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Gene Therapy for AADC Deficiency (AADC Trial)
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.
AADC Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowAADC Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.AADC Trial Design
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Who is running the clinical trial?
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- I am at least 24 months old.My brain MRI is clear for stereotactic surgery.I have a blood clotting disorder or need continuous blood thinner treatment.Standard treatments haven't improved my eye movement issues or developmental delays.I couldn't walk on my own without help by 18 months old.My skull is fully formed for a specific head frame placement.I have not taken any experimental drugs in the last 60 days.I do not have major health issues that would make surgery or anesthesia very risky.I have been diagnosed with AADC deficiency based on specific test results.I have had precise brain surgery before.I cannot be sedated for surgery or certain imaging tests.I do not have significant brain abnormalities that would increase risk in a study.I show signs of an active adenovirus or herpes virus infection.
- Group 1: Single treatment arm
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
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."
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."
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."
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"
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."
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."
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."
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