Gaucher DiseasePhase 1NCT00258778
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
A Phase 1 study testing Human Glucocerebrosidase (prGCD) in people with Gaucher Disease, run by Protalix.
Trial summary
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD)leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer)in the cells of the monocyte-macrophage system. Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- Human Glucocerebrosidase (prGCD)
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Nov 2005to Jan 2006
- 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 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy male or female between 18 and 45 years of age.
- Female subjects must agree to use a medically acceptable method of contraception at all times during the study and must have a negative serum pregnancy test at baseline and during the study period.
- Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives.
- Negative laboratory tests for HIV, HBsAg or HCV.
- Naive to any previous recombinant protein therapy.
- Provide written informed consent.
- Have the ability to understand the requirements of the study and to comply with the study protocol and dosing regimen.
Who cannot join
- Have 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 and thus increase the risk to the subject to unacceptable levels.
- Are pregnant or nursing.
- Presence of any acute or chronic diseases.
- Have a history of any allergies.
- Have been exposed to long-term steroid treatment.
- Had a minor operation in the last 6 months.
Show all 13Show fewer
- Have ever been exposed to any previous recombinant protein therapy.
- Have received immuno-suppressive treatment.
- Have a positive HIV, HBsAG and HCV laboratory result.
- Use any medication other than vitamins or oral contraceptives (for female).
- Have participated in another clinical trial during the previous 3 months
- Have history of alcohol or drug abuse.
- Are considered by the Investigator to be unsuitable candidate for this study.
About this study
Official title: A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD)leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer)in the cells of the monocyte-macrophage system.
This is the first trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD)which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
- Conditions
- Gaucher Disease
- Being tested
- Human Glucocerebrosidase (prGCD)
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Glucocerebrosidase (prGCD)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as measured by:
adverse events
change in vital signs
physical examination
laboratory test results
Secondary outcomes (2)
Pharmacokinetic parameters
Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Nov 2005 (estimated)
- Study startNov 2005
- Study completionJan 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Eithan Galun, MD, Principal investigator, Protalix Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00258778
- Sponsor's protocol ID
- P-01-2005
- Status last verified
- Dec 2006
- First submitted
- 23 Nov 2005
- Last updated
- 5 Dec 2006
- Results posted
- No
Publications
Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E. A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation. PLoS One. 2009;4(3):e4792. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11.
PubMed 19277123
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Non-Randomized, Open Label, Single Dose-Escalation Safety Study of Recombinant Human Glucocerebrosidase (prGCD) in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT00258778
- Acronym / sponsor ID
- P-01-2005
- Conditions
- Gaucher Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- Nov 2005
- Study completion
- Jan 2006
- First posted
- 28 Nov 2005 (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.
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD)leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer)in the cells of the monocyte-macrophage system.
This is the first trial to utilize a recombinant active form of lysosomal enzyme, glucocerebrosidase, (human prGCD)which is expressed and purified in a bioreactor system from transformed carrot plant root cell line.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Healthy male or female between 18 and 45 years of age.
- Female subjects must agree to use a medically acceptable method of contraception at all times during the study and must have a negative serum pregnancy test at baseline and during the study period.
- Females of child-bearing potential must be non-pregnant and not lactating and using adequate birth control such as oral contraceptives.
- Negative laboratory tests for HIV, HBsAg or HCV.
- Naive to any previous recombinant protein therapy.
Key exclusion criteria (13 listed)
- Have 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 and thus increase the risk to the subject to unacceptable levels.
- Are pregnant or nursing.
- Presence of any acute or chronic diseases.
- Have a history of any allergies.
- Have been exposed to long-term steroid treatment.
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 |
|---|---|---|
| Human Glucocerebrosidase (prGCD) | Drug | – |
Primary outcome
Safety as measured by:
adverse events
change in vital signs
physical examination
laboratory test results
2 secondary outcomes
Pharmacokinetic parameters
Immunological profile including: IgE, anti human prGCD antibodies, eosinophils and proteinuria
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 5 Dec 2006.
- Derived from this study: PubMed 19277123Aviezer D, Brill-Almon E, Shaaltiel Y, Hashmueli S, Bartfeld D, Mizrachi S, Liberman Y, Freeman A, Zimran A, Galun E. A plant-derived recombinant human glucocerebrosidase enzyme--a preclinical and phase I investigation. PLoS One. 2009;4(3):e4792. doi: 10.1371/journal.pone.0004792. Epub 2009 Mar 11.
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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.
Eithan Galun, MD principal investigator
Protalix Ltd.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Human Glucocerebrosidase (prGCD) in people with Gaucher Disease, run by Protalix.
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 7 inclusion and 13 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 5 Dec 2006. Status can change between updates, so confirm with the study contact before making plans.
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?
Safety as measured by:. The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00258778, which also lists 1 linked publication.
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 5 Dec 2006.