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Recruiting NCT04998396

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

Phase I / Phase II Interventional Canavan Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AAV9 BBP-812.
Who it may be relevant to
Registry conditions: Canavan Disease. Basic parameters: up to 30 months · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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

Overview

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.

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.

Interventions

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

Primary outcome measures

  • Number of Participants with Adverse Events (AEs) [Time frame: Baseline up to Week 52]
  • Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels [Time frame: Baseline, Month 12]
  • Change from Baseline to 12 Months Post-Infusion in Central Nervous System (CNS) NAA, as Measured by Magnetic Resonance Spectroscopy (MRS) [Time frame: Baseline, Month 12]
Secondary outcome measures (5)
  • Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88 [Time frame: Baseline, Week 52]
  • Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4 [Time frame: Baseline, Week 52]
  • Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4 [Time frame: Baseline, Week 52]
  • Change from Baseline to Week 52 in Communication Assessment, Bayley-4 [Time frame: Baseline, Week 52]
  • Change from Baseline to Week 52 in Adaptive Function, Vineland-3 [Time frame: Baseline, Week 52]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 4 centers
  • UCSF Benioff Children's Hospital Oakland — Oakland
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) — Boston
  • Weill Cornell Medicine; Division of Pediatric Neurology — New York

Identifiers

NCT: NCT04998396 · CVN-102

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗