Clinical trial NCT02053896: A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gaucher Disease
Keywords: Type I Gaucher
ISU302
Imiglucerase
Lead sponsor: ISU Abxis Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2014-02-04
Enrollment: 5
Sex: ALL
Minimum age (years): 8
Maximum age (years): 29
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patient diagnosed with type-1 Gaucher disease

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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:
* 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

Exclusion Criteria:

* 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
* 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

Primary outcomes:
measure: Number of participants with Adverse Events
timeFrame: From Screening to Week 24
Secondary outcomes:
measure: Pharmacokinetics
timeFrame: From Screening to Week 24
measure: hemoglobin concentration
timeFrame: From Screening to Week 24
measure: platelet count
timeFrame: From Screening to Week 24
measure: liver and spleen volumes and liver function
timeFrame: From Screening to Week 24
measure: biomarker levels
timeFrame: From Screening to Week 24
measure: skeletal status and bone mineral density
timeFrame: From Screening to Week 24
Other outcomes:
Locations:
Study officials:
References:
citation: 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.