An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

• Register: ClinicalTrials.gov
• Last refreshed on: 10 October 2016
• Main ID: NCT02107846
• Date of registration: 04/04/2014
• Prospective Registration: No
• Primary sponsor: Protalix
• Public title: An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112
• Scientific title: A Phase 2a, Open-Label, Sequential Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112 (Plant Recombinant Human Glucocerebrosidase) in Enzyme Replacement Therapy-Naïve Subjects With Gaucher Disease
• Date of first enrolment: April 2014
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02107846
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Israel

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female age 18 or older

- Historical diagnosis of Gaucher disease by low leukocyte GCD activity level

- Haemoglobin = 10 g/dL

- Body mass index (BMI) of 18 kg/m2-30 kg/m2 inclusive

- Subject is able to provide written informed consent

- Female subjects of child bearing potential must not be pregnant or lactating with a
negative urine pregnancy test result at the screening visit.

- Female subjects of child-bearing potential and male subjects with female partners of
childbearing potential must use two methods of contraception at all times during the
study, one of which must be a barrier method. Acceptable methods of contraception are
oral contraceptives, barrier methods (male condom, female condom, diaphragm, cervical
cap, spermicide or intrauterine device), surgical sterility (documented doctor's
report of vasectomy, hysterectomy and/or bilateral oophorectomy) and/or
postmenopausal status (defined as at least 1 year without menses as demonstrated by
medical history or subject report).

- Negative laboratory tests for HIV, HBsAg and HCV at the screening visit

- Naïve to any previous ERT or have received the last ERT treatment 12 months before
signing IC

Exclusion Criteria:

- Presence of a gastrointestinal (GI) disease affecting motility or absorption

- Subjects with any history of allergic response to biological drugs or other allergies
deemed clinically significant by the Investigator

- Reported history of alcohol or drug abuse

- Subject has donated blood in the 3 months prior to screening or subject has received
plasma derivatives in the 6 months prior to screening

- Use of any investigational drug or participation in another clinical trial in the 3
months prior to screening (subject report)

- Subjects who have previously received ERT with positive anti-human plant recombinant
GCD (prGCD) antibodies

- Clinical evidence of any active significant disease that could potentially compromise
the ability of the Investigator to evaluate or interpret the effects of the study
treatment on safety assessment, thus increasing the risk to the subject to
unacceptable levels

- Presence of any medical, emotional, behavioural or psychological condition that, in
the judgement of the Investigator, would interfere the compliance requirements of the
study

- Subject has used any medication (excluding acetaminophen or dyprione) within 7 days
of screening, including laxatives, teas and food additives known to be used for the
treatment of constipation or diarrhea



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)

Area under the curve [Time Frame: 24 hours]

Adverse Events [Time Frame: 7 Days]

Secondary ID(s)

PB-112-02a

Source(s) of Monetary Support

Please refer to primary and secondary sponsors