Your session is about to expire
← Back to Search
Virus Therapy
Gene Therapy for Batten Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Able to walk independently at least 50 feet
Aged ≥ 3 to < 11 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 36 months
Awards & highlights
Study Summary
This trial is testing a new treatment for CLN3 Batten disease, a rare genetic disorder that causes progressive neurological problems. The treatment involves delivering a virus carrying a healthy gene directly into the spinal cord.
Who is the study for?
This trial is for children aged 3 to under 11 with CLN3 Batten disease, able to walk independently. They must have a specific diagnosis confirmed by an accredited lab and a low physical impairment score. Kids can't join if they've had recent immunosuppression therapy, other neurological issues, active infections, uncontrolled seizures, certain medical procedures or conditions that prevent MRI scans or lumbar punctures.Check my eligibility
What is being tested?
The trial tests two doses of AT-GTX-502 gene therapy delivered into the spinal cord region for safety and effectiveness in treating CLN3 Batten disease. It's an early-stage study where all participants receive the treatment but at different dosage levels.See study design
What are the potential side effects?
As this is an early-phase trial for gene therapy, potential side effects are not fully known but may include reactions related to intrathecal administration like headache or back pain, immune responses to the viral vector used in gene delivery, and possible inflammation.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can walk by myself for at least 50 feet.
Select...
I am between 3 and 10 years old.
Select...
I have been diagnosed with CLN3 Batten disease by a certified lab.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 36 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~36 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Efficacy: Change in rating as determined using the Unified Batten Disease Rating Scale (UBDRS) rating scale.
Safety evaluation based on the development of dose-limiting toxicity (DLT).
Secondary outcome measures
Global impression: Change in disease severity using the UBDRS clinical global impression (CGI) subscale.
QOL: Change in Quality of Life (QOL) as determined using the Pediatric Quality of Life (PedsQL™) scale.
Seizures: Change is seizure subscore as determined using Seizure subscale of the UBDRS scale.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Cohort 2: AT-GTX-502 High-DoseExperimental Treatment1 Intervention
No more than 10 mL of 1.2 x 1014 vg of AT-GTX-502 administered via intrathecal injection
Group II: Cohort 1: AT-GTX-502 Low-DoseExperimental Treatment1 Intervention
No more than 5 mL of 6 x 1013 vg AT-GTX-502 administered via intrathecal injection
Find a Location
Who is running the clinical trial?
Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,717 Total Patients Enrolled
Clinical ResearchStudy DirectorAmicus Therapeutics
9 Previous Clinical Trials
609 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I had a major seizure episode within the last 4 weeks.I have had surgery on my eye or cornea.Your blood test results show important health issues.I have undergone a stem cell or bone marrow transplant.I do not have any inherited neurological or metabolic diseases other than CLN3 Batten disease.Your physical impairment score is less than or equal to 7 on the Unified Parkinson's Disease Rating Scale (UBDRS).My anti-AAV9 antibody levels are low, as confirmed by a blood test.My family and I choose not to share my study participation with my doctor.I have an immune system disorder.I haven't had chemotherapy, radiotherapy, or immunosuppression in the last 30 days.I can walk by myself for at least 50 feet.I have not had a severe infection like pneumonia or meningitis in the last 4 weeks.I am currently using cannabinoids or their by-products.I am between 3 and 10 years old.My seizures are not well-controlled by medication.My liver tests are high, more than 3 times the normal or 1.5 times if I'm on valproic acid.I have not had a brain condition like trauma or meningitis that could affect my thinking.I have been diagnosed with CLN3 Batten disease by a certified lab.I don't have conditions like bleeding disorders that prevent spinal procedures.I have had an organ transplant.I do not have an active viral infection like HIV or hepatitis B or C.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1: AT-GTX-502 Low-Dose
- Group 2: Cohort 2: AT-GTX-502 High-Dose
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- 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
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is the current research initiative open to those aged 55 and older?
"This trial seeks to recruit children aged 3 to 10 years old. Additionally, 6 other medical studies are welcoming participants below the age of 18 while a separate study is available for those above 65."
Answered by AI
What eligibility criteria must be met by participants in this trial?
"This trial is searching for 7 children with juvenile neuronal ceroid lipofuscinosis aged between 3 and 10 years old. To qualify, participants must fulfil the following requirements: A genotype-confirmed diagnosis of CLN3 Batten disease from a CAP/CLIA laboratory (or its equivalent abroad), UBDRS physical impairment score not exceeding 7, able to walk independently at least 50 feet."
Answered by AI
Is the research program currently enrolling participants?
"This medical trial is no longer recruiting, according to clinicaltrials.gov; the first post was made in November 13th 2018 and the latest update happened on July 18th 2022. However, 6 other studies are currently enrolling patients for participation."
Answered by AI
Who else is applying?
What site did they apply to?
Nationwide Children's Hospital
What portion of applicants met pre-screening criteria?
Did not meet criteria
Why did patients apply to this trial?
My son is 4.5 years old and has CLN3 Batten disease.
PatientReceived 2+ prior treatments
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger