Gaucher DiseasePhase 2NCT02107846
An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112
A Phase 2 study testing PRX-112 in people with Gaucher Disease, run by Protalix at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- PRX-112
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteIsrael
- Study dates
- Apr 2014to Dec 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- PRX-112
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRX-112Drug
Also called Carrot Cells Expressing Recombinant Human Glucocerebrosidase
| Study group | Role | What they receive |
|---|---|---|
| 50 Units | Experimental | PRX-112 50 Units daily for 5 days Receives: Drug: PRX-112 |
| 100 Units | Experimental | PRX-112 100 Units daily for 5 days Receives: Drug: PRX-112 |
| 200 Units | Experimental | PRX-112 200 Units daily for 5 days Receives: Drug: PRX-112 |
| 400 Units | Experimental | PRX-112 400 Units daily for 5 days Receives: Drug: PRX-112 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area under the curve
Blood samples for GCD level every 2 hours for 24 hours
Measured: 24 hours
Adverse Events
Reporting of adverse events collected daily
Measured: 7 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2014 (estimated)
- Study startApr 2014
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02107846
- Sponsor's protocol ID
- PB-112-02a
- Status last verified
- Sep 2016
- First submitted
- 4 Apr 2014
- Last updated
- 28 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02107846
- Acronym / sponsor ID
- PB-112-02a
- Conditions
- Gaucher Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- Apr 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 8 Apr 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 50 Units | Experimental | Drug: PRX-112PRX-112 50 Units daily for 5 days |
| 100 Units | Experimental | Drug: PRX-112PRX-112 100 Units daily for 5 days |
| 200 Units | Experimental | Drug: PRX-112PRX-112 200 Units daily for 5 days |
| 400 Units | Experimental | Drug: PRX-112PRX-112 400 Units daily for 5 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (9 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PRX-112Also: Carrot Cells Expressing Recombinant Human Glucocerebrosidase | Drug | 100 Units, 200 Units, 400 Units, 50 Units |
Primary outcome
Area under the curve
Time frame: 24 hours
Blood samples for GCD level every 2 hours for 24 hours
Adverse Events
Time frame: 7 Days
Reporting of adverse events collected daily
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2016.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PRX-112 in people with Gaucher Disease, run by Protalix at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area under the curve (time frame: 24 hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2016; AzTrials last refreshed its copy on 29 Oct 2026.