Gaucher DiseasePhase 1NCT01747980
Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients
A Phase 1 study testing PRX-112 in people with Gaucher Disease, run by Protalix at 2 sites in Israel.
Trial summary
Absorption of therapeutic proteins taken orally has remained the major hurdle for treatment in humans. The proteins are generally degraded by enzymes in the stomach and intestine and the intestine lining that prevents absorption into the circulation. 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 2 sitesIsrael
- Study dates
- Mar 2013to Mar 2014
- Phase
- Phase 1
- 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
- Males and females, 18 years or older.
- Historical diagnosis of Gaucher disease with leukocyte GCD activity level ≤3 nmol/mg*hr (≤30 % of the mean activity of the reference range)
- Subjects who have not received enzyme replacement therapy (ERT) or substrate replacement therapy (SRT) in the past or patients who have not received ERT in the past twelve months
- Body Mass Index (BMI) 19 to 25 kg/m2 (inclusive).
- Non-smoking (by declaration) for a period of at least 6 months prior to screening visit.
- Subjects in generally good health in the opinion of the investigator as determined by medical history, vital signs and a physical examination.
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- Negative hepatitis B or hepatitis C serology tests at screening.
- Ability to provide a written informed consent
- Female subjects of child-bearing potential or male subjects with female partners of child-bearing potential must agree to use two methods of contraception, one of which must be a barrier method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms.
Who cannot join
- Presence of any co-morbidity other than Gaucher Disease
- Presence of any GIT disease or symptomatology suspected to be GIT related using a study specific GI questionnaire
- Subjects with any history of allergic response to drugs or other allergies deemed clinically significant or exclusionary for the study, including known food allergies
- History of alcohol or drug abuse
- Subjects who donated blood in the three months, or received blood or plasma derivatives in the six months, preceding study drug administration.
- Use of any investigational drug at screening or within 3 months of dosing.
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- Subjects with an inability to communicate well with the investigators and study staff (i.e., language problem, poor mental development or impaired cerebral function).
- Subjects who are non-cooperative or unwilling to sign the consent form.
- Pregnant or nursing or planning to be pregnant during the study period.
- Have used any medication (excluding paracetamol), within 7 days of study drug administration including laxatives or other drugs, teas or food additives known to be used to treat constipation or diarrhea.
- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the investigator would interfere with the subject's compliance with the requirements of the study.
Trial locations and contacts
2 sites in 1 country.
Israel2 sites
Rambam Medical Center
Haifa, 31096
Shaare Zedek Medical Center
Jerusalem, 91031
About this study
Official title: An Exploratory, Open-label Study to Evaluate the Safety of PRX-112 and Pharmacokinetics of Oral prGCD (Plant Recombinant Human Glucocerebrosidase) in Gaucher Patients
Absorption of therapeutic proteins taken orally has remained the major hurdle for treatment in humans. The proteins are generally degraded by enzymes in the stomach and intestine and the intestine lining that prevents absorption into the circulation. Administration of PRX-112, a plant recombinant human glucocerebrosidase (prGCD) using plant cells as carrier vehicle, may help overcome many of these hurdles. The plant cell wall protects the protein from degradation in its transport through the upper GI and allows release in the lower intestine. Studies in animals have shown that prGCD delivered in this way can be found in the blood stream in an active form.
Read the detailed description
This exploratory, open-label safety and pharmacokinetic (PK) study is designed to assess the delivery of prGCD after oral administration of PRX-112 in Gaucher subjects. Subjects will receive an oral dose of PRX-112 in a single administration and followed by 3 consecutive daily administrations at the same dose. prGCD levels in plasma will be determined at selected time points. Safety parameters will also be assessed at selected time points. Enrollment will proceed into the next dosage cohort after the pharmacokinetic and safety data of the previous cohort have been reviewed. A different dosage may be selected based on the pharmacokinetic results of the first cohort.
- Conditions
- Gaucher Disease
- Being tested
- PRX-112
- Keywords
- Gaucher disease, human glucocerebrosidase, plant recombinant human glucocerebrosidase
- 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 plant expressed recombinant human glucocerebrosidase
Single dose level, four doses per cohort
| Study group | Role | What they receive |
|---|---|---|
| PRX-112 | Experimental | 250 mL of resuspended carrot cells administered orally in a vehicle Receives: Drug: PRX-112 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Spontaneous reports of adverse events, or events identified during physical examination or clinical laboratory testing
Measured: 3 days after the last dose
Secondary outcomes (3)
Area Under the Curve (AUC)
Area under the GCD level curve 0-30 hours (AUC0-30h)
Measured: From start of infusion to 30 hours after infusion
Maximum Concentration (Cmax)
Measured: From start of infusion to 30 hours after infusion
Time of maximum prGCD concentration (Tmax)
Measured: From start of infusion to 30 hours after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2012 (estimated)
- Study startMar 2013
- Primary completionJan 2014
- Study completionMar 2014
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Einat Almon, PhD, Study chair, Protalix Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01747980
- Sponsor's protocol ID
- PB-112-01
- Status last verified
- Apr 2014
- First submitted
- 5 Dec 2012
- Last updated
- 7 Apr 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory, Open-label Study to Evaluate the Safety of PRX-112 and Pharmacokinetics of Oral prGCD (Plant Recombinant Human Glucocerebrosidase) in Gaucher Patients
- ClinicalTrials.gov ID
- NCT01747980
- Acronym / sponsor ID
- PB-112-01
- Conditions
- Gaucher Disease
- Keywords
- Gaucher disease, human glucocerebrosidase, plant recombinant human glucocerebrosidase
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- Mar 2013
- Primary completion
- Jan 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 12 Dec 2012 (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.
Absorption of therapeutic proteins taken orally has remained the major hurdle for treatment in humans. The proteins are generally degraded by enzymes in the stomach and intestine and the intestine lining that prevents absorption into the circulation. Administration of PRX-112, a plant recombinant human glucocerebrosidase (prGCD) using plant cells as carrier vehicle, may help overcome many of these hurdles. The plant cell wall protects the protein from degradation in its transport through the upper GI and allows release in the lower intestine. Studies in animals have shown that prGCD delivered in this way can be found in the blood stream in an active form.
Detailed description
This exploratory, open-label safety and pharmacokinetic (PK) study is designed to assess the delivery of prGCD after oral administration of PRX-112 in Gaucher subjects. Subjects will receive an oral dose of PRX-112 in a single administration and followed by 3 consecutive daily administrations at the same dose. prGCD levels in plasma will be determined at selected time points. Safety parameters will also be assessed at selected time points. Enrollment will proceed into the next dosage cohort after the pharmacokinetic and safety data of the previous cohort have been reviewed. A different dosage may be selected based on the pharmacokinetic results of the first cohort.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PRX-112 | Experimental | Drug: PRX-112250 mL of resuspended carrot cells administered orally in a vehicle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males and females, 18 years or older.
- Historical diagnosis of Gaucher disease with leukocyte GCD activity level ≤3 nmol/mg*hr (≤30 % of the mean activity of the reference range)
- Subjects who have not received enzyme replacement therapy (ERT) or substrate replacement therapy (SRT) in the past or patients who have not received ERT in the past twelve months
- Body Mass Index (BMI) 19 to 25 kg/m2 (inclusive).
- Non-smoking (by declaration) for a period of at least 6 months prior to screening visit.
Key exclusion criteria (11 listed)
- Presence of any co-morbidity other than Gaucher Disease
- Presence of any GIT disease or symptomatology suspected to be GIT related using a study specific GI questionnaire
- Subjects with any history of allergic response to drugs or other allergies deemed clinically significant or exclusionary for the study, including known food allergies
- History of alcohol or drug abuse
- Subjects who donated blood in the three months, or received blood or plasma derivatives in the six months, preceding study drug administration.
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: plant expressed recombinant human glucocerebrosidaseSingle dose level, four doses per cohort | Drug | PRX-112 |
Primary outcome
Adverse Events
Time frame: 3 days after the last dose
Spontaneous reports of adverse events, or events identified during physical examination or clinical laboratory testing
3 secondary outcomes
Area Under the Curve (AUC)
Time frame: From start of infusion to 30 hours after infusion
Maximum Concentration (Cmax)
Time frame: From start of infusion to 30 hours after infusion
Time of maximum prGCD concentration (Tmax)
Time frame: From start of infusion to 30 hours after infusion
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 7 Apr 2014.
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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.
Einat Almon, PhD study chair
Protalix Ltd.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PRX-112 in people with Gaucher Disease, run by Protalix at 2 sites 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 11 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 7 Apr 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Adverse Events (time frame: 3 days after the last dose). 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 7 Apr 2014.