Intravitreal Cerliponase Alfa for Batten Disease

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: David L Rogers, MD

Must be taking: Cerliponase alfa

Must not be taking: Chemotherapy, Radiotherapy, Immunosuppressants

Disqualifiers: Ocular trauma, Severe infection, others

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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

5 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on chemotherapy, radiotherapy, or other immunosuppression therapy, you must have stopped these at least 30 days before joining the trial. Corticosteroid treatment might be allowed, but you should check with the trial investigator.

How is the drug Cerliponase Alfa unique in treating Batten Disease?

Cerliponase Alfa is unique because it is an enzyme replacement therapy specifically designed for CLN2, a type of Batten Disease, and is administered directly into the brain (intracerebroventricular infusion) to help slow neurological decline, although it does not prevent vision loss. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial involves injecting a medication called cerliponase alfa directly into the eyes of 5 subjects. The goal is to see if this treatment is safe and effective over a period of time. The subjects will then be monitored periodically for an extended duration to collect more data.

Research Team

David Rogers, MD

Principal Investigator

Nationwide Children's Hospital

Eligibility Criteria

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.

Inclusion 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.

Exclusion Criteria

Has a medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability

You have any cloudiness in your eyes that could affect vision.

I had a major seizure episode within the last month.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravitreal injections of cerliponase alfa under sedation every 4 weeks

24 months

Bi-monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

Bi-annual visits (in-person)

Treatment Details

Interventions

• Cerliponase Alfa

Trial Overview 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.

Participant Groups

Group I: Intervention Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

David L Rogers, MD

Lead Sponsor

Trials

1

Recruited

5+

References

1. England pubmed.ncbi.nlm.nih.gov

Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease). [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Combined photodynamic therapy and intravitreal triamcinolone for choroidal neovascularization secondary to punctate inner choroidopathy or of idiopathic origin: one-year results of a prospective series. [2018]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Evolution of the retinal function by flash-ERG in one child suffering from neuronal ceroid lipofuscinosis CLN2 treated with cerliponase alpha: case report. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Treatment of polypoidal choroidal vasculopathy with photodynamic therapy. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Treatment of polypoidal choroidal vasculopathy with photodynamic therapy. 2002. [2022]