Trial record 2 of 7 for:
PRX-102 | Phase 3
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function (BALANCE)
|
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: NCT02795676 |
|
Recruitment Status :
Completed
First Posted : June 10, 2016
Last Update Posted : October 13, 2022
|
Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with 1mg/kg agalsidase beta or to treatment with 1 mg/kg of PRX-102. The identity of the enzyme will be blinded to the patient and the investigator. Patients will receive intravenous infusions every two weeks. Patients will be randomized in a 2:1 ratio of PRX-102 to agalsidase beta. Randomization will be stratified by urinary protein to creatinine ratio (UPCR) of < or ≥ 1 g/g by spot urine sample. No more than 50% of the patients will be female.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fabry Disease | Biological: PRX-102 (pegunigalsidase alfa) Biological: agalsidase beta | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 78 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Randomized, Double Blind, Active Control Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function in Patients With Fabry Disease Previously Treated With Agalsidase Beta |
| Actual Study Start Date : | June 2016 |
| Actual Primary Completion Date : | October 2021 |
| Actual Study Completion Date : | July 2022 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Fabry disease
MedlinePlus related topics:
Kidney Tests
| Arm | Intervention/treatment |
|---|---|
|
Experimental: PRX-102 (pegunigalsidase alfa)
PRX-102 infusion every 2 weeks
|
Biological: PRX-102 (pegunigalsidase alfa)
PRX-102 1 mg/kg every 2 weeks
Other Names:
|
|
Active Comparator: agalsidase beta
agalsidase beta infusion every 2 weeks
|
Biological: agalsidase beta
agalsidase beta 1 mg/kg every 2 weeks
Other Name: Fabrazyme |
Primary Outcome Measures :
- eGFR Slope [ Time Frame: Every month for 2 years ]eGFR Slope
Secondary Outcome Measures :
- Left Ventricular Mass Index (g/m2) by MRI [ Time Frame: Every 12 months for 2 years ]
- Plasma Lyso-Gb3 [ Time Frame: 1.5 month, every 3 months for 1 year and then every 6 months up to 24 months ]
- Plasma Gb3 [ Time Frame: 1.5 month, every 3 months for 1 year and then every 6 months up to 24 months ]
- Urine Lyso-GB3 [ Time Frame: 1.5 month, every 3 months for 1 year and then every 6 months up to 24 months ]
- Protein/creatinine ratio [ Time Frame: Every 3 months for 2 years ]
- Frequency of pain medication use [ Time Frame: Every 2 weeks for 2 years ]
- Exercise tolerance (Stress Test) [ Time Frame: Every year for 2 years ]
- Short Form Brief Pain Inventory (BPI) [ Time Frame: Every 6 months for 2 years ]
- Mainz Severity Score Index (MSSI) [ Time Frame: Every 6 months for 2 years ]
- Quality of life EQ-5D-5L [ Time Frame: Every 6 months for 2 years ]
Other Outcome Measures:
- PRX-102 pharmacokinetics [ Time Frame: Day 1, 6 months, 12 months and 24 months ]PRX-102 pharmacokinetics parameters
- Anti-drug IgG antibodies [ Time Frame: Every 2 weeks for 1 month, then every month for the first 6 months and every 3 month up to 24 months ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 60 Years (Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
-
Symptomatic adult Fabry disease patients, age 18-60 years
-
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
-
Females:
a. historical genetic test results consistent with Fabry pathogenic mutation and 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
-
- Screening eGFR by CKD-EPI equation 40 to 120 mL/min/1.73 m²
- Linear negative slope of eGFR 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) of ≥ 2 mL/min/1.73 m²/year
- 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.
- 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.
Exclusion Criteria:
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase beta
- Known non-pathogenic Fabry mutations
- History of renal dialysis or transplantation
- 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)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Patient with a screening eGFR value between 91-120 mL/min/1.73 m², having an historical eGFR value higher than 120 mL/min/1.73 m² (during 9 to 18 months before screening)
- Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- Known history of hypersensitivity to Gadolinium contrast agent that is not managed by the use of pre-medication
- Female subjects who are pregnant, planning to become pregnant during the study, or are breastfeeding
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02795676
Locations
Show 29 study locations
Sponsors and Collaborators
Protalix
Investigators
| Study Director: | Raul Chertkoff, MD | Protalix Ltd. |
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT02795676 |
| Other Study ID Numbers: |
PB-102-F20 |
| First Posted: | June 10, 2016 Key Record Dates |
| Last Update Posted: | October 13, 2022 |
| Last Verified: | October 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
Keywords provided by Protalix:
|
Glomerular filtration rate Proteinuria PRX-102 pegunigalsidase alfa Fabry Disease |
Additional relevant MeSH terms:
|
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |