Gaucher DiseaseNCT02053896
A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
An observational study of Gaucher Disease, run by ISU Abxis Co., Ltd..
Trial summary
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase. Read more about this study
- Who is it for?
- Ages 8 to 29 years
- Conditions the study is for
- Gaucher Disease
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- May 2011to Feb 2012
- Phase
- Observational
- 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 8 to 29 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patient diagnosed with type-1 Gaucher disease
Who may be able to join
- Patient diagnosed with type-1 Gaucher disease
- Patient who was stably treating Gaucher disease with Cerezyme® and who was maintaining the usage and dosage of Cerezyme® for at least 6 months prior to study drug administration
- Patient aged 2 years or higher
- Female patient with contraception during the study period (oral or injectable contraceptive hormones, intrauterine device, physical devices using condom, sponge form, jelly, and femidom, and abstinence)
- Patient who signed the informed consent form after hearing the detailed explanation about this study
- Definition of the stable treatment of type-1 Gaucher disease:
Show all 10Show fewer
- No neurologic deficit
- Normal hemoglobin concentration, and platelet count that has increased to ≥100,000/㎣, or maintained to 100,000/㎣
- Normal or no deteriorated bone mineral density
- Normal or no deteriorated splenomegaly or hepatomegaly
Who cannot join
- Patient who participated in other clinical studies within 90 days before study drug administration
- Patient with unstable hemoglobin and platelet counts for at least 6 months before study drug administration
- Patient with hypersensitivity to Cerezyme®
- Patient positive to HIV antibody, hepatitis B antigen, and hepatitis C antibody
- Patient with Fe, folic acid, or vitamin B12-deficcient anemia
- Patient who received miglustat within 6 months before study drug administration
Show all 11Show fewer
- Patient who received erythrocyte growth factor or chronic systemic corticosteroids within 6 months before study drug administration
- Patient who had clinically significant splenic obstruction within 12 months before study drug administration
- Pregnant or lactating patient
- Patient who had serious concurrent diseases such as infectious diseases or drug-addicted patient
- Patient who was considered inappropriate for this study by the investigators or sub-investigators
About this study
Official title: A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.
- Conditions
- Gaucher Disease
- Keywords
- Type I Gaucher, ISU302, Imiglucerase
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| ISU302 | 15~60U/kg (once every 2 weeks for 6 months) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Adverse Events
To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme®
Measured: From Screening to Week 24
Secondary outcomes (6)
Pharmacokinetics
Measurement of blood glucocerebrosidase activity for pharmacokinetic assessment after initial study drug administration
Measured: From Screening to Week 24
hemoglobin concentration
Change in hemoglobin concentration
Measured: From Screening to Week 24
platelet count
Change in platelet count
Measured: From Screening to Week 24
liver and spleen volumes and liver function
Changes in liver and spleen volumes and liver function
Measured: From Screening to Week 24
biomarker levels
Changes in biomarker levels (Acid Phosphatase, Angiotensin Converting Enzyme, and Chitotriosidase)
Measured: From Screening to Week 24
skeletal status and bone mineral density
Changes in skeletal status and bone mineral density
Measured: From Screening to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2014 (estimated)
- Study startMay 2011
- Primary completionFeb 2012
- Study completionFeb 2012
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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- ISU Abxis Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02053896
- Sponsor's protocol ID
- ISU302-003
- Status last verified
- Feb 2014
- First submitted
- 26 Jan 2014
- Last updated
- 4 Feb 2014
- Results posted
- No
Publications
Choi JH, Lee BH, Ko JM, Sohn YB, Lee JS, Kim GH, Heo SH, Park JY, Kim YM, Kim JH, Yoo HW. A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin(R) in patients with type 1 Gaucher disease. J Korean Med Sci. 2015 Apr;30(4):378-84. doi: 10.3346/jkms.2015.30.4.378. Epub 2015 Mar 19.
PubMed 25829804
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Multi-Center, Open-Label, Switch-Over Trial to Evaluate the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Previously Treated With Imiglucerase
- ClinicalTrials.gov ID
- NCT02053896
- Acronym / sponsor ID
- ISU302-003
- Conditions
- Gaucher Disease
- Keywords
- Type I Gaucher, ISU302, Imiglucerase
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- May 2011
- Primary completion
- Feb 2012 (actual)
- Study completion
- Feb 2012 (actual)
- First posted
- 4 Feb 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.
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease previously treated with Imiglucerase.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ISU302 | – | –15~60U/kg (once every 2 weeks for 6 months) |
Who may qualify
- Ages
- 8 to 29 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patient diagnosed with type-1 Gaucher disease
Key inclusion criteria (10 listed)
- Patient diagnosed with type-1 Gaucher disease
- Patient who was stably treating Gaucher disease with Cerezyme® and who was maintaining the usage and dosage of Cerezyme® for at least 6 months prior to study drug administration
- Patient aged 2 years or higher
- Female patient with contraception during the study period (oral or injectable contraceptive hormones, intrauterine device, physical devices using condom, sponge form, jelly, and femidom, and abstinence)
- Patient who signed the informed consent form after hearing the detailed explanation about this study
Key exclusion criteria (11 listed)
- Patient who participated in other clinical studies within 90 days before study drug administration
- Patient with unstable hemoglobin and platelet counts for at least 6 months before study drug administration
- Patient with hypersensitivity to Cerezyme®
- Patient positive to HIV antibody, hepatitis B antigen, and hepatitis C antibody
- Patient with Fe, folic acid, or vitamin B12-deficcient anemia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of participants with Adverse Events
Time frame: From Screening to Week 24
To evaluate the safety of ISU302 in patients with Type 1 Gaucher disease previously treated with Cerezyme® after administering ISU302 alternative to Cerezyme®
6 secondary outcomes
Pharmacokinetics
Time frame: From Screening to Week 24
hemoglobin concentration
Time frame: From Screening to Week 24
platelet count
Time frame: From Screening to Week 24
liver and spleen volumes and liver function
Time frame: From Screening to Week 24
biomarker levels
Time frame: From Screening to Week 24
skeletal status and bone mineral density
Time frame: From Screening to Week 24
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 4 Feb 2014.
- Derived from this study: PubMed 25829804Choi JH, Lee BH, Ko JM, Sohn YB, Lee JS, Kim GH, Heo SH, Park JY, Kim YM, Kim JH, Yoo HW. A phase 2 multi-center, open-label, switch-over trial to evaluate the safety and efficacy of Abcertin(R) in patients with type 1 Gaucher disease. J Korean Med Sci. 2015 Apr;30(4):378-84. doi: 10.3346/jkms.2015.30.4.378. Epub 2015 Mar 19.
Related trials for Gaucher Disease
RecruitingObservational study
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Neurological Regression, Myoclonus
RecruitingObservational study
Parkinson's Disease in Type 1 Gaucher Disease
Parkinson Disease (PD), Type 1 Gaucher Disease
RecruitingObservational study
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Gaucher Disease Type I, Oxidative Stress
Not yet recruitingPhase 1/2
4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
Neuroblastoma
Enrolling by invitationObservational study
Genetic Studies of Lysosomal Storage Disorders
Lysosomal Storage Disorders, Gaucher Disease
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?
An observational study of 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 10 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 4 Feb 2014. 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.
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?
Number of participants with Adverse Events (time frame: From Screening to Week 24). 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 NCT02053896, 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 4 Feb 2014.