ Time Frame: Every 3 months for 12 months ]
  • Protein/Creatinine ratio [ Time Frame: Every 3 months for 12 months ]
    Protein/Creatinine ratio spot urine test
  • Frequency of pain medication use [ Time Frame: Every 2 weeks for 12 months ]
  • Exercise tolerance (Stress Test) [ Time Frame: Every 6 months for 12 months ]
  • Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 3 months for 12 months ]
  • Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 12 months ]
  • Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 12 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Official Title  ICMJE An Open Label Study of the Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Brief Summary This is an open label switch over study to assess the safety and efficacy of PRX-102 (pegunigalsidase alfa). Patients treated with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months. Patients will be screened and evaluated over 3 months while continuing on agalsidase alfa. Following the screening period, the patient will be enrolled and switched from their agalsidase alfa treatment to receive intravenous (IV) infusions of PRX-102 1 mg/kg every two weeks for 12 months. No more than 25% of treated patients will be female.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fabry Disease
Intervention  ICMJE Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
  • pegunigalsidase alfa
  • Recombinant human alpha galactosidase-A
Study Arms  ICMJE Experimental: PRX-102
PRX-102 infusion every 2 weeks
Intervention: Biological: PRX-102 (pegunigalsidase alfa)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 10, 2017)
22
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2019
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Age: 18-60 years
  2. A documented diagnosis of Fabry disease
  3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to laboratory range and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma
  4. Females: historical genetic test results consistent with Fabry mutations, or in the case of novel mutations a first degree male relative with Fabry disease, and one or more of the characteristic features of Fabry disease i. Neuropathic pain ii. Cornea verticillata iii. Clustered angiokeratoma
  5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
  6. eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation
  7. Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
  8. 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

Exclusion Criteria:

  1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
  2. History of renal dialysis or transplantation
  3. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
  4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
  5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
  6. Known history of hypersensitivity to Gadolinium contrast agent
  7. Females who are pregnant, planning to become pregnant during the study, or are breast feeding
  8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
  9. Congestive heart failure NYHA Class IV
  10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
  11. 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   Czechia,   Germany,   Netherlands,   Norway,   Slovenia,   Spain,   United Kingdom
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT03018730
Other Study ID Numbers  ICMJE PB-102-F30
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Protalix
Study Sponsor  ICMJE Protalix
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Raul Chertkoff, MD Protalix Ltd
PRS Account Protalix
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP