Mucopolysaccharidosis IIPhase 1NCT05422482
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
A Phase 1 study testing GC1123 in people with Mucopolysaccharidosis II and Hunter Syndrome, run by GC Biopharma Corp at 3 sites in South Korea.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug Read more about this study
- Who is it for?
- Ages 18 months to 18 years
- Conditions the study is for
- Mucopolysaccharidosis II, Hunter Syndrome
- What is being tested?
- GC1123
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 3 sitesSouth Korea
- Study dates
- 20 Sep 2022to Jun 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 18 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient who has been diagnosed with severe MPS Ⅱ (Hunter syndrome)
- Patient, aged 1.5 years (18 months) to 18 years at the time of the screening
- Patient who has received and tolerated a minimum of 12 weeks of treatment with weekly intravenous treatment, and who has received 80% of the total planned infusions within that time frame.
- Patient who is capable of undergoing neurosurgery, which has been confirmed by neurosurgeons and anesthesiologist.
- Patient eligible to execute patient evaluation activities during the clinical trial period, as assessed by the investigator
- Patient whose parents or legal representative are willing to participate in this clinical trial and provide written informed consent form
Who cannot join
- Patient who has been administered with intrathecal Idursulfase in the past
- Patient with a history of bone marrow transplantation or cord blood transplant
- Patient with a history of ventriculoperitoneal shunt or other intracranial surgeries
- Patient with end-stage multiple organ dysfunction syndrome or other severe diseases
- Patient who is exposed to malignant neoplasm
- Patient who has received treatment with any investigational drug or device within 30 days prior to study entry
Show all 9Show fewer
- Patient who have experience of hypersensitivity or anaphylaxis to ingredients of the investigational product at the time of screening
- Patient with a history of bronchotomy/tracheostomy, or patient with acute respiratory disease at the time of screening
- Patient who is ineligible to participate in the clinical trial due to laboratory test results or other reasons, as determined by the investigator
Trial locations and contacts
3 sites in 1 country.
South Korea3 sites
Pusan National University Yangsan Hospital
Pusan, 50612
Seoul National University
Seoul, 03080
Samsung Medical Center
Seoul, 06351
About this study
Official title: An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug
Read the detailed description
This study is designed as prospective, open-label, phase I and extension study. Safety, tolerability, pharmacokinetic, and pharmacodynamic properties of repeat-dose treatment of ICV-administered investigational product will be studied in patients undergoing standard treatments.
Patients will undergo cerebrospinal fluid (CSF) reservoir device implantation surgery on their scalps, and the reservoirs will be used to administer GC1123 to the cerebral ventricles monthly (every 28 days). The planned administering dose is 30 mg. After the 2nd dose on the 6th patient, Data and Safety Monitoring Boards (DSMB) will evaluate the safety and tolerability data of GC1123. The planned duration of the sutdy is total about 2 years (phase I and extension)
- Conditions
- Mucopolysaccharidosis II, Hunter Syndrome
- Being tested
- GC1123
- Medical subject headings
- Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC1123Biological
ICV-administered Hunterase, Idursulfase-ß
| Study group | Role | What they receive |
|---|---|---|
| GC1123 30mg | Experimental | 30 mg of IP will be administered every 28 days for all enrolled patients Receives: Biological: GC1123 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and frequency of serious adverse events (SAEs)
Incidence and frequency of serious adverse events (SAEs) after administration of ICV-Hunterase (GC1123)
Measured: Every 28 days from Week 1 through study completion (about 110 weeks)
Frequency and characteristics (severity, outcome, etc.) of adverse events
Frequency and characteristics (severity, outcome, etc.) of adverse events after administration of ICV-Hunterase (GC1123)
Measured: Every 28 days from Week 1 through study completion (about 110 weeks)
Presence of clinically significant abnormal echocardiography results
Presence of clinically significant abnormal echocardiography results after administration of ICV-Hunterase (GC1123); phase I only
Measured: Week 1 to Phase I study completion (about 26 weeks)
Secondary outcomes (12)
Pharmacokinetic (PK) parameters - Cmax
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Tmax
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - AUClast
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - AUCinf
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - t1/2
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - CL/F (or CL)
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Vd/F (or Vd)
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Bioavailability (F)
Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF
Measured: Week 2 to Week 22
Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in CSF
Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123)
Measured: Every 28 days from Week 1 through study completion (about 110 weeks)
Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in serum
Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123)
Measured: Every 28 days from Week 1 through study completion (about 110 weeks)
Pharmacodynamic (PD) parameters - Urine Glycosaminoglycan (GAG)
Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123)
Measured: Every 28 days from Week 1 through study completion (about 110 weeks)
Presence of anti-drug antibodies (ADAs)
Presence of anti-drug antibodies (ADAs) in CSF and serum, and neutralizing antibodies of ICV-Hunterase (GC1123)
Measured: Approximately every 6 months (Week 2 [baseline], Week 18, Week 26, Week 54, Week 82, Week 110)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2022
- Study start20 Sep 2022
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IIStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- GC Biopharma Corp
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05422482
- Sponsor's protocol ID
- GC1123_MPS2_P0101
- Status last verified
- Oct 2024
- First submitted
- 31 May 2022
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug
- ClinicalTrials.gov ID
- NCT05422482
- Acronym / sponsor ID
- GC1123_MPS2_P0101
- Conditions
- Mucopolysaccharidosis II, Hunter Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- GC Biopharma CorpIndustry (pharma, biotech, devices)
- Study start
- 20 Sep 2022 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 16 Jun 2022 (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.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug
Detailed description
This study is designed as prospective, open-label, phase I and extension study. Safety, tolerability, pharmacokinetic, and pharmacodynamic properties of repeat-dose treatment of ICV-administered investigational product will be studied in patients undergoing standard treatments.
Patients will undergo cerebrospinal fluid (CSF) reservoir device implantation surgery on their scalps, and the reservoirs will be used to administer GC1123 to the cerebral ventricles monthly (every 28 days). The planned administering dose is 30 mg. After the 2nd dose on the 6th patient, Data and Safety Monitoring Boards (DSMB) will evaluate the safety and tolerability data of GC1123. The planned duration of the sutdy is total about 2 years (phase I and extension)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GC1123 30mg | Experimental | Biological: GC112330 mg of IP will be administered every 28 days for all enrolled patients |
Who may qualify
- Ages
- 18 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Patient who has been diagnosed with severe MPS Ⅱ (Hunter syndrome)
- Patient, aged 1.5 years (18 months) to 18 years at the time of the screening
- Patient who has received and tolerated a minimum of 12 weeks of treatment with weekly intravenous treatment, and who has received 80% of the total planned infusions within that time frame.
- Patient who is capable of undergoing neurosurgery, which has been confirmed by neurosurgeons and anesthesiologist.
- Patient eligible to execute patient evaluation activities during the clinical trial period, as assessed by the investigator
Key exclusion criteria (9 listed)
- Patient who has been administered with intrathecal Idursulfase in the past
- Patient with a history of bone marrow transplantation or cord blood transplant
- Patient with a history of ventriculoperitoneal shunt or other intracranial surgeries
- Patient with end-stage multiple organ dysfunction syndrome or other severe diseases
- Patient who is exposed to malignant neoplasm
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 |
|---|---|---|
| GC1123ICV-administered Hunterase, Idursulfase-ß | Biological | GC1123 30mg |
Primary outcome
Incidence and frequency of serious adverse events (SAEs)
Time frame: Every 28 days from Week 1 through study completion (about 110 weeks)
Incidence and frequency of serious adverse events (SAEs) after administration of ICV-Hunterase (GC1123)
Frequency and characteristics (severity, outcome, etc.) of adverse events
Time frame: Every 28 days from Week 1 through study completion (about 110 weeks)
Frequency and characteristics (severity, outcome, etc.) of adverse events after administration of ICV-Hunterase (GC1123)
Presence of clinically significant abnormal echocardiography results
Time frame: Week 1 to Phase I study completion (about 26 weeks)
Presence of clinically significant abnormal echocardiography results after administration of ICV-Hunterase (GC1123); phase I only
12 secondary outcomes
Pharmacokinetic (PK) parameters - Cmax
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Tmax
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - AUClast
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - AUCinf
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - t1/2
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - CL/F (or CL)
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Vd/F (or Vd)
Time frame: Week 2 to Week 22
Pharmacokinetic (PK) parameters - Bioavailability (F)
Time frame: Week 2 to Week 22
Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in CSF
Time frame: Every 28 days from Week 1 through study completion (about 110 weeks)
Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in serum
Time frame: Every 28 days from Week 1 through study completion (about 110 weeks)
Pharmacodynamic (PD) parameters - Urine Glycosaminoglycan (GAG)
Time frame: Every 28 days from Week 1 through study completion (about 110 weeks)
Presence of anti-drug antibodies (ADAs)
Time frame: Approximately every 6 months (Week 2 [baseline], Week 18, Week 26, Week 54, Week 82, Week 110)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- 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 GC1123 in people with Mucopolysaccharidosis II and Hunter Syndrome, run by GC Biopharma Corp 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 9 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: under way, not looking for participants, as of the sponsor's last update on 15 Oct 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 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?
This study is still under way. Results are not available in the source record yet.
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 and frequency of serious adverse events (SAEs) (time frame: Every 28 days from Week 1 through study completion (about 110 weeks)). The study lists 3 primary outcomes in all. 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 15 Oct 2024.