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

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

This is an open-label, dose escalation study to evaluate the safety of oral PRX-112 and pharmacokinetics of GCD in subjects with Gaucher disease naive to enzyme replacement therapy. The dose levels of PRX-112 are 50 units, 100 units, 200 units and 400 units GCD. Subjects will receive once daily oral administrations of PRX-112 for 5 consecutive days at each dose level with a 2-day washout period between doses.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: PRX-112

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Jerusalem, Israel
    • Shaare Zedek Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 50 Units; PRX-112 50 Units daily for 5 days	Drug: PRX-112; Other Names: Carrot Cells Expressing Recombinant Human Glucocerebrosidase
Experimental: 100 Units; PRX-112 100 Units daily for 5 days	Drug: PRX-112; Other Names: Carrot Cells Expressing Recombinant Human Glucocerebrosidase
Experimental: 200 Units; PRX-112 200 Units daily for 5 days	Drug: PRX-112; Other Names: Carrot Cells Expressing Recombinant Human Glucocerebrosidase
Experimental: 400 Units; PRX-112 400 Units daily for 5 days	Drug: PRX-112; Other Names: Carrot Cells Expressing Recombinant Human Glucocerebrosidase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Area under the curve	Blood samples for GCD level every 2 hours for 24 hours	24 hours
Adverse Events	Reporting of adverse events collected daily	7 Days

Collaborators and Investigators

• Sponsor: Protalix

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (Actual): December 1, 2015
• Study Completion (Actual): December 1, 2015

Study Registration Dates

• First Submitted: April 4, 2014
• First Submitted That Met QC Criteria: April 4, 2014
• First Posted (Estimate): April 8, 2014

Study Record Updates

• Last Update Posted (Estimate): September 28, 2016
• Last Update Submitted That Met QC Criteria: September 27, 2016
• Last Verified: September 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: PB-112-02a

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No