Safety and effectiveness study comparing PRX-102 and Agalsidase Beta on Kidney function for patients with Fabry Disease who have previously been treated with Agalsidase Beta

Phase 1

• Conditions: Fabry disease (a-galactosidase A deficiency); MedDRA version: 20.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2016-000378-38-HU
• Lead Sponsor: Protalix Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-07-11
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 78

Inclusion Criteria

Eligible subjects must fulfill the following inclusion criteria:
1. Symptomatic adult Fabry disease patients, age 18-60 years
2.Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than 30% mean normal levels
and one or more of the characteristic features of Fabry disease i. neuropathic pain ii. cornea verticillata iii. clustered angiokeratoma 3.Females: a.historical genetic test results consistent with Fabry pathogenic mutation One or more of the described characteristic features of Fabry disease:
i. neuropathic pain,
ii. cornea verticillata,
iii. clustered angiokeratoma b. or in the case of novel mutations a first degree male family member with Fabry disease with the same mutation, and one or more of the characteristic features of Fabry disease i. neuropathic pain, ii.cornea verticillata, iii.clustered angiokeratoma
4. Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m2
5. Linear negative slope of eGFR of = 2 mL/min/1.73 m2 based on at least 3 serum creatinine values over approximately 1 year (range of 9 to 18 months, including the value obtained at the screening visit)
6. Treatment with a dose of 1 mg/kg agalsidase beta per infusion every 2 weeks for at least one year and at least 80% of 13 (10.4) mg/kg total dose over the last 6 months
7. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted method of contraception, not including the rhythm method.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 78
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The presence of any of the following excludes a subject from study enrollment:
1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
2.Known non-pathogenic Fabry mutations 3. History of renal dialysis or transplantation
4. 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)
5. Patient with a screening eGFR value between 91-120mL/min/1.73m2, having an historical eGFR value higher than 120 mL/min/1.73m2(during 9 to18 months before screening) 6. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
7. Urine protein to creatinine ratio (UPCR) > 0.5 g/g (0.5 mg/mg or 500 mg/g)and not treated with an ACE inhibitor or ARB
8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
9. Congestive heart failure NYHA Class IV
10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
11. Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of premedication
12. Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
13. 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified