Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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18 September 2023 |
Main ID: |
NCT01678898 |
Date of registration:
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31/08/2012 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
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Scientific title:
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A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Months to Adult Fabry Patients |
Date of first enrolment:
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October 2012 |
Target sample size:
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18 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/ct2/show/NCT01678898 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Australia
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Paraguay
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Serbia
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Spain
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United Kingdom
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Symptomatic adult Fabry patients (=18 yrs)
- Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less
than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32
nmol/hr/mg/protein)
- Females: historical genetic test results consistent with Fabry mutations
- Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit
- Patients who have never received enzyme replacement therapy (ERT) in the past, or
patients who have not received ERT in the past 6 months and have a negative anti alpha
galactosidase antibody test
- eGFR = 60 mL/min/1.73m2
- The patient signs informed consent
- 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:
- Participation in any trial of an investigational drug within 30 days prior to study
screening
- Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7)
- History of dialysis or renal transplantation
- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated or dose changed in the 4 weeks prior to screening
- Severe myocardial fibrosis by MRI (=2 late-enhancement [LE] positive left ventricular
segments) (Weidemann et al. 2009)
- History of clinical stroke
- Pregnant or nursing
- Presence of HIV and/or HBsAg and/or Hepatitis C infections
- Known allergies to ERT
- Known allergy to Gadolinium based contrast agents
- 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:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Drug: PRX-102
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Primary Outcome(s)
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Adverse Events
[Time Frame: 12 months]
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Secondary ID(s)
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PB-102-F01 & PB-102-F02
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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