Fabry DiseasePhase 1NCT01653444
Evaluate the Safety and Exploratory Efficacy of GC1119
A Phase 1 study testing GC1119 in people with Fabry Disease, run by Green Cross Corporation at 3 sites in South Korea.
Trial summary
The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients. Read more about this study
- Who is it for?
- Ages 16 years and older, men only
- Conditions the study is for
- Fabry Disease
- What is being tested?
- GC1119
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 3 sitesSouth Korea
- Study dates
- Nov 2012to Oct 2015
- 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 16 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects with a current diagnosis of Fabry's disease
- Plasma α-gal activity of ≤ 1.5mnol/hr/ml and have a mutation in α-galactosidase A gene
- Males ≥ 16 years old
- Subjects capable of performing this clinical trial in an appropriate manner
- Informed consent form voluntarily signed by the subject(or his legally acceptable representative if the subject is under 20 years old) to participation in the study
- Agreement to contraception during the study period
Who cannot join
- Serum creatinine > 2.5mg/dl
- Subjects have a plan to kidney transplantation
- Subjects have undergone kidney transplantation
- Subjects are currently on dialysis
- Subjects have a clinically significant organic disease(cardiovascular, hepatic, pulmonary, neurologic, or renal disease)that in the opinion of the investigator would preclude participation in the trial
- Known life-threatening hypersensitivity(anaphylactic reaction) to α-galactosidase
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- Treatment with another investigational product within 30days from the administration of study drug dosing or plans to be treated with another investigational product during the study period
- Known hypersensitivity to any of the ingredients of study drug(including excipients)
- Subjects need the medication of prohibited drug
- Alcoholism or drug addiction
Trial locations and contacts
3 sites in 1 country.
South Korea3 sites
Asan Medical Center
Songpa-gu, Seoul
Ajou University School of Medicine
Yeongtong-gu, Suwon
Soon Cung Hyang University Hospital
Yongsan-gu, Seoul
About this study
Official title: Multicenter and Dose Escalation Phase 1 Study to Evaluate the Safety and Exploratory Efficacy of GC1119(Recombinant Human α-galactosidase A) for Enzyme Replacement Therapy in Fabry Disease Patients
The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
- Conditions
- Fabry Disease
- Being tested
- GC1119
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC1119Drug
biweekly, IV infusion
| Study group | Role | What they receive |
|---|---|---|
| GC1119 0.5 mg/kg | Experimental | 0.5 mg/kg biweekly Receives: Drug: GC1119 |
| GC1119 1.0 mg/kg | Experimental | 1.0 mg/kg biweekly Receives: Drug: GC1119 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Measured: 10weeks
Secondary outcomes (8)
change and %change of Plasma GL-3 concentration
Measured: baseline and 10weeks
The ratio of subjects whose plasma GL-3 values are within reference range
Measured: 10weeks
change and %change of urine GL-3 concentration
Measured: baseline and 10weeks
change and %change of kidney function
Measured: baseline and 10weeks
change and %change of kidney size
Measured: baseline and 10weeks
change and %change of heart size
Measured: baseline and 10weeks
change of results of cornial opacity examination
Measured: baseline and 10weeks
change of scores that are measured by pain questionnaire
Measured: baseline and 10weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2012 (estimated)
- Study startNov 2012
- Primary completionMay 2014
- Study completionOct 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Green Cross Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01653444
- Sponsor's protocol ID
- GC1119_P1
- Status last verified
- Jun 2016
- First submitted
- 24 Jul 2012
- Last updated
- 24 Jun 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter and Dose Escalation Phase 1 Study to Evaluate the Safety and Exploratory Efficacy of GC1119(Recombinant Human α-galactosidase A) for Enzyme Replacement Therapy in Fabry Disease Patients
- ClinicalTrials.gov ID
- NCT01653444
- Acronym / sponsor ID
- GC1119_P1
- Conditions
- Fabry Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Green Cross CorporationIndustry (pharma, biotech, devices)
- Study start
- Nov 2012
- Primary completion
- May 2014 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 31 Jul 2012 (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 exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GC1119 0.5 mg/kg | Experimental | Drug: GC11190.5 mg/kg biweekly |
| GC1119 1.0 mg/kg | Experimental | Drug: GC11191.0 mg/kg biweekly |
Who may qualify
- Ages
- 16 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Subjects with a current diagnosis of Fabry's disease
- Plasma α-gal activity of ≤ 1.5mnol/hr/ml and have a mutation in α-galactosidase A gene
- Males ≥ 16 years old
- Subjects capable of performing this clinical trial in an appropriate manner
- Informed consent form voluntarily signed by the subject(or his legally acceptable representative if the subject is under 20 years old) to participation in the study
Key exclusion criteria (10 listed)
- Serum creatinine > 2.5mg/dl
- Subjects have a plan to kidney transplantation
- Subjects have undergone kidney transplantation
- Subjects are currently on dialysis
- Subjects have a clinically significant organic disease(cardiovascular, hepatic, pulmonary, neurologic, or renal disease)that in the opinion of the investigator would preclude participation in the trial
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 |
|---|---|---|
| GC1119biweekly, IV infusion | Drug | GC1119 0.5 mg/kg, GC1119 1.0 mg/kg |
Primary outcome
Incidence of adverse events
Time frame: 10weeks
8 secondary outcomes
change and %change of Plasma GL-3 concentration
Time frame: baseline and 10weeks
The ratio of subjects whose plasma GL-3 values are within reference range
Time frame: 10weeks
change and %change of urine GL-3 concentration
Time frame: baseline and 10weeks
change and %change of kidney function
Time frame: baseline and 10weeks
change and %change of kidney size
Time frame: baseline and 10weeks
change and %change of heart size
Time frame: baseline and 10weeks
change of results of cornial opacity examination
Time frame: baseline and 10weeks
change of scores that are measured by pain questionnaire
Time frame: baseline and 10weeks
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 24 Jun 2016.
- Individual participant dataSharing planned: No
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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.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GC1119 in people with Fabry Disease, run by Green Cross Corporation at 3 sites in South Korea.
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 6 inclusion and 10 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 24 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
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 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?
Incidence of adverse events (time frame: 10weeks). 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 24 Jun 2016.