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Gene Therapy Trial Report

Summary

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)


NCTID NCT04998396 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Canavan Disease
Disease Ontology Term DOID:3613
Compound Name BBP-812
Compound Description AAV9-ASPA
Sponsor Aspa Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 26 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant ASPA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed dose escalation, 2 levels
Dose 2 Undisclosed expansion dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-08-05
Completion Date 2030-10-08
Last Update 2024-10-22

Participation Criteria


Eligible Age <=30 Months
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: * Maximum age for inclusion is 30 months. * Participant has stable health in the opinion of the investigator and as confirmed by medical history and laboratory studies with no acute or chronic hematologic, renal, liver, immunologic, or neurologic disease (other than Canavan disease). * Participant has biochemical, genetic, and clinical diagnosis of Canavan disease: * Elevated urinary NAA and * Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history. * Active clinical signs of Canavan disease * Participant is up to date on all immunizations per local guidelines Key Exclusion Criteria: * Tests positive for total anti-AAV9 antibodies determined by enzyme-linked immunosorbent assay (ELISA). * Received prior gene therapy or other therapy (including vaccines) involving AAV. * Participant is receiving high-dose therapy with immunosuppressants. * Participant has significantly progressed Canavan disease characterized as: * Presence of continuous/constant decerebrate or decorticate posturing, * Recurrent status epilepticus, or * Recalcitrant seizures that do not respond while on 3 or more anti-epileptic medications
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates Program terminated, effective Feb 2025

Resources/Links