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Clinical Trial Report

Gene Therapy Trial Report

Summary

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)


NCTID NCT06615206 (View at clinicaltrials.gov)
Description
Development Status Active
Indication MECP2 Duplication Syndrome
Disease Ontology Term DOID:0060799
Compound Name HG204
Sponsor HuidaGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant MECP2
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intracerebroventricular
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type hfCas13Y
Dose 1 Up to 2 dose cohorts, undisclosed concentration
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Na
Submit Date 2024-09-04
Completion Date 2026-10-31
Last Update 2024-11-26

Participation Criteria


Eligible Age 2 Years - 18 Years
Standard Ages Child, Adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed announced in December 2024

Resources/Links