Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
Study Identifier:
PB-102-F50
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Study Contact Information:
N/A
Study Complete
Trial Documents
Protocol
Available Languages: English
Statistical Analysis Plan
Available Languages: English
Study Details
Medical Condition
- Fabry Disease
Study Drug
- Biological: Pegunigalsidase alfa
Date
Jul 2017 - Aug 2020
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 60 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Eligible subjects must fulfill the following inclusion criteria:
- Age: 18-60 years
- A documented diagnosis of Fabry disease
- Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to the laboratory reference ranges and one or more of the characteristic features of Fabry disease
- Neuropathic pain
- Cornea verticillata
- Clustered angiokeratoma
- 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
- Neuropathic pain
- Cornea verticillata
- Clustered angiokeratoma
- Treatment with agalsidase alfa or agalsidase beta for at least 3 years and on a stable dose (\>80% labelled dose/kg) for at least last 6 months
- eGFR ≥ 30 mL/min/1.73m\^2 by CKD-EPI equation at screening visit
- Availability of at least 3 historical serum creatinine evaluations since starting agalsidase alfa or agalsidase beta treatment and not more than 2 years old
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence
- Patients whose clinical condition, in the opinion of the Investigator, is suitable for treatment with ERT every 4 weeks.
Exclusion Criteria
- The presence of any of the following excludes a subject from study enrollment:
- History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa or agalsidase beta
- History of renal dialysis or transplantation
- Linear negative slope of eGFR of ≥ 2 mL/min/1.73m\^2/year based on at least 4 serum creatinine values over approximately 2 years (including the value obtained at the screening visit)
- 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 post renal obstructive nephropathy)
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
- Urine protein to creatinine ratio (UPCR) at screening \> 0.5 g/g or mg/mg or 500 mg/g and not treated with an ACE inhibitor or ARB
- Females who are pregnant, planning to become pregnant during the study, or are breast feeding
- Cardiovascular event (myocardial infarction, unstable angina) in the 6-month period before screening
- Cerebrovascular event (stroke, transient ischemic attack) in the 6-month period before screening
- 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.
Healthy Volunteers
No
Protocol Summary
This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase alfa or agalsidase beta for at least 3 years. Safety and efficacy exploratory endpoints will be evaluated throughout the study period and pharmacokinetics will be obtained on Day 1 and Week 52.
Study Locations
Location
Status
Location
UAB Medicine
Birmingham, Alabama, United States, 35294
Status
N/A
Location
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Status
N/A
Location
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Status
N/A
Location
Infusion Associates
Grand Rapids, Michigan, United States, 49525
Status
N/A
Location
Institute of Metabolic Disease
Dallas, Texas, United States, 75226
Status
N/A
Location
University of Utah Hospital & Clinics
Salt Lake City, Utah, United States, 84132
Status
N/A
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