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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Randomized Study of SPK-10001 Gene Therapy in Participants with Huntington's Disease

NCTID NCT06826612 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Huntington's Disease
Disease Ontology Term DOID:12858
Compound Name SPK-10001
Sponsor Spark Therapeutics, Inc.
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 53
Results Posted Not Available

Therapy Information

Target Gene/Variant MiHTT
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intraparenchymal (basal ganglia)
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV
Editor Type
Dose 1 Undisclosed dose escalation
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2025-02-03
Completion Date 2035-01-12
Last Update 2025-03-20

Participation Criteria

Eligible Age 25 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 1
Locations United States

Regulatory Information

Has US IND True
FDA Designations
Recent Updates

Resources/Links

Preclinical Publications