Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

This is an interventional treatment trial for Gaucher Disease focused on measuring Gaucher disease, human glucocerebrosidase, plant recombinant human glucocerebrosidase Read More

Inclusion Criteria:

• Males and females, 18 years or older.
• Historical diagnosis of Gaucher disease with leukocyte GCD activity level ≤3 nmol/mg*hr (≤30 % of the mean activity of the reference range)
• Subjects who have not received enzyme replacement therapy (ERT) or substrate replacement therapy (SRT) in the past or patients who have not received ERT in the past twelve months
• Body Mass Index (BMI) 19 to 25 kg/m2 (inclusive).
• Non-smoking (by declaration) for a period of at least 6 months prior to screening visit.
• Subjects in generally good health in the opinion of the investigator as determined by medical history, vital signs and a physical examination.
• Negative hepatitis B or hepatitis C serology tests at screening.
• Ability to provide a written informed consent
• Female subjects of child-bearing potential or male subjects with female partners of child-bearing potential must agree to use two methods of contraception, one of which must be a barrier method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms.

Exclusion Criteria:

• Presence of any co-morbidity other than Gaucher Disease
• Presence of any GIT disease or symptomatology suspected to be GIT related using a study specific GI questionnaire
• Subjects with any history of allergic response to drugs or other allergies deemed clinically significant or exclusionary for the study, including known food allergies
• History of alcohol or drug abuse
• Subjects who donated blood in the three months, or received blood or plasma derivatives in the six months, preceding study drug administration.
• Use of any investigational drug at screening or within 3 months of dosing.
• Subjects with an inability to communicate well with the investigators and study staff (i.e., language problem, poor mental development or impaired cerebral function).
• Subjects who are non-cooperative or unwilling to sign the consent form.
• Pregnant or nursing or planning to be pregnant during the study period.
• Have used any medication (excluding paracetamol), within 7 days of study drug administration including laxatives or other drugs, teas or food additives known to be used to treat constipation or diarrhea.
• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the investigator would interfere with the subject's compliance with the requirements of the study.