A Study of AAV9 Gene Therapy in Participants With Canavan Disease

Canavan Disease Clinical Trial

— CANaspire  

Official title:

A Phase 1/2 Open-Label Study of the Safety and Clinical Activity of Gene Therapy for Canavan Disease Through Administration of an Adeno-Associated Virus (AAV) Serotype 9-Based Recombinant Vector Encoding the Human ASPA Gene

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04998396
• Other study ID #: CVN-102
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: September 8, 2021
• Est. completion date: March 15, 2028

Study information

• Verified date: September 2023
• Source: Aspa Therapeutics
• Contact: Michelle Nelken
• Phone: 833-764-2267 or 617-861-4617
• Email: CANaspire[@]aspatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.

Description:

Canavan disease is an ultra-rare, profoundly disabling and fatal disease with no approved therapy. The Sponsor is developing BBP-812, an investigational gene therapy product for systemic delivery in participants with Canavan disease. BBP-812 is a recombinant adeno-associated virus serotype 9 (rAAV9) vector engineered to deliver the aspartoacylase (ASPA) transgene under control of a ubiquitous promoter to restore ASPA expression in both neuronal and non-neuronal cell types.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: March 15, 2028
• Est. primary completion date: October 15, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 30 Months

Eligibility

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

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

Related Conditions & MeSH terms

• Canavan Disease

Intervention

Biological:
• AAV9 BBP-812
Sterile solution for injection for 1-time use via volumetric infusion pump

Locations

Country	Name	City	State
United States	Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Weill Cornell Medicine; Division of Pediatric Neurology	New York	New York
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Aspa Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Adverse Events (AEs)		Baseline up to Week 52
Primary	Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels		Baseline, Month 12
Primary	Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS)		Baseline, Month 12
Secondary	Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88		Baseline, Week 52
Secondary	Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4		Baseline, Week 52
Secondary	Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4		Baseline, Week 52
Secondary	Change from Baseline to Week 52 in Communication Assessment, Bayley-4		Baseline, Week 52
Secondary	Change from Baseline to Week 52 in Adaptive Function, Vineland-3		Baseline, Week 52

External Links

•   Aspa Therapeutics Company Website
•   Canavan Disease Program Website