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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04998396
Other study ID # CVN-102
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date September 8, 2021
Est. completion date October 8, 2030

Study information

Verified date June 2024
Source Aspa Therapeutics
Contact Michelle Nelken
Phone 833-764-2267 or 617-861-4617
Email CANaspire@aspatx.com
Is FDA regulated No
Health authority
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 26
Est. completion date October 8, 2030
Est. primary completion date October 13, 2026
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

Study Design


Related Conditions & MeSH terms


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
See also
  Status Clinical Trial Phase
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Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT04126005 - Natural History Study of Patients With Canavan Disease
Completed NCT02851563 - A Natural History Study of Canavan Disease
Recruiting NCT00724802 - Oral Glyceryl Triacetate (GTA) in Newborns With Canavan N/A
Completed NCT01999257 - Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. N/A
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project
Recruiting NCT04833907 - rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease Phase 1/Phase 2
No longer available NCT05317780 - Canavan-Single Patient IND