Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

• Register: ClinicalTrials.gov
• Last refreshed on: 18 September 2023
• Main ID: NCT03180840
• Date of registration: 29/05/2017
• Prospective Registration: Yes
• Primary sponsor: Protalix
• Public title: Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks BRIGHT
• Scientific title: Phase 3 Open-Label Switch Over Study to Assess Safety, Efficacy & PK of Pegunigalsidase Alfa (PRX-102) 2mg/kg IV Every 4 Weeks for 52 Weeks in Fabry Disease Patients Currently Treated With Enzyme Replacement Therapy Fabrazyme® or Replagal™
• Date of first enrolment: July 10, 2017
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03180840
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, Czechia, Denmark, Italy, Netherlands, Norway, Spain, Taiwan, Turkey, United Kingdom, United States

Key inclusion & exclusion criteria

Key inclusion criteria:

Eligible subjects must fulfill the following 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 the laboratory reference ranges and one or
more of the characteristic features of Fabry disease

1. Neuropathic pain

2. Cornea verticillata

3. 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

1. Neuropathic pain

2. Cornea verticillata

3. Clustered angiokeratoma

5. 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

6. eGFR = 30 mL/min/1.73m^2 by CKD-EPI equation at screening visit

7. Availability of at least 3 historical serum creatinine evaluations since starting
agalsidase alfa or agalsidase beta treatment and not more than 2 years old

8. 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

9. Patients whose clinical condition, in the opinion of the Investigator, is suitable for
treatment with ERT every 4 weeks.

Key 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 alfa or
agalsidase beta

2. History of renal dialysis or transplantation

3. 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)

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 post renal obstructive
nephropathy)

5. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated or dose changed in the 4 weeks prior to screening

6. 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

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. Cerebrovascular event (stroke, transient ischemic attack) in the 6-month period before
screening

10. 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.



Age minimum: 18 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: Pegunigalsidase alfa

Primary Outcome(s)

Number of Participants With Treatment-related Adverse Events (TEAE) as Assessed by CTCAE v4.03 [Time Frame: Month 12]

Secondary ID(s)

PB-102-F50

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Chiesi Farmaceutici S.p.A.

Results

• Results available: Yes
• Date Posted: 05/01/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03180840