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Gene Therapy for Canavan Disease (CAN-GT Trial)
CAN-GT Trial Summary
This trial is testing a new technology targeted at the cells most affected by Canavan Disease in the safest way possible.
CAN-GT Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowCAN-GT Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.CAN-GT Trial Design
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Who is running the clinical trial?
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- You have been diagnosed with typical CD by a neurologist who is certified by the board.You are expected to live for less than 12 months for any reason.Your lab test results are not within the normal range and the doctor in charge decides they are important.Your GMFM-88 score is higher than 35%.I do not have any chronic conditions that would make surgery risky for me.You have a medical reason that makes it unsafe for you to have a MRI scan.I cannot receive treatments that weaken my immune system.My child is younger than 15 months.I have had brain surgery before.I haven't had any vaccinations in the last month.I am between 3 and 5 years old.I am between 15 and 36 months old.
- Group 1: 3.7 x 10^13 v.g. rAAV-Olig001-ASPA
- 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
Are elderly patients eligible for this research?
"This trial is for patients that are 3 to 60 months old, which falls in between the 46 studies for children and the 77 trials concerning adults over 65 years old."
What do we know about rAAV-Olig001-ASPA from other research?
"Presently, 112 different clinical trials are underway for rAAV-Olig001-ASPA. Of those active studies, 36 are in Phase 3 testing. Though many of the trials take place in Brisbane, Queensland, there are 3020 total locations running these sorts of tests."
For what reasons is rAAV-Olig001-ASPA commonly given to patients?
"RAAV-Olig001-ASPA can be used to treat a variety of conditions, such as ulcerative colitis, varicella-zoster virus acute retinal necrosis, and brain cancer."
If I enroll, am I able to complete the whole clinical trial?
"This study is for children aged 3 months to 5 years old that have canavan disease. A total of 24 participants are needed."
How many patients are being accepted into this clinical trial?
"At the moment, this clinical trial is not open for enrollment. The first posting was on April 1st 2021 with the most recent update being on June 23rd, 2022. If you are interested in participating in other studies, there are 3 trials involving canavan disease and 112 rAAV-Olig001-ASPA studies that have active participant recruitment."
Are there any vacancies in this clinical trial for new participants?
"Unfortunately, this particular clinical trial is not currently looking for new patients. Although the study was first posted on 4/1/2021 and was last edited on 6/23/2022, it has since finished recruiting participants. That said, there are 115 other studies that are still recruiting patients."
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