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Virus Therapy

Gene Therapy for AADC Deficiency (AADC Trial)

Phase 1
Recruiting
Led By Nalin Gupta, MD, PhD
Research Sponsored by Krystof Bankiewicz
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 24 months and older
Brain MRI does not show any conditions or malformations that are clinically significant with respect to risks for stereotactic brain surgery
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

AADC Trial Summary

This trial is testing a gene therapy for children with AADC deficiency, a disease that affects the brain's ability to produce certain neurotransmitters. The therapy involves injecting a virus carrying the healthy gene into the brain.

Who is the study for?
This trial is for children aged 4 and older with AADC deficiency, a rare metabolic disorder. They must have a stable medication regimen, confirmed diagnosis through specific tests, be unable to walk independently, and have no recent changes in medications. Children who haven't benefited from standard treatments may qualify. Those with certain medical conditions or previous brain surgery are excluded.Check my eligibility
What is being tested?
The study is testing the safety and effectiveness of delivering the gene therapy drug AAV2-hAADC directly into parts of the brain responsible for movement control in kids with AADC deficiency. The goal is to see if this treatment can improve symptoms related to this genetic condition.See study design
What are the potential side effects?
While not explicitly listed here, potential side effects could include reactions at the injection site in the brain, immune responses to the viral vector used for gene delivery (AAV2), and any complications arising from neurosurgical procedures.

AADC Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 24 months old.
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My brain MRI is clear for stereotactic surgery.
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I couldn't walk on my own without help by 18 months old.
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My skull is fully formed for a specific head frame placement.
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I have been diagnosed with AADC deficiency based on specific test results.

AADC Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Operative Surgical Procedures
CSF neurotransmitter metabolite concentrations
Secondary outcome measures
Fluorodopa PET scan
Gross Motor Function Measure
Symptom Diary created by PI

AADC Trial Design

1Treatment groups
Experimental Treatment
Group I: Single treatment armExperimental Treatment1 Intervention
Single-stage dose-escalation, open-label safety study of AAV2-hAADC delivered by image-guided convection-enhanced delivery bilaterally into the substantia nigra pars compacta and the ventral tegmental area of pediatric patients with AADC deficiency. Primary aim is to determine the dose for future studies based on safety, biomarkers of pharmacological activity of AADC and clinical outcomes. Cohort 1 (3 subjects) will receive a single low dose of AAV2 hAADC. The total AAV2-hAADC dose will be infused via MR guided infusion into 4 sites in both the left and right SNc and VTA. Dose intervals will be 90 days between the first 3 subjects. Cohort 2 dose (4 subjects) will be determined by Cohort 1 results. Following Cohort 2, Cohort 3/4 will be dose and divided divided by age. Cohorts 3/4 will receive the same dose by MR guided infusion to 1-2 sites bilaterally in-between the SNc and VTA. Cohort 5 (24-47mo old) will have same vector concentration and lower volume of infusion than Cohorts 3/4
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AAV2-hAADC
2016
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Krystof BankiewiczLead Sponsor
Krzysztof BankiewiczLead Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,337 Previous Clinical Trials
648,802 Total Patients Enrolled

Media Library

AAV2-hAADC (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02852213 — Phase 1
Aromatic L-amino Acid Decarboxylase Deficiency Research Study Groups: Single treatment arm
Aromatic L-amino Acid Decarboxylase Deficiency Clinical Trial 2023: AAV2-hAADC Highlights & Side Effects. Trial Name: NCT02852213 — Phase 1
AAV2-hAADC (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02852213 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants are currently being monitored in this clinical investigation?

"Affirmative, the information available on clinicaltrials.gov points to this trial's current recruitment status. It was originally published in July of 2016 and updated most recently in September of 2022. 31 patients are needed between two separate medical facilities."

Answered by AI

Has there been any previous experimentation with AAV2-hAADC?

"Presently, there are 6 protocols for AAV2-hAADC in operation. Of these, 1 is currently at Phase 3 of development. While the main trials are held in Columbus, Ohio, 23 clinical centres across the country offer this medication."

Answered by AI

Is this trial currently recruiting participants?

"Affirmative, according to the online records on clinicaltrials.gov this medical study is still recruiting patients, with 31 needed from 2 centers. The trial was initially posted July 1st 2016 and recently modified on September 21st 2022."

Answered by AI

What criteria must be met for individuals to qualify as participants in this research project?

"This investigation is enrolling 31 minors suffering from AADC deficiency. Notably, the candidates must be between 4 and 18 years old as well as satisfy numerous other criteria such as having a maturing cranium with closed sutures to permit surgical placement of SmartFrame® system on their skull for MRI-guided stereotactic targeting. Moreover, they need to have taken stable medications for treatment of this condition without any alterations in dose or new drugs being introduced 6 months prior to baseline; it is also essential that hematology, chemistry and coagulation values are within normal pediatric laboratory value ranges. In addition, parent(s) or"

Answered by AI

Does this research accept participants of a younger age bracket?

"Per the entry requirements, individuals aged 4 to 18 are qualified for enrollment in this trial. Additionally, there are 9 trials dedicated exclusively to children and 8 specifically focused on elderly patients."

Answered by AI

Has AAV2-hAADC been given the go-ahead from the Food and Drug Administration?

"As this is an early-stage trial, data supporting AAV2-hAADC's safety and efficacy is sparse. Therefore, it was assigned a rating of 1 on our risk assessment scale."

Answered by AI

What ailments are usually treated with AAV2-hAADC?

"Patients with parenteral nutrition-associated complications, premature birth (pregnancy), and uremia can receive treatment through AAV2-hAADC."

Answered by AI
Recent research and studies
~12 spots leftby Jul 2027