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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01747980
• Date of registration: 05/12/2012
• Prospective Registration: Yes
• Primary sponsor: Protalix
• Public title: Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients
• Scientific title: An Exploratory, Open-label Study to Evaluate the Safety of PRX-112 and Pharmacokinetics of Oral prGCD (Plant Recombinant Human Glucocerebrosidase) in Gaucher Patients
• Date of first enrolment: March 2013
• Target sample size: 16
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01747980
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Israel

Contacts

• Name: Einat Almon, PhD
• Affiliation: Protalix Ltd

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Gaucher Disease

Intervention(s)

Drug: PRX-112

Primary Outcome(s)

Adverse Events [Time Frame: 3 days after the last dose]

Secondary Outcome(s)

Area Under the Curve (AUC) [Time Frame: From start of infusion to 30 hours after infusion]

Time of maximum prGCD concentration (Tmax) [Time Frame: From start of infusion to 30 hours after infusion]

Maximum Concentration (Cmax) [Time Frame: From start of infusion to 30 hours after infusion]

Secondary ID(s)

PB-112-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors