Gaucher DiseasePhase 3NCT01161914
The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
A Phase 3 study testing Cerezyme® and ISU302 in people with Gaucher Disease, run by ISU Abxis Co., Ltd..
Trial summary
The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients Read more about this study
- Who is it for?
- Ages 2 to 75 years
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- Cerezyme® and ISU302
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jan 2011to Aug 2011
- Phase
- Phase 3
- 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 2 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The subjects have a diagnosis of Type I Gaucher Disease
- Subjects between 2 years old and 75 years old
- Subjects documented with glucocerebrosidase deficiency
- Subjects with splenomegaly (as indicated by CT volumetric analysis as 5 times over than the standard size (0.2% of total body weight in kilograms))
- A hemoglobin concentration level:Male > 12 years of age <12.0 g/dL Female>12 years of age<11.0 g/dL Child > 2 years of age and <12 years of age <10.5 g/dL
- Platelet count of < 120,000 / ㎣
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- Treatment-naive to enzyme replacement therapy (ERT) or treatment- experienced subjects who have not received ERT in the 12 months before screening and antibody test result is negative
- Treatment naive to substrate reduction therapy (SRT) or treatment- experienced subjects who have not received SRT in the 12 months before screening
- Subjects or their spouses who provide consent to use one of following contraception methods, or women in menopause. (In this case, menopause is defined as a period after 12 months from the last menstruation)
- Condoms, sponge, foams, jellies, diaphragm, or intrauterine device (Spouse or patient who had vasectomy or tubal ligation/ hysterectomy)
- Total abstinence from sexual intercourse: Female patient using oral contraceptives must use other contraception method (barrier method) also during the trial period and after the completion of trial as well as up to 90 days from the conclusion of trial.
- The subject or their legal representative has signed the informed consent.
Who cannot join
- Treatment with any investigational product in 90 days before study entry
- Partial or total splenectomy
- Subjects who have a serious concurrent disease like infection or who abuse addictive drug and substances.
- Pregnant and/or breast-feeding women
- Presence of Hepatitis B surface antigen or Hepatitis C or the patients show positive reaction to human immunodeficiency virus (HIV) type1
- Subjects with a history of allergic reaction to Imiglucerase
- Subjects with a history of severe pulmonary hypertension caused by Gaucher Disease
- Any subject whom the investigator or the sub investigator considers as inad equate for this trial
About this study
Official title: A Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher Disease
The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients
- Conditions
- Gaucher Disease
- Being tested
- Cerezyme®, ISU302
- Keywords
- Type I Gaucher
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cerezyme®Drug
administered by IV infusion for a dose of 60 U/kg
ISU302Drug
administered by IV infusion for a dose of 60 U/kg
| Study group | Role | What they receive |
|---|---|---|
| Cerezyme® | Active comparator | 60 U/kg infusion (every 2 weeks for 24 weeks) Receives: Drug: Cerezyme® |
| ISU302 | Experimental | 60 U/kg infusion (every 2 weeks for 24 weeks) Receives: Drug: ISU302 |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in splenic volume compared to baseline
Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.
Measured: one year
Secondary outcomes (1)
change in organ parameters and biochemical value compared to baseline
- Change in splenic volume compared to baseline for all subjects enrolled
- Change in hemoglobin concentration
- Change in platelet counts
- Change in liver volume compared to baseline
- Change in range variation of liver function test (ALT/AST)
- Change in skeletal status improvement level
- Change in bone mineral density
- Change in biomarkers (acid phosphatase, angiotensin-converting enzyme (ACE) and chitotriosidase)
- After the 1st administration, to evaluate pharmacokinetic profile by glucocerebrosidase activity assay
Measured: one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2010 (estimated)
- Study startJan 2011
- Primary completionAug 2011 (estimated)
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 3
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- ISU Abxis Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01161914
- Sponsor's protocol ID
- ISU302-2008
- Status last verified
- Jul 2010
- First submitted
- 11 Jul 2010
- Last updated
- 23 Aug 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-national Randomized Double Blinded Phase III Study to Evaluate the Safety and Efficacy of ISU302(Imiglucerase for Injection) or Cerezyme in Patient With Type I Gaucher Disease
- ClinicalTrials.gov ID
- NCT01161914
- Acronym / sponsor ID
- ISU302-2008
- Conditions
- Gaucher Disease
- Keywords
- Type I Gaucher
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Jan 2011
- Primary completion
- Aug 2011 (estimated)
- First posted
- 14 Jul 2010 (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.
The purpose of this study is to compare and evaluate the efficacy and safety of ISU302, an investigational product, and Cerezyme®, comparator, for Type 1 Gaucher Disease patients
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cerezyme® | Active comparator | Drug: Cerezyme®60 U/kg infusion (every 2 weeks for 24 weeks) |
| ISU302 | Experimental | Drug: ISU30260 U/kg infusion (every 2 weeks for 24 weeks) |
Who may qualify
- Ages
- 2 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- The subjects have a diagnosis of Type I Gaucher Disease
- Subjects between 2 years old and 75 years old
- Subjects documented with glucocerebrosidase deficiency
- Subjects with splenomegaly (as indicated by CT volumetric analysis as 5 times over than the standard size (0.2% of total body weight in kilograms))
- A hemoglobin concentration level:Male > 12 years of age <12.0 g/dL Female>12 years of age<11.0 g/dL Child > 2 years of age and <12 years of age <10.5 g/dL
Key exclusion criteria (8 listed)
- Treatment with any investigational product in 90 days before study entry
- Partial or total splenectomy
- Subjects who have a serious concurrent disease like infection or who abuse addictive drug and substances.
- Pregnant and/or breast-feeding women
- Presence of Hepatitis B surface antigen or Hepatitis C or the patients show positive reaction to human immunodeficiency virus (HIV) type1
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 |
|---|---|---|
| Cerezyme®administered by IV infusion for a dose of 60 U/kg | Drug | Cerezyme® |
| ISU302administered by IV infusion for a dose of 60 U/kg | Drug | ISU302 |
Primary outcome
change in splenic volume compared to baseline
Time frame: one year
Primary endpoints to demonstrate non-inferiority of ISU302 against Cerezyme® for change in splenic volume compared to baseline.
1 secondary outcome
change in organ parameters and biochemical value compared to baseline
Time frame: one year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2016.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cerezyme® and ISU302 in people with Gaucher Disease, run by ISU Abxis Co., Ltd..
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 12 inclusion and 8 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: withdrawn, as of the sponsor's last update on 23 Aug 2016. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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?
change in splenic volume compared to baseline (time frame: one year). 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 23 Aug 2016.