San Francisco, CANCT02852213Now EnrollingIRB Ready

AADC Deficiency Clinical Trial in San Francisco, CA

Access cutting-edge aadc deficiency treatment through this phase 1 clinical trial at a research site in San Francisco. Study-provided care at no cost to qualified participants.

Sponsored by Krzysztof Bankiewicz

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Expert Care in San Francisco

Access aadc deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related aadc deficiency treatment provided free

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Check if you qualify for this aadc deficiency clinical trial in San Francisco, CA

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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 San Francisco

    Convenient for CA 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 San Francisco site if eligible
  4. 4Begin participation

About This AADC Deficiency Study in San Francisco

The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.

Sponsor: Krzysztof Bankiewicz

Who Can Participate

Inclusion Criteria

Definite diagnosis of AADC deficiency, confirmed by at least two of the following three criteria: (1) CSF neurotransmitter profile demonstrating reduced HVA and 5-HIAA, and elevated 3-OMD concentrations; (2) Plasma AADC activity less than or equal to 5 pmol/min/mL; (3) Molecular genetic confirmation of homozygous or compound heterozygous mutations in DDC.
Age 24 months and older.
Failed to derive adequate benefit from standard medical therapy (dopamine agonists, monoamine oxidase inhibitor, pyridoxine or related form of Vitamin B6), as judged by presence of residual oculugyric crises and developmental delay.
Documented history of motor developmental delay, with inability to walk independently without support by age 18 months.
Cranium sufficiently developed, with sutures closed, to enable surgical placement of SmartFrame® system on the head for MRI-guided stereotactic targeting.
Brain MRI does not show any conditions or malformations that are clinically significant with respect to risks for stereotactic brain surgery.
Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including the need for frequent and prolonged follow-up.
Both parents (or legal guardians) must give their consent for their child's participation in the study parents unless (i.) one parent is deceased, unknown or incompetent; (ii.) one parent is not reasonably available; or (iii.) one parent has responsibility for the care and custody of the child (if consistent with state law).
Baseline hematology, chemistry, and coagulation values within the normal pediatric laboratory value ranges, unless in the Investigator's judgment, the out-of-range values are not clinically significant with respect to subject's suitability for surgery.

Exclusion Criteria

Intracranial neoplasm or any structural brain abnormality or lesion (e.g., severe brain atrophy, white matter degenerative changes), which, in the opinion of the study investigators, would confer excessive risk and/or inadequate potential for benefit.
Presence of other significant medical or neurological conditions that would create an unacceptable operative or anesthetic risk (including congenital heart disease, respiratory disease with home oxygen requirement, history of serious anesthesia complications during previous elective procedures, history of cardiorespiratory arrest), liver or renal failure, malignancy, or HIV positive.
Previous stereotactic neurosurgery.
Coagulopathy, or need for ongoing anticoagulant therapy.
Contraindication to sedation during surgery or imaging studies (SPECT, PET or MRI).
Receipt of any investigational agent within 60 days prior to Baseline and during study participation.
Evidence of clinically active infection with adenovirus or herpes virus on physical examination.

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 San Francisco?

Yes, this clinical trial (NCT02852213) has an active research site in San Francisco, CA 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.

AADC Deficiency Treatment Options in San Francisco, CA

If you're searching for aadc deficiency treatment options in San Francisco, CA, this clinical trial (NCT02852213) 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 San Francisco research site is actively enrolling participants for this phase 1 clinical trial. You'll receive care from experienced aadc deficiency 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 aadc deficiency clinical trials near you to find additional studies recruiting in your area.

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