Gaucher Disease, Type 1Phase 3NCT02770625
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
A Phase 3 study testing ISU302 in people with Gaucher Disease, Type 1, 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Gaucher Disease, Type 1
- What is being tested?
- ISU302
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- No sites listed yet
- Study dates
- Sep 2011to Aug 2014
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of Type 1 GD.
- Documented glucocerebrosidase deficiency.
- GD-related anemia, defined as hemoglobin levels of at least 1 g/dL below the lower limit of normal for age and gender and one or more of the following 3 criteria:
- At least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation,
- GD-related thrombocytopenia, defined as a platelet count <90 x 109 platelets/L,
- GD-related readily palpable enlarged liver.
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- Not received treatment for GD (investigational products, miglustat, velaglucerase alfa, or imiglucerase) within 12 months prior to study entry.
- Ability to comprehend and willing to sign the ICF.
- Legal guardian (and patient if age appropriate) understood the nature of the procedure, was willing to comply with associated follow-up evaluations, and provided written informed consent and assent prior to the procedure.
- Female patients of childbearing potential must had agreed to use a medically acceptable method of contraception at all the times during the study. Male patients must have used a medically acceptable method of birth control throughout their participation in the study and were required to report the pregnancy of a partner.
Who cannot join
- Type 2 or 3 GD.
- Splenectomy.
- Antibody positive to ISU302 or imiglucerase during screening or the patient had experienced an anaphylactic reaction to ISU302 or imiglucerase. - Treatment with any non-GD-related investigational drug or medical device within 30 days prior to study entry; such use during the study was also not permitted.
- Currently receiving red blood cell (RBC) growth factor (eg, erythropoietin) chronic systemic corticosteroids or received such treatment within the last 6 months.
- Positive for human immunodeficiency virus (HIV) and hepatitis B or C.
- Exacerbated anemia at screening (due to iron, folic acid, or vitamin B12 deficiency or infectious/immune-mediated cause).
- Significant comorbidity(ies) that could affect study data or confounded the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease).
- Pregnant or lactating female patients and those not willing to use highly effective barrier or medical method of contraception.
About this study
Official title: A Multicenter, Open-Label Phase III Study to Evaluate the Safety and Efficacy of ISU302 (Imiglucerase for Injection) in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
Read the detailed description
The objectives of this clinical study were to evaluate the efficacy and safety of every other week (EOW) dosing of ISU302 at a dose of 60 U/kg as an effective glucocerebrosidase enzyme replacement therapeutic product in patients with Type 1 Gaucher disease (GD). Primary efficacy endpoint was the difference in hemoglobin concentration between baseline and Week 24. Secondary efficacy endpoints included assessment of platelet counts, spleen and liver volume, and biomarker levels in plasma at Week 24 compared to baseline; skeletal change and bone mineral density (BMD); and single-dose pharmacokinetic (PK) analysis. Secondary safety endpoints included the assessment of adverse events (AEs), vital signs, physical examination, and electrocardiogram (ECG); clinical safety laboratory analyses included serum chemistry, urinalysis, hematology and coagulation, and the measurement of anti-ISU302 antibodies.
- Conditions
- Gaucher Disease, Type 1
- Being tested
- ISU302
- 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.
ISU302Drug
Also called Imiglucerase
60 U/kg given intravenously
| Study group | Role | What they receive |
|---|---|---|
| ISU302 | Experimental | 60 U/kg (once every 2 weeks for 6 months) Receives: Drug: ISU302 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Difference in Hemoglobin Concentration [g/dL]
Measured: from baseline to Week 24
Secondary outcomes (9)
Platelet Counts [10^3 Platelets/uL]
Measured: from baseline to Week 24
Spleen Volume
Measured: from baseline to Week 24
Liver Volume
Measured: from baseline to Week 24
Angiotensin-converting Enzyme Level
Measured: from baseline to Week 24
Chitotriosidase Level (Nmol/mL/hr)
Measured: from baseline to Week 24
Chemokine Ligand (CCL-18) Level [ng/mL]
Measured: from baseline to Week 24
Acid Phosphatase (ACP) Level (U/L)
Measured: from baseline to Week 24
Skeletal Status Improvement
The number of participant who have the skeletal status diagnosed as Osteosclerosis
Measured: from baseline to Week 24
Change in Bone Mineral Density
Measured: from baseline 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 posted12 May 2016 (estimated)
- Study startSep 2011
- Primary completionMar 2013
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Gaucher Disease, Type 1Studies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- ISU Abxis Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amel El Beshlawy, Prof., Principal investigator, Abou El Reesh Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02770625
- Sponsor's protocol ID
- ISU302-004
- Status last verified
- Feb 2017
- First submitted
- 7 May 2016
- Last updated
- 12 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label Phase III Study to Evaluate the Safety and Efficacy of ISU302 (Imiglucerase for Injection) in Patients With Type 1 Gaucher Disease
- ClinicalTrials.gov ID
- NCT02770625
- Acronym / sponsor ID
- ISU302-004
- Conditions
- Gaucher Disease, Type 1
- Keywords
- Type I Gaucher, ISU302, Imiglucerase
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Sep 2011
- Primary completion
- Mar 2013 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 12 May 2016 (estimated)
- Results
- Posted 12 Jul 2017
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.
Detailed description
The objectives of this clinical study were to evaluate the efficacy and safety of every other week (EOW) dosing of ISU302 at a dose of 60 U/kg as an effective glucocerebrosidase enzyme replacement therapeutic product in patients with Type 1 Gaucher disease (GD). Primary efficacy endpoint was the difference in hemoglobin concentration between baseline and Week 24. Secondary efficacy endpoints included assessment of platelet counts, spleen and liver volume, and biomarker levels in plasma at Week 24 compared to baseline; skeletal change and bone mineral density (BMD); and single-dose pharmacokinetic (PK) analysis. Secondary safety endpoints included the assessment of adverse events (AEs), vital signs, physical examination, and electrocardiogram (ECG); clinical safety laboratory analyses included serum chemistry, urinalysis, hematology and coagulation, and the measurement of anti-ISU302 antibodies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ISU302 | Experimental | Drug: ISU30260 U/kg (once every 2 weeks for 6 months) |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of Type 1 GD.
- Documented glucocerebrosidase deficiency.
- GD-related anemia, defined as hemoglobin levels of at least 1 g/dL below the lower limit of normal for age and gender and one or more of the following 3 criteria:
- Not received treatment for GD (investigational products, miglustat, velaglucerase alfa, or imiglucerase) within 12 months prior to study entry.
- Ability to comprehend and willing to sign the ICF.
Key exclusion criteria (8 listed)
- Type 2 or 3 GD.
- Splenectomy.
- Antibody positive to ISU302 or imiglucerase during screening or the patient had experienced an anaphylactic reaction to ISU302 or imiglucerase. - Treatment with any non-GD-related investigational drug or medical device within 30 days prior to study entry; such use during the study was also not permitted.
- Currently receiving red blood cell (RBC) growth factor (eg, erythropoietin) chronic systemic corticosteroids or received such treatment within the last 6 months.
- Positive for human immunodeficiency virus (HIV) and hepatitis B or C.
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 |
|---|---|---|
| ISU302Also: Imiglucerase60 U/kg given intravenously | Drug | ISU302 |
Primary outcome
The Difference in Hemoglobin Concentration [g/dL]
Time frame: from baseline to Week 24
9 secondary outcomes
Platelet Counts [10^3 Platelets/uL]
Time frame: from baseline to Week 24
Spleen Volume
Time frame: from baseline to Week 24
Liver Volume
Time frame: from baseline to Week 24
Angiotensin-converting Enzyme Level
Time frame: from baseline to Week 24
Chitotriosidase Level (Nmol/mL/hr)
Time frame: from baseline to Week 24
Chemokine Ligand (CCL-18) Level [ng/mL]
Time frame: from baseline to Week 24
Acid Phosphatase (ACP) Level (U/L)
Time frame: from baseline to Week 24
Skeletal Status Improvement
Time frame: from baseline to Week 24
Change in Bone Mineral Density
Time frame: from baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Undecided
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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.
Amel El Beshlawy, Prof. principal investigator
Abou El Reesh Children's Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ISU302 in people with Gaucher Disease, Type 1, 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 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: finished, as of the sponsor's last update on 12 Jul 2017. 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?
Yes. Results are posted in the registry (first posted 12 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Difference in Hemoglobin Concentration [g/dL] (time frame: from baseline to Week 24). 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 12 Jul 2017.