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Enzyme Replacement Therapy
Intravitreal Cerliponase Alfa for Batten Disease
Phase 1 & 2
Waitlist Available
Led By David Rogers, MD
Research Sponsored by David L Rogers, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Minimum age requirement: 24 months of age at enrollment
Currently receiving intraventricular cerliponase alfa
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
Study Summary
This trial is testing a new drug to treat a disease called Batten. It is testing to see if the drug is safe and effective.
Who is the study for?
This trial is for children aged 24 to 72 months with classical CLN2 Batten's disease, confirmed by a certified lab. They must have a specific retinal thickness, be deficient in an enzyme called tripeptidyl-peptidase, and already be receiving intraventricular cerliponase alfa. Kids can't join if they've had recent immunosuppression therapy, ocular trauma/surgery, severe infections or bleeding disorders.Check my eligibility
What is being tested?
The trial tests the safety and effectiveness of injecting cerliponase alfa directly into the eye (intravitreal) to prevent worsening of retinal disease in kids with CLN2 Batten's disease. It's a phase I/II study where participants are randomly assigned and masked to treatment conditions.See study design
What are the potential side effects?
Potential side effects aren't specified but may include typical reactions related to eye injections such as discomfort, redness, swelling at the injection site or inside the eye, increased intraocular pressure or temporary vision changes.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am at least 2 years old.
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I am currently being treated with cerliponase alfa.
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My CLN2 Batten's disease diagnosis was confirmed through genetic testing.
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I am 6 years old or younger.
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My eyes are clear and free from any cloudiness.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Monitoring for the development of unacceptable toxicity.
Secondary outcome measures
Efficacy of intravitreal cerliponase alfa to stabilize fundoscopic features.
Efficacy of intravitreal cerliponase alfa to stabilize retinal architecture.
Trial Design
1Treatment groups
Experimental Treatment
Group I: InterventionExperimental Treatment1 Intervention
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Enzyme replacement therapy (ERT) for Batten Disease, such as Cerliponase Alfa, involves administering a recombinant form of the deficient enzyme, in this case, tripeptidyl peptidase 1 (TPP1). This enzyme is crucial for breaking down certain proteins within lysosomes.
In Batten Disease, mutations in the CLN2 gene lead to TPP1 deficiency, causing toxic accumulation of proteins and subsequent neurodegeneration. By providing the missing enzyme, ERT helps reduce this accumulation, potentially slowing disease progression and improving neurological function.
This approach is significant for Batten Disease patients as it directly addresses the underlying enzyme deficiency, offering a targeted treatment that can mitigate some of the severe symptoms associated with the disease.
Oral cysteamine bitartrate and N-acetylcysteine for patients with infantile neuronal ceroid lipofuscinosis: a pilot study.
Oral cysteamine bitartrate and N-acetylcysteine for patients with infantile neuronal ceroid lipofuscinosis: a pilot study.
Find a Location
Who is running the clinical trial?
David L Rogers, MDLead Sponsor
David Rogers, MDPrincipal InvestigatorNationwide Children's Hospital
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am at least 2 years old.I am currently being treated with cerliponase alfa.My CLN2 Batten's disease diagnosis was confirmed through genetic testing.You have any cloudiness in your eyes that could affect vision.Your retinal thickness must be at least 140 micrometers, as measured by an OCT scan.I had a major seizure episode within the last month.My vision loss is due to CLN2 disease, not other eye conditions.I haven't had chemotherapy, radiotherapy, or immunosuppression in the last 30 days.I have had an eye injury or eye surgery in the past.I have a history of bleeding disorders.I am 6 years old or younger.I have not had a severe infection like pneumonia or meningitis in the last 4 weeks.Your body doesn't have enough of the tripeptidyl-peptidase enzyme.My eyes are clear and free from any cloudiness.
Research Study Groups:
This trial has the following groups:- Group 1: Intervention
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.
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