Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06615206
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-04
Brief Title:
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
Sponsor:
None
Organization:
None
Study Overview
Official Title:
An Open-label Multiple-dose Clinical Study to Evaluating the Safety Tolerability and Preliminary Efficacy of a Single Intracerebroventricular Injection of HG204 for the Treatment of MECP2 Duplication Syndrome
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HERO
Brief Summary:
Methyl-CpG binding protein 2 MECP2 is a dosage-sensitive X-linked gene critical for central nervous system development and functional maintenance which gain-of-function causes MECP2 duplication syndrome MDS Affecting primarily in males this disorder is characterized by severe intellectual disability motor dysfunction infantile hypotonia epilepsy respiratory tract infections and premature death before 25 years of age with no curative therapy
HG204 is a CRISPR RNA-editing therapy packaging novel high-fidelity Cas13Y hfCas13Y technology using one single adeno-associated virus AAV vector to target and knock down MECP2 mRNA in the brain Preclinical studies showed that a single intracerebroventricular injection of HG204 persistently decreased MECP2 mRNA and MECP2 protein in the cortex of the MDS mice reversed the abnormal motor and social phenotypes and significantly prolonged survival in MDS mouse models
HG204 is a CRISPR RNA-editing therapy packaging novel high-fidelity Cas13Y hfCas13Y technology using one single adeno-associated virus AAV vector to target and knock down MECP2 mRNA in the brain Preclinical studies showed that a single intracerebroventricular injection of HG204 persistently decreased MECP2 mRNA and MECP2 protein in the cortex of the MDS mice reversed the abnormal motor and social phenotypes and significantly prolonged survival in MDS mouse models
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None