Fabry DiseasePhase 3NCT06081062
Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
A Phase 3 study testing Fabagal® (Agalsidase beta) and Active comparator (Agalsidase beta) in people with Fabry Disease, run by ISU Abxis Co., Ltd. at 3 sites in 2 countries.
Trial summary
Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta). Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Fabagal® (Agalsidase beta) and Active comparator (Agalsidase beta)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 14 Apr 2023to Dec 2025
- Phase
- Phase 3
- 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 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Those who have been diagnosed with Fabry disease by genetic and alpha-galactosidase A enzyme tests and grouped by sex are as follows:
- Male: Those who have confirmed GLA mutation (variation of α-galactosidase A gene) by genetic testing, and whose activity of alpha-galactosidase A in leukocytes is 5% or less than the normal mean value
- Female: Those who have confirmed GLA mutation by genetic testing, and whose alpha-galactosidase A is within the normal range or is deficient
- Age: Those who are aged 8 years or older
- Those who have at least one of the following symptoms and signs:
- Glomerular filtration rate decreased (Inclusion criteria: 2 or more cases of 30 ≦ eGFR < 90 mL/min/1.73 m2 [adjusted for age >40] [including results within 6 months of the screening visit, but including results within 12 months for patients with a 60 ≦ eGFR < 90 mL/min/1.73 m2])
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- Proteinuria that is equivalent to microalbuminuria or worse (Inclusion criteria: 2 or more cases of creatinine 30 mg/g in random urine at least 24 hours apart [including results within 6 months of the screening visit] or ≥30 mg of albuminuria in 24-hour urine)
- For 24 hr urinary protein extraction (>4 mg/m2/hr) or for spot urinary protein/creatinine ratio (≥200 mg/g [Cr]) *Pediatrics: Aged <19 years
- Abnormal left ventricular function as evidenced by MRI or echocardiography
- Left ventricular mass index (LVMI)* >115 g/m2 (male), >95 g/m2 (female) or
- Left ventricular wall thickness >12 mm (However, in the case of patients with hypertension, patients must have blood pressure treatment for at least 6 months prior to administration of the same drug) etc.
- Clinically significant arrhythmias and conduction disturbances, etc.
- Stroke or transient ischemic attack, etc., as evidenced by objective testing
- Patients who have not previously received enzyme replacement therapy (ERT) or Chaperone therapy for treatment of Fabry disease
- Patients who voluntarily consented and signed the informed consent form
- Patients (female patients and partners of male patients who are of childbearing potential) who have agreed to use a medically appropriate method of contraception (intrauterine device, condoms, surgical methods such as vasectomy) during the clinical study
Who cannot join
- Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
- Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
- Patients who are currently on dialysis or have a history of kidney transplantation, or patients scheduled for dialysis at the time of screening, or waitlisted for kidney transplantation
- Patients who have started angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) treatment within 4 weeks prior to the screening visit or whose dose has been changed
- Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
- Patients with a history of HIV, hepatitis B/C or HIV antibodies, hepatitis B surface antigens, or hepatitis C antibodies
- Patient whose medical, emotional, behavioral, or psychological conditions appear to interfere with compliance with the requirements of the clinical study according to the investigator's judgment
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Philippines2 sites, 2 recruiting
- Recruiting
Philippine General Hospital
Manila
Maria Melanie Liberty Alcausin, 639178501569, mbalcausin@up.edu.ph
Roxanne Janica Merencilla, 639178908314, remerencilla@up.edu.ph
- Recruiting
St.Luke's Medical Center
Manila
Cheryll Magbanua-Calalo, 639175138378, cjmagbanua@up.edu.ph
Luna Reyes, 639178019052, luna_reyes1226@yahoo.com
South Korea1 site, 1 recruiting
- Recruiting
Seoul Asan Center
Seoul, Songpa-gu, 05505
Beomhee Lee
About this study
Official title: A Phase 3 Randomized, Double-blinded, Active-controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).
Read the detailed description
The primary objective is to evaluate the efficacy of Fabagal compared to active comparator (Agalsidase beta). The secondary objectives will evaluate the safety, pharmacokinetics, and immunogenicity of Fabagal compared to active comparator (Agalsidase beta).
- Conditions
- Fabry Disease
- Being tested
- Fabagal® (Agalsidase beta), Active comparator (Agalsidase beta)
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fabagal® (Agalsidase beta)Biological
1 mg/kg every 2 weeks for 12 months
Active comparator (Agalsidase beta)Drug
1 mg/kg every 2 weeks for 12 months
| Study group | Role | What they receive |
|---|---|---|
| Fabagal® (Agalsidase beta) | Experimental | 1 mg/kg, administered every 2 weeks for 12 months Receives: Biological: Fabagal® (Agalsidase beta) |
| Active Comparator (Agalsidase beta) | Active comparator | 1 mg/kg, administered every 2 weeks for 12 months Receives: Drug: Active comparator (Agalsidase beta) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the efficacy of Fabagal compared with active comparator (Agalsidase beta)
The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (Agalsidase beta) in patients with Fabry disease
The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (agalsidase beta) in patients with Fabry disease The primary endpoint is a comparison of the proportion of subjects in each group who achieve a score of Zero on the renal capillary endothelium histology.
The score is graded on a scale from 0-3 (normal, mild, moderate, and severe).
- 0: signified no visible inclusions
- 1: signified multiple discrete lipid granules
- 2: signified single or multiple aggregates of lipid granules
- 3: signified aggregates of lipid granules either large enough or numerous enough to cause clear distortion of the luminal surface
Measured: Screening visit (Visit 0) and Visit 25 (Day 336)
Secondary outcomes (5)
Change from baseline in GL-3 levels in renal cells after administration of 24 doses of Fabagal compared to active comparator
After administering Fabagal injection and active control group 24 times, changes in GL-3 levels in kidney cells compared to baseline will be confirmed.
Measured: Screening visit (Visit 0) and Visit 25 (Day 336)
Change from baseline in GL-3/lyso Gb-3 concentration in urine and blood after administration of 24 doses of Fabagal compared to active comparator
Plasma and urine GL-3 are often elevated in the plasma of patients diagnosed with Fabry disease. After administering Fabagal injection and the active control group 24 times, the change in GL-3 and lyso Gb-3 concentrations in urine and blood compared to the baseline value will be confirmed.
Measured: Screening visit (Visit 0), Visits 3, 5, 7, 9, and 11 (Days 28, 56, 84, 112, and 140), Visit 13 (Day 168), Visits 15, 17, 19, 21, and 23 (Days 196, 224, 252, 280, and 308) and Visit 25 (Day 336)
Change from baseline in renal function test values (estimated Glomerular Filtration Rate, eGFR) after administration
Evaluated at Screening visit (Visit 0, Baseline), Visit 13 (Day 168) and Visit 25 (Day 336). eGFR is an estimation of the glomerular filtration rate of the renal (how much blood the kidneys are filtering).
Measured: Screening visit (Visit 0), Visit 13 (Day 168), and Visit 25 (Day 336)
Change from baseline in pain score after administration of 24 doses of Fabagal compared to active comparator (Short Form McGill Pain Questionnaire-2)
The SF-MPQ-2 has 22 questions (6 on persistent pain, 6 on intermittent pain, 6 on neuropathic pain, and 4 on emotional pain) to be rated on an 11-point intensity scale (0 for not at all, to 10 for worst thinkable). Pain scores are presented as the mean of the ratings of all items.
Measured: Visits 1, 13, and 25 (Days 0, 168, and 336)
Change from baseline in quality of life after administration of 24 doses of Fabagal compared to active comparator (SF-36v2 questionnaire)
The SF-36v2 consists of 36 questions(8-domain) to measure functional health and happiness from the patient's perspective. The 8 domains are physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health. The domain score is the sum of the weights of questions in each item. The score distribution ranges from 0 to 100, with a low score indicating a poor quality of life.
Measured: Visits 1, 13, and 25 (Days 0, 168, and 336)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2023
- Study start14 Apr 2023
- Primary completionJun 2025 (estimated)
- Study completionDec 2025 (estimated)
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (estimated)
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
- NCT06081062
- Sponsor's protocol ID
- ISU303-003
- Status last verified
- Jul 2024
- First submitted
- 8 Aug 2023
- Last updated
- 29 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double-blinded, Active-controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT06081062
- Acronym / sponsor ID
- ISU303-003
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- ISU Abxis Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 14 Apr 2023 (actual)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 12 Oct 2023 (actual)
- 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.
Evaluate the safety and efficacy of Fabagal® developed by ISU ABXIS Co., Ltd., which has similar efficacy to active comparator (Agalsidase beta).
Detailed description
The primary objective is to evaluate the efficacy of Fabagal compared to active comparator (Agalsidase beta). The secondary objectives will evaluate the safety, pharmacokinetics, and immunogenicity of Fabagal compared to active comparator (Agalsidase beta).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fabagal® (Agalsidase beta) | Experimental | Biological: Fabagal® (Agalsidase beta)1 mg/kg, administered every 2 weeks for 12 months |
| Active Comparator (Agalsidase beta) | Active comparator | Drug: Active comparator (Agalsidase beta)1 mg/kg, administered every 2 weeks for 12 months |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Those who have been diagnosed with Fabry disease by genetic and alpha-galactosidase A enzyme tests and grouped by sex are as follows:
- Age: Those who are aged 8 years or older
- Those who have at least one of the following symptoms and signs:
- Patients who have not previously received enzyme replacement therapy (ERT) or Chaperone therapy for treatment of Fabry disease
- Patients who voluntarily consented and signed the informed consent form
Key exclusion criteria (7 listed)
- Patients who participated in other studies in which investigational products are administered within 30 days prior to the screening visit
- Patients with chronic kidney disease stage 4 to 5 (CKD 4-5; see Section 16.1)
- Patients who are currently on dialysis or have a history of kidney transplantation, or patients scheduled for dialysis at the time of screening, or waitlisted for kidney transplantation
- Patients who have started angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) treatment within 4 weeks prior to the screening visit or whose dose has been changed
- Patients who are pregnant, breastfeeding, or planning to become pregnant or breastfeed during the clinical study
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 |
|---|---|---|
| Fabagal® (Agalsidase beta)1 mg/kg every 2 weeks for 12 months | Biological | Fabagal® (Agalsidase beta) |
| Active comparator (Agalsidase beta)1 mg/kg every 2 weeks for 12 months | Drug | Active Comparator (Agalsidase beta) |
Primary outcome
Evaluate the efficacy of Fabagal compared with active comparator (Agalsidase beta)
Time frame: Screening visit (Visit 0) and Visit 25 (Day 336)
The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (Agalsidase beta) in patients with Fabry disease The proportion of patients achieving a GL-3 score (score 0) in renal cells after treatment with Fabagal or Active comparator (agalsidase beta) in patients with Fabry disease The primary endpoint is a comparison of the proportion of subjects in each group who achieve a score of Zero on the renal capillary endothelium histology. The score is graded on a scale from 0-3 (normal, mild, moderate, and severe). * 0: signified no visible inclusions * 1: signified multiple discrete lipid granules * 2: signified single or multiple aggregates of lipid granules * 3: signified aggregates of lipid granules either large enough or numerous enough to cause clear distortion of the luminal surface
5 secondary outcomes
Change from baseline in GL-3 levels in renal cells after administration of 24 doses of Fabagal compared to active comparator
Time frame: Screening visit (Visit 0) and Visit 25 (Day 336)
Change from baseline in GL-3/lyso Gb-3 concentration in urine and blood after administration of 24 doses of Fabagal compared to active comparator
Time frame: Screening visit (Visit 0), Visits 3, 5, 7, 9, and 11 (Days 28, 56, 84, 112, and 140), Visit 13 (Day 168), Visits 15, 17, 19, 21, and 23 (Days 196, 224, 252, 280, and 308) and Visit 25 (Day 336)
Change from baseline in renal function test values (estimated Glomerular Filtration Rate, eGFR) after administration
Time frame: Screening visit (Visit 0), Visit 13 (Day 168), and Visit 25 (Day 336)
Change from baseline in pain score after administration of 24 doses of Fabagal compared to active comparator (Short Form McGill Pain Questionnaire-2)
Time frame: Visits 1, 13, and 25 (Days 0, 168, and 336)
Change from baseline in quality of life after administration of 24 doses of Fabagal compared to active comparator (SF-36v2 questionnaire)
Time frame: Visits 1, 13, and 25 (Days 0, 168, and 336)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2024.
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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.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fabagal® (Agalsidase beta) and Active comparator (Agalsidase beta) in people with Fabry Disease, run by ISU Abxis Co., Ltd. at 3 sites in 2 countries.
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 16 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 29 Jul 2024. 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 Philippines, 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Beomhee Lee (1688-7575, webmaster@amc.seoul.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the efficacy of Fabagal compared with active comparator (Agalsidase beta) (time frame: Screening visit (Visit 0) and Visit 25 (Day 336)). 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 29 Jul 2024.