Clinical trial NCT06567769: Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA)
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Sanfilippo Syndrome Type A
Keywords: Mucopolysaccharidosis Type III Subtype A
MPS IIIA
Lead sponsor: GC Biopharma Corp
Sponsor class: INDUSTRY
First posted: 2024-08-23
Enrollment: 9
Sex: ALL
Minimum age (years): 24
Maximum age (years): 72
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief 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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).


Eligibility criteria: Inclusion Criteria:

* Participants with documented MPS IIIA diagnosis
* Participants aged ≥ 24 months and ≤ 72 months

Exclusion Criteria:

* 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

Primary outcomes:
measure: Incidences and characteristics of adverse events
timeFrame: up to 108 weeks
Secondary outcomes:
measure: Maximum concentration in cerebrospinal fluid (CSF)
timeFrame: up to 104 weeks
measure: Area under the concentration-time curve in CSF
timeFrame: up to 104 weeks
measure: Maximum concentration in serum
timeFrame: up to 104 weeks
measure: Area under the concentration-time curve in serum
timeFrame: up to 104 weeks
measure: Change from baseline in CSF heparan sulfate concentration
timeFrame: up to 104 weeks
measure: Change from baseline in serum heparan sulfate concentration
timeFrame: up to 104 weeks
measure: Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF
timeFrame: up to 104 weeks
measure: Incidence of anti-drug and neutralizing antibodies of GC1130A in serum
timeFrame: up to 104 weeks
measure: Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)
timeFrame: up to 104 weeks
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital
state: California
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Setagaya
country: Japan
facility: National Center for Child Health and Development
state: Tokyo
city: Suwon-si
country: Korea, Republic of
facility: Ajou University Medical Center
state: Gyeongi-do
city: Seoul-si
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
References: