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Purpose

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.

Condition

Eligibility

Eligible Ages
Under 30 Months
Eligible Genders
All
Accepts Healthy Volunteers
No

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

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

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1) 		Participants will receive a single intravenous (IV) infusion of low-dose BBP-812 on Day 0 in the dose-finding phase of the study.
  • Biological: AAV9 BBP-812
    Sterile solution for injection for 1-time use via volumetric infusion pump
Experimental
Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2) 		Participants will receive a single IV infusion of high-dose BBP-812 on Day 0 in the dose-finding phase of the study.
  • Biological: AAV9 BBP-812
    Sterile solution for injection for 1-time use via volumetric infusion pump
Experimental
Enrollment Expansion Phase: BBP-812 		Participants will receive a single IV infusion of BBP-812 at the selected dose from the dose-finding phase on Day 0 in expansion phase of the study.
  • Biological: AAV9 BBP-812
    Sterile solution for injection for 1-time use via volumetric infusion pump

Recruiting Locations

Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND)
Boston, Massachusetts 02114
Contact:
Riya Saxena
603-674-6743
rsaxena2@mgh.harvard.edu

More Details

Status
Recruiting
Sponsor
Aspa Therapeutics

Study Contact

Mary Rohrer
833-764-2267 or 617-861-4617
CANaspire@aspatx.com

Detailed 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.