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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 below

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

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

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

David L Rogers, MDLead Sponsor

David Rogers, MDPrincipal InvestigatorNationwide Children's Hospital

Media Library

Cerliponase Alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05152914 — Phase 1 & 2

Batten Disease Research Study Groups: Intervention

Batten Disease Clinical Trial 2023: Cerliponase Alfa Highlights & Side Effects. Trial Name: NCT05152914 — Phase 1 & 2

Cerliponase Alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05152914 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research include adults above the age of 30?

"Eligible patients for this trial must be between 24 and 72 months old. For context, there are 42 clinical trials underway for children under 18 and 162 for adults over 65."

Answered by AI

How can I get involved in this medical research?

"To join this study, participants must be between 24 and 72 months old, have retinal diseases, and meet the following additional requirements: a willingness to participate in proposed study visits over 2 years, no ocular pathology present that would explain vision loss (besides optic atrophy and pigmentary retina due to CLN2 disease process)."

Answered by AI

How many people can join this experiment at its full capacity?

"Unfortunately, this particular study is no longer recruiting patients. However, there are 191 other trials concerning retinal diseases and 1 for Cerliponase Alfa that are still looking for participants."

Answered by AI

Are there any precedents for using Cerliponase Alfa in a medical capacity?

"Right now, there is only 1 ongoing clinical trial investigating Cerliponase Alfa. That being said, it is a Phase 3 study so it's one of the later stages of research. The majority of studies for this treatment are based in Columbus, Ohio; however, there is at least 1 other location running trials."

Answered by AI

Are patients being sought for this test currently?

"As of now, this clinical trial is not looking for new candidates according to the latest update on clinicaltrials.gov from November 29th, 2021. This study was originally posted on November 1st, 2021. Although this specific trial is not currently recruiting, there are 192 other trials that are actively looking for patients right now."

Answered by AI

Recent research and studies

New England Journal of MedicineJournal

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Schulz, Angela, Temitayo Ajayi, Nicola Specchio, Emily de Los Reyes, Paul Gissen, Douglas Ballon, Jonathan P. Dyke, et al.. 2018. “Study of Intraventricular Cerliponase Alfa for CLN2 Disease”. New England Journal of Medicine. Massachusetts Medical Society. doi:10.1056/nejmoa1712649.

ScienceJournal

Association of Mutations in a Lysosomal Protein with Classical Late-infantile Neuronal Ceroid Lipofuscinosis

Sleat, David E., Robert J. Donnelly, Henry Lackland, Chang-Gong Liu, Istvan Sohar, Raju K. Pullarkat, and Peter Lobel. 1997. “Association of Mutations in a Lysosomal Protein with Classical Late-infantile Neuronal Ceroid Lipofuscinosis”. Science. American Association for the Advancement of Science (AAAS). doi:10.1126/science.277.5333.1802.

tpp1Journal

Mutation Update: Review of <i>tpp1</i> Gene Variants Associated with Neuronal Ceroid Lipofuscinosis CLN2 Disease

Gardner, Emily, Mitch Bailey, Angela Schulz, Mikel Aristorena, Nicole Miller, and Sara E. Mole. 2019. “Mutation Update: Review of tpp1 Gene Variants Associated with Neuronal Ceroid Lipofuscinosis CLN2 Disease”. Human Mutation. Wiley. doi:10.1002/humu.23860.

Biochimica et Biophysica Acta (BBA) - Molecular Basis of DiseaseJournal