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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02107846
Recruitment Status : Completed
First Posted : April 8, 2014
Last Update Posted : September 28, 2016
Sponsor:
Information provided by (Responsible Party):
Protalix

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.

Condition or disease Intervention/treatment Phase
Gaucher Disease Drug: PRX-112 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Study Start Date : April 2014
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arm Intervention/treatment
Experimental: 50 Units
PRX-112 50 Units daily for 5 days
Drug: PRX-112
Other Name: Carrot Cells Expressing Recombinant Human Glucocerebrosidase

Experimental: 100 Units
PRX-112 100 Units daily for 5 days
Drug: PRX-112
Other Name: Carrot Cells Expressing Recombinant Human Glucocerebrosidase

Experimental: 200 Units
PRX-112 200 Units daily for 5 days
Drug: PRX-112
Other Name: Carrot Cells Expressing Recombinant Human Glucocerebrosidase

Experimental: 400 Units
PRX-112 400 Units daily for 5 days
Drug: PRX-112
Other Name: Carrot Cells Expressing Recombinant Human Glucocerebrosidase




Primary Outcome Measures :
  1. Area under the curve [ Time Frame: 24 hours ]
    Blood samples for GCD level every 2 hours for 24 hours

  2. Adverse Events [ Time Frame: 7 Days ]
    Reporting of adverse events collected daily



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02107846


Locations
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Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Sponsors and Collaborators
Protalix

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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT02107846     History of Changes
Other Study ID Numbers: PB-112-02a
First Posted: April 8, 2014    Key Record Dates
Last Update Posted: September 28, 2016
Last Verified: September 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
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Gaucher Disease
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders