Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Phase 1Active Not RecruitingNCT06567769

GC Biopharma Corp9 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Sanfilippo Syndrome Type A

Interventions

GC1130ADRUG

ICV injection

Eligibility

Sex: ALLMin age: 12 MonthsMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Participants with documented MPS IIIA diagnosis * Participants aged ≥ 12 months and ≤ 18 years 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

Locations (4)

UCSF Benioff Children's Hospital

Oakland, California, United States

University of Minnesota

Minneapolis, Minnesota, United States

Ajou University Medical Center

Suwon, Gyeongi-do, South Korea

Samsung Medical Center

Seoul, South Korea

Outcomes

Primary Outcomes

Incidences and characteristics of adverse events

Time frame: up to 108 weeks

Secondary Outcomes

Maximum concentration in cerebrospinal fluid (CSF)

GC1130A PK parameters

Time frame: up to 104 weeks

Area under the concentration-time curve in CSF

GC1130A PK parameters

Time frame: up to 104 weeks

Maximum concentration in serum

GC1130A PK parameters

Time frame: up to 104 weeks

Area under the concentration-time curve in serum

GC1130A PK parameters

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

Data from ClinicalTrials.gov

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