AT-GTX-501 for Batten Disease

Overseen By Amicus Therapeutics Patient Advocacy

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Emily de los Reyes

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on understanding the long-term safety and effectiveness of AT-GTX-501, a gene therapy for individuals with CLN6 Batten disease. The study specifically includes those who have already received this gene therapy in a previous trial. Participants must have a legally authorized representative who has consented to their participation. The research aims to provide insights into the treatment's long-term effectiveness in managing this rare genetic disorder. As a Phase 4 trial, it helps determine how the FDA-approved treatment benefits a broader patient population.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What is the safety track record for AT-GTX-501?

Research shows that AT-GTX-501 is generally well tolerated in children with CLN6 Batten disease. Early safety results from studies with 13 patients indicate that most have managed this gene therapy treatment well. Although data collection continues, no major safety issues have been reported. This suggests that AT-GTX-501 could be a safe option for those considering participation in the clinical trial. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

AT-GTX-501 is unique because it targets the genetic root of CLN6 Batten disease through gene transfer, offering a potentially transformative approach. Unlike current treatments, which mainly focus on managing symptoms and slowing disease progression, AT-GTX-501 aims to correct the underlying genetic defect. This approach could lead to more significant and long-lasting improvements in patient outcomes, which is why researchers are particularly excited about its potential.

What evidence suggests that AT-GTX-501 might be an effective treatment for CLN6 Batten disease?

Research has shown that the gene therapy AT-GTX-501, which participants in this trial may receive, may help children with CLN6 Batten disease. In earlier studies, this therapy helped maintain motor and language skills in children for two years after treatment. Administered once directly into the spine, the treatment aims to slow the disease's progression. These findings suggest that AT-GTX-501 could help manage symptoms and provide some stability for children with this condition. ^{1 2 3 6 7}

Who Is on the Research Team?

Emily de los Reyes, MD

Principal Investigator

Nationwide Children's Hospital

Are You a Good Fit for This Trial?

Inclusion Criteria

Subject has a legally authorized representative who has provided written informed consent and authorization for use and disclosure of personal health information or research-related health information.

Subject received AT-GTX-501 (scAAV9.CB.CLN6) in the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9."

Subject completed or prematurely discontinued from the study "Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9."

Timeline for a Trial Participant

Initial Treatment

Participants received a single intrathecal administration of AT-GTX-501

2 years

Long-Term Follow-Up

Participants complete safety and efficacy assessments following the initial treatment study

3 years

Overall Follow-Up

Combining the initial treatment and long-term follow-up, participants are monitored for up to 5 years since gene transfer

5 years

What Are the Treatments Tested in This Trial?

Interventions

• AT-GTX-501

How Is the Trial Designed?

Group I: Subjects who received AT-GTX-501 gene transfer Experimental Treatment 1 Intervention

Subjects with CLN6 Batten disease who previously received AT-GTX-501 in the preceding study (Study AT-GTX-501-01).

Who Is Running the Clinical Trial?

Emily de los Reyes

Lead Sponsor

Trials

1

Recruited

30+

Amicus Therapeutics

Lead Sponsor

Trials

55

Recruited

2,700+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02725580

Study Details | NCT02725580 | Gene Therapy For Children ...

This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord ...

2.

ir.amicusrx.com

ir.amicusrx.com/news-releases/news-release-details/amicus-therapeutics-announces-additional-positive-interim

Investors

The interim results show that this investigational gene therapy has the potential to slow the neurological disease progression in children with CLN6 Batten ...

3.

battendiseasenews.com

battendiseasenews.com/news/at-gtx-501-effective-after-2-years-cln6-batten-children-phase-1-2-trial/

In CLN6 Batten, AT-GTX-501 Effective After 2 Years, Interim...

Amicus' AT-GTX-501 gene therapy stabilized motor and language function in children with CLN6 Batten disease two years after treatment.

4.

scge.mcw.edu

scge.mcw.edu/platform/data/clinicalTrials/report/NCT02725580

Gene Therapy For Children With Variant Late Infantile ...

This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04273243

Long-Term Follow Up of CLN6 Batten Disease Subjects ...

The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease. The secondary outcome measure of this ...

6.

ctv.veeva.com

ctv.veeva.com/study/long-term-follow-up-of-cln6-batten-disease-subjects-following-gene-transfer

Long-Term Follow Up of CLN6 Batten Disease Subjects ...

The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease. ... Data sourced from ...

7.

patientworthy.com

patientworthy.com/2020/10/15/positive-data-announced-cln6-batten-gene-therapy/

Positive Data Announced for CLN6 Batten Disease Gene ...

Currently, an ongoing Phase 1/2 clinical trial is determining the safety, efficacy, and tolerability of AT-GTX-501 for pediatric patients. So ...