Phase 3
N=8
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
Gaucher Disease, Type 1
Bottom Line
View on ClinicalTrials.gov: NCT02770625 ↗Enrolled (actual)
8
Serious AEs
37.5%
Results posted
Jul 2017
Primary outcome: Primary: The Difference in Hemoglobin Concentration [g/dL] — 9.49; 11.44 g/dL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ISU302 (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- ISU Abxis Co., Ltd.
- Primary completion
- Mar 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Difference in Hemoglobin Concentration [g/dL] |
9.49; 11.44 | — |
| SECONDARY Platelet Counts [10^3 Platelets/uL] |
132.6; 180.3 | — |
| SECONDARY Spleen Volume |
29.047; 15.207 | — |
| SECONDARY Liver Volume |
1.530; 1.537 | — |
| SECONDARY Angiotensin-converting Enzyme Level |
195.71; 159.31 | — |
| SECONDARY Chitotriosidase Level (Nmol/mL/hr) |
11093.67; 3409.63 | — |
| SECONDARY Chemokine Ligand (CCL-18) Level [ng/mL] |
927.05; 577.331 | — |
| SECONDARY Acid Phosphatase (ACP) Level (U/L) |
25.44; 15.44 | — |
| SECONDARY Skeletal Status Improvement |
7; 7 | — |
| SECONDARY Change in Bone Mineral Density |
-0.954; -0.104 | — |
Summary
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
Eligibility Criteria
Inclusion Criteria
- 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.
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT02770625). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.