Gaucher DiseasePhase 1NCT01881633
A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
A Phase 1 study testing ISU302 in people with Gaucher Disease, run by ISU Abxis Co., Ltd..
Trial summary
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of single dosing study with three ascending dose cohorts of ISU302 in healthy subjects. Read more about this study
- Who is it for?
- Ages 20 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Gaucher Disease
- What is being tested?
- ISU302
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- Oct 2010to Nov 2010
- Phase
- Phase 1
- 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 20 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy male volunteers, aged between ≥ 20 and ≤ 45 years old
- Weight ≥ 50, with calculated body mass index of 17 and 25 kg/m2
- BMI = (Weight [kg])/(height [m])2
- Subject is informed of the investigational nature of this study and voluntarily agrees to participate in this study and comply with the relevant instructions in written
- Considered ineligible through screening test (such as medical history, physical examination, ECG, safety laboratory test) performed within 35 days prior to study start (dosing of investigational products)
Who cannot join
- With symptoms indicating acute diseases within 28 days prior to start of study (dosing of investigational product)
- History or presence of clinically significant and active cardiovascular, respiratory, renal, endocrine, hematological, gastrointestinal, central nervous system, psychiatric disorder, autoimmune disease, or malignant tumor
- Any medical history that may affect drug absorption, distribution, metabolism and excretion(e.g., inflammatory gastric disease, gastric or intestinal ulcer, hepatic or renal disease)
- With presence of clinically significant allergic disease (including mild allergic rhinitis or allergic dermatitis which does not need medication)
- With presence of clinically significant hypersensitivity to any drugs
- With hemolytic anemia, anemia due to blood loss (Hb < 14g/dL and Hct <42%)
Show all 17Show fewer
- With the results of safety laboratory test
- AST (Aspartate Transaminase) or ALT (Alanine Transaminase) > 1.5 times of upper normal limit
- Total bilirubin > 1.5 times of upper normal limit
- Subject who has immune deficiency or medication with immune suppressants
- Participation in other clinical study within 60 days prior to start of study (dosing of investigational products)
- Use of any drugs, possibly affecting drug metabolizing enzymes, within 1 month prior dosing, or any drugs, possibly affecting the results of clinical trial within 10 days or use of drug was not passes 5 x half-life of drug
- Donated whole blood within 60 days, or transfused within 20 days before the study
- History of alcohol abuse (> 14 units/week) and the subject could not stop drinking alcohol beverage during study period
- Heavy smoker (>10 cigarettes/day) or the subject could not stop smoking during study period
- Unwillingness or inability to follow the procedures outlined in the protocol
- Positive in pregnancy test in urine and unwilling to follow contraception during study period and following 3 months (for female subjects).
About this study
Official title: A Dose Block-randomized, Double-blind, Placebo-controlled, Single Dosing, Dose-escalation Phase I Clinical Trial to Evaluate the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of single dosing study with three ascending dose cohorts of ISU302 in healthy subjects.
- Conditions
- Gaucher Disease
- Being tested
- ISU302, Placebo
- Keywords
- gaucher disease, imiglucerase, ISU302
- 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
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| ISU302 | Experimental | 15 U/kg I.V. injection Receives: Drug: ISU302 |
| ISU302 30 U/kg | Experimental | Drug ISU302 I.V. injection Receives: Drug: ISU302 |
| ISU302 60 U/kg | Experimental | Drug ISU302 I.V. injection Receives: Drug: ISU302 |
| Placebo | Placebo comparator | ISU302 Placebo I.V. injection Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability
Evaluation for safety data by treatment group/ Confirmative evaluation for serious adverse drug reaction/ Descriptive statistics (arithmetic mean and standard deviation) for quantitative analysis and evaluation of change from baseline/ Frequency counts for qualitative categorization of safety data
Parameters:
- Adverse events including subjective/objective symptoms
- Physical examination
- 12-lead ECG
- Vital signs
- Local tolerability test
- Clinical laboratory test: Hematology, Coagulation, Blood Chemistry, Urinalysis
- Immunogenicity
Measured: From Screening to Day 5 post-dose
Secondary outcomes (1)
Pharmacokinetics
Pharmacokinetic Parameter assessment using non-compartmental analysis from the concentration-time data/ Summary of Pharmacokinetic parameters by treatment group using descriptive statistics/ Dose proportionality
Parameters: Cmax, AUCt, AUCinf, Tmax, t1/2, Clearance, Vd, MRTr
Measured: Day1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2013 (estimated)
- Study startOct 2010
- Primary completionNov 2010
- Study completionNov 2010
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 1
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 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
- NCT01881633
- Sponsor's protocol ID
- ISU-002
- Status last verified
- Jun 2013
- First submitted
- 14 Jun 2013
- Last updated
- 19 Jun 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose Block-randomized, Double-blind, Placebo-controlled, Single Dosing, Dose-escalation Phase I Clinical Trial to Evaluate the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
- ClinicalTrials.gov ID
- NCT01881633
- Acronym / sponsor ID
- ISU-002
- Conditions
- Gaucher Disease
- Keywords
- gaucher disease, imiglucerase, ISU302
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Oct 2010
- Primary completion
- Nov 2010 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 19 Jun 2013 (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 assess the safety, tolerability, and pharmacokinetics of single dosing study with three ascending dose cohorts of ISU302 in healthy subjects.
Study design
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| ISU302 | Experimental | Drug: ISU30215 U/kg I.V. injection |
| ISU302 30 U/kg | Experimental | Drug: ISU302Drug ISU302 I.V. injection |
| ISU302 60 U/kg | Experimental | Drug: ISU302Drug ISU302 I.V. injection |
| Placebo | Placebo comparator | Drug: PlaceboISU302 Placebo I.V. injection |
Who may qualify
- Ages
- 20 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- Healthy male volunteers, aged between ≥ 20 and ≤ 45 years old
- Weight ≥ 50, with calculated body mass index of 17 and 25 kg/m2
- Subject is informed of the investigational nature of this study and voluntarily agrees to participate in this study and comply with the relevant instructions in written
- Considered ineligible through screening test (such as medical history, physical examination, ECG, safety laboratory test) performed within 35 days prior to study start (dosing of investigational products)
Key exclusion criteria (17 listed)
- With symptoms indicating acute diseases within 28 days prior to start of study (dosing of investigational product)
- History or presence of clinically significant and active cardiovascular, respiratory, renal, endocrine, hematological, gastrointestinal, central nervous system, psychiatric disorder, autoimmune disease, or malignant tumor
- Any medical history that may affect drug absorption, distribution, metabolism and excretion(e.g., inflammatory gastric disease, gastric or intestinal ulcer, hepatic or renal disease)
- With presence of clinically significant allergic disease (including mild allergic rhinitis or allergic dermatitis which does not need medication)
- With presence of clinically significant hypersensitivity to any drugs
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 |
|---|---|---|
| ISU302 | Drug | ISU302, ISU302 30 U/kg, ISU302 60 U/kg |
| Placebo | Drug | Placebo |
Primary outcome
Safety and tolerability
Time frame: From Screening to Day 5 post-dose
Evaluation for safety data by treatment group/ Confirmative evaluation for serious adverse drug reaction/ Descriptive statistics (arithmetic mean and standard deviation) for quantitative analysis and evaluation of change from baseline/ Frequency counts for qualitative categorization of safety data Parameters: * Adverse events including subjective/objective symptoms * Physical examination * 12-lead ECG * Vital signs * Local tolerability test * Clinical laboratory test: Hematology, Coagulation, Blood Chemistry, Urinalysis * Immunogenicity
1 secondary outcome
Pharmacokinetics
Time frame: Day1
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 19 Jun 2013.
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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 1 study testing 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 5 inclusion and 17 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 19 Jun 2013. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 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?
Safety and tolerability (time frame: From Screening to Day 5 post-dose). 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 19 Jun 2013; AzTrials last refreshed its copy on 8 Nov 2026.