Sanfilippo Syndrome Type APhase 1NCT06567769
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A Phase 1 study testing GC1130A in people with Sanfilippo Syndrome Type A, run by GC Biopharma Corp at 4 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA). Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Sanfilippo Syndrome Type A
- What is being tested?
- GC1130A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 21 Nov 2024to 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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
Who cannot join
- Participants with significant non-MPS IIIA related central nervous system impairment
- Participants with previous complication from intraventricular drug administration
- Participants with contraindications for MRI scans and for neurosurgery
- Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
- Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
United States2 sites
University of Minnesota
Minneapolis, Minnesota, 55455
UCSF Benioff Children's Hospital
Oakland, California, 94609
South Korea2 sites
Samsung Medical Center
Seoul
Ajou University Medical Center
Suwon, Gyeongi-do
About this study
Official title: A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
- Conditions
- Sanfilippo Syndrome Type A
- Being tested
- GC1130A
- Keywords
- Mucopolysaccharidosis Type III Subtype A, MPS IIIA
- Medical subject headings
- Mucopolysaccharidosis III
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC1130ADrug
ICV injection
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Not described Receives: Drug: GC1130A |
| Cohort 2 | Experimental | Not described Receives: Drug: GC1130A |
| Cohort 3 | Experimental | Not described Receives: Drug: GC1130A |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidences and characteristics of adverse events
Measured: up to 108 weeks
Secondary outcomes (9)
Maximum concentration in cerebrospinal fluid (CSF)
GC1130A PK parameters
Measured: up to 104 weeks
Area under the concentration-time curve in CSF
GC1130A PK parameters
Measured: up to 104 weeks
Maximum concentration in serum
GC1130A PK parameters
Measured: up to 104 weeks
Area under the concentration-time curve in serum
GC1130A PK parameters
Measured: up to 104 weeks
Change from baseline in CSF heparan sulfate concentration
Measured: up to 104 weeks
Change from baseline in serum heparan sulfate concentration
Measured: up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF
Measured: up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in serum
Measured: up to 104 weeks
Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)
Measured: up to 104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2024
- Study start21 Nov 2024
- 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 Sanfilippo Syndrome Type AStudies of the same condition, open and under way.
- More trials run by GC Biopharma CorpThe sponsor's other registered studies.
- 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 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
- NCT06567769
- Sponsor's protocol ID
- GC1130_MPS3A_P0101
- Status last verified
- Sep 2026
- First submitted
- 20 Aug 2024
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
- ClinicalTrials.gov ID
- NCT06567769
- Acronym / sponsor ID
- GC1130_MPS3A_P0101
- Conditions
- Sanfilippo Syndrome Type A
- Keywords
- Mucopolysaccharidosis Type III Subtype A, MPS IIIA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- GC Biopharma CorpIndustry (pharma, biotech, devices)
- Study start
- 21 Nov 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 23 Aug 2024 (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, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: GC1130A |
| Cohort 2 | Experimental | Drug: GC1130A |
| Cohort 3 | Experimental | Drug: GC1130A |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
Key exclusion criteria (5 listed)
- Participants with significant non-MPS IIIA related central nervous system impairment
- Participants with previous complication from intraventricular drug administration
- Participants with contraindications for MRI scans and for neurosurgery
- Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
- Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
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 |
|---|---|---|
| GC1130AICV injection | Drug | Cohort 1, Cohort 2, Cohort 3 |
Primary outcome
Incidences and characteristics of adverse events
Time frame: up to 108 weeks
9 secondary outcomes
Maximum concentration in cerebrospinal fluid (CSF)
Time frame: up to 104 weeks
Area under the concentration-time curve in CSF
Time frame: up to 104 weeks
Maximum concentration in serum
Time frame: up to 104 weeks
Area under the concentration-time curve in serum
Time frame: up to 104 weeks
Change from baseline in CSF heparan sulfate concentration
Time frame: up to 104 weeks
Change from baseline in serum heparan sulfate concentration
Time frame: up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF
Time frame: up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in serum
Time frame: up to 104 weeks
Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)
Time frame: up to 104 weeks
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 8 Sep 2026.
- Individual participant dataSharing planned: No
Related trials for Sanfilippo Syndrome Type A
RecruitingPhase 1/2
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Mucopolysaccharidosis Type IIIA
Not yet recruitingObservational study
MPS IIIA Disease Monitoring Program
MPS IIIA, Sanfilippo Syndrome
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
Not yet recruitingPhase 3
Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)
MPS IIIB
Not yet recruitingPhase 1/2
Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB
Sanfilippo Syndrome (MPS III)
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.
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GC1130A in people with Sanfilippo Syndrome Type A, run by GC Biopharma Corp at 4 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 2 inclusion and 5 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 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States, 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?
Incidences and characteristics of adverse events (time frame: up to 108 weeks). 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 8 Sep 2026.