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Gene Therapy Trial Report

Summary

A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease


NCTID NCT05541627 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Huntington's Disease
Disease Ontology Term DOID:12858
Compound Name AB-1001
Compound Alias BV-101
Compound Description AAVrh10.CAG.hCYP46A1
Sponsor Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 5 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CYP46A1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAVrh10
Editor Type none
Dose 1 4E8 vg/uL
Dose 2 1.1E9 vg/uL

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-09-13
Completion Date 2028-04
Last Update 2025-09-18

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Male or Female subjects between ages 18 and 65 years (both inclusive) at time of consenting, able to provide Informed Consent and able to understand and comply with all study procedures. * Documented genetic confirmation of pathological CAG expansion in the huntingtin gene ≥40. * Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression. * Striatal MRI volumes per hemisphere: Putamen ≥ 2.3 cm3 (per side); Caudate ≥ 1.7 cm3 (per side) on Screening MRI. * All HD concomitant medications stable for at least 30 days prior to screening at the investigator's discretion. Key Exclusion Criteria: * Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, would impact subject's safety and compliance with the study procedures. * Metastatic neoplasms within the five years prior to screening. * Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator's clinical judgment. * Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization. * History of prior suicide attempt or imminent risk of self-harm based on investigator's judgment or with a "yes" answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS). * Patients with history of confirmed stroke, known intracranial neoplasms, vascular malformations, or intracranial hemorrhage. * Subjects not deemed suitable for the surgical procedure as per the Neurosurgeon's judgment. * Any history of gene therapy, cell transplantation or any other experimental brain surgery. * Any RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides within 6 months prior to screening. * Subjects unable to tolerate or unwilling to undergo multiple lumbar punctures. * Participation in any clinical trial of an approved or non-approved investigational drug or intervention within 12 weeks or 5 half-lives whichever is longer prior to treatment.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
FDA Designations
Recent Updates Bayer announced the discontinuation of this program November 2024

Resources/Links