Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients



Status:Completed
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:4/21/2016
Start Date:October 2012
End Date:January 2016

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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 Weeks to Adult Fabry Patients

This is the first human treatment with PRX-102, an enzyme being developed as a long-term
enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha
galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be
evaluated in this study of increasing doses. Patients will be treated with infusions every
two weeks for 12 weeks (7 infusions).


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
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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733 North Broadway
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(410) 955-3182
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
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Fairfax, Virginia 22031
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Iowa City, Iowa 52242
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3901 Rainbow Blvd
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