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New treatment option for Canavan Disease
Official title A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
ClinicalTrials.gov ID: NCT04998396
What this study is testing
What is AAV9 BBP-812?
AAV9 BBP-812 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for canavan disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 30
You may be able to join if
- 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...
- 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.
You likely can't join if
- 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
See the full eligibility 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...
- 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 Key
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 30 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States; New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.