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

ConditionCanavan Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeUp to 30
SponsorAspa Therapeutics

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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).

Elevated urinary NAA and

Biallelic mutation of the ASPA gene determined at Screening or documented in the participant's medical history.

Disqualifiers

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.

Presence of continuous/constant decerebrate or decorticate posturing,

Trial design

Design model

Sequential

Treatments tested in this trial

  • AAV9 BBP-812

    Biological/Vaccine

    Sterile solution for injection for 1-time use via volumetric infusion pump

Treatment groups

26 Participants
are divided into 3 treatment groups
Group A: Dose-Finding Phase: BBP-812 Dose Level 1 (Cohort 1)Experimental treatment 1 intervention
Group B: Dose-Finding Phase: BBP-812 Dose Level 2 (Cohort 2)Experimental treatment 1 intervention
Group C: Enrollment Expansion Phase: BBP-812Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants with Adverse Events (AEs)

Time frame
Baseline up to Week 52
2

Change from Baseline to 12 Months Post-Infusion in Urine N-acetylaspartate (NAA) Levels

Time frame
Baseline, Month 12
3

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 outcomes

1

Change from Baseline to Week 52 in Gross Motor Assessment, Gross Motor Function Measure-88

Time frame
Baseline, Week 52
2

Change from Baseline to Week 52 in Fine Motor Assessment, Bayley-4

Time frame
Baseline, Week 52
3

Change from Baseline to Week 52 in Cognitive Assessment, Bayley-4

Time frame
Baseline, Week 52
4

Change from Baseline to Week 52 in Communication Assessment, Bayley-4

Time frame
Baseline, Week 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Aspa Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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