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

NCT02107846 | Completed Phase 2

• 1 other identifier: PB-112-02a
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (2)

• 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

Study Arms (4)

50 Units

EXPERIMENTAL

PRX-112 50 Units daily for 5 days

Drug: PRX-112

100 Units

EXPERIMENTAL

PRX-112 100 Units daily for 5 days

Drug: PRX-112

200 Units

EXPERIMENTAL

PRX-112 200 Units daily for 5 days

Drug: PRX-112

400 Units

EXPERIMENTAL

PRX-112 400 Units daily for 5 days

Drug: PRX-112

Interventions

PRX-112 DRUG

Also known as: Carrot Cells Expressing Recombinant Human Glucocerebrosidase

100 Units; 200 Units; 400 Units; 50 Units

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Protalix: lead

Study Sites (1)

Shaare Zedek Medical Center

Jerusalem, Israel

Location

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 4, 2014
• First Posted: April 8, 2014
• Study Start: April 1, 2014
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: September 28, 2016

Record last verified: 2016-09

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)