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GC1130A

Phase 1Small molecule

GGC Biopharma Corp

1

Indications

1

Total Trials

1

Active Trials

0

Competitors

Pipeline

Status by Indication[3][1]

Indication

Sanfilippo Syndrome Type aActive, not recruiting

Sanfilippo Syndrome Type aPhase 1

Active, not recruiting

Timeline

Expected Milestones

Q2 2027

Phase 1Active, not recruiting

Sanfilippo Syndrome Type a

NCT06567769

Clinical

Trials by Indication[1]

Sanfilippo Syndrome Type A1 trial

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)

Active, not recruiting·GGC Biopharma Corp·Phase 1·NCT06567769·9 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for GC1130A. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — GC1130A compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for GC1130A. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — GC Biopharma Corp company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 1[1]
Company: GGC Biopharma Corp

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