Boston, MANCT04998396Now EnrollingIRB Ready

Canavan Disease Clinical Trial in Boston, MA

Access cutting-edge canavan disease treatment through this clinical trial at a research site in Boston. Study-provided care at no cost to qualified participants.

Sponsored by Aspa Therapeutics

Quick Self-Assessment

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Expert Care in Boston

Access canavan disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related canavan disease treatment provided free

Apply for This Boston Location

Check if you qualify for this canavan disease clinical trial in Boston, MA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to Boston

    Convenient for MA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Boston site if eligible
  4. 4Begin participation

About This Canavan Disease Study in Boston

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.

Sponsor: Aspa Therapeutics

Who Can Participate

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 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 cha

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Boston?

Yes, this clinical trial (NCT04998396) has an active research site in Boston, MA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Canavan Disease Treatment Options in Boston, MA

If you're searching for canavan disease treatment options in Boston, MA, this clinical trial (NCT04998396) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Boston research site is actively enrolling participants for this clinical trial. You'll receive care from experienced canavan disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all canavan disease clinical trials near you to find additional studies recruiting in your area.

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