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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called cerliponase alfa for Batten disease, a rare condition affecting the nervous system. Researchers aim to determine if injecting this medicine into the eye is safe and effective. It targets children diagnosed with the CLN2 type of Batten disease who are already receiving a similar treatment in the brain. Participants should not have any eye problems other than those caused by the disease. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking research.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that cerliponase alfa has been used in children with CLN2 disease. Studies have found it can slow movement and language problems for over five years, suggesting the treatment is generally well-tolerated. However, like any treatment, serious risks exist. These risks were identified when cerliponase alfa received approval for CLN2 disease. The FDA has requested more studies to ensure its safety for children. While researchers are testing this treatment in new ways, evidence from past use shows many patients have tolerated it over time. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Cerliponase Alfa is unique because it is specifically designed to treat Batten Disease by replacing the missing enzyme TPP1 directly in the brain. Most treatments for Batten Disease focus on managing symptoms rather than addressing the root cause. This treatment is administered intravitreally, meaning it's delivered directly into the eye, which helps it reach the brain more effectively. Researchers are excited because this method has the potential to slow down or even halt the progression of the disease, offering hope for improved quality of life for patients.

What evidence suggests that this treatment might be an effective treatment for Batten Disease?

Studies have shown that cerliponase alfa can significantly slow the loss of movement and speech abilities in children with CLN2 disease, a type of Batten disease. Over more than 5 years of treatment, it meaningfully slowed these declines. Research supports that this treatment helps children maintain abilities like walking and talking longer than those who do not receive it. The FDA has approved cerliponase alfa for CLN2 disease, demonstrating strong confidence in its effectiveness. This treatment offers hope by helping to manage the symptoms of this challenging condition. ^{1 2 5 6 7}

Who Is on the Research Team?

David Rogers, MD

Principal Investigator

Nationwide Children's Hospital

Are You a Good Fit for This Trial?

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 for a Trial Participant

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)

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: Intervention Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

David L Rogers, MD

Lead Sponsor

Trials

1

Recruited

5+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11951263/

Real-world clinical outcomes of patients with CLN2 ... In conclusion, cerliponase alfa was found to significantly slow the deterioration in motor and language function in children with CLN2 disease ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1474442223003848

Articles Safety and efficacy of cerliponase alfa in children ... Cerliponase alfa over a mean treatment period of more than 5 years was seen to confer a clinically meaningful slowing of decline of motor and language function ...

3. ninds.nih.gov ninds.nih.gov/about-ninds/what-we-do/impact/ninds-contributions-approved-therapies/cerliponase-alfa-brineurar-ceroid-lipofuscinosis-2-cln2-disease

Cerliponase alfa (Brineura®) – Ceroid lipofuscinosis 2 (CLN2 ... The results showed that treatment slowed declines in walking ability in children with CLN2 disease23. In April 2017, the FDA approved BioMarin' ...

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa1712649

Study of Intraventricular Cerliponase Alfa for CLN2 Disease Intraventricular infusion of cerliponase alfa in patients with CLN2 disease resulted in less decline in motor and language function than that in historical ...

5. biomarin.com biomarin.com/news/press-releases/new-england-journal-of-medicine-published-open-label-study-showing-brineura-cerliponase-alfa-reduced-the-rate-of-clinical-decline-of-children-with-cln2-disease-a-form-of-batten-disease/

New England Journal of Medicine Published Open-label ... Open-label study showing Brineura® (cerliponase alfa) reduced the rate of clinical decline of children with CLN2 disease, a form of batten disease.

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000RiskR.pdf

761052Orig1s000 Cerliponase alfa showed substantial evidence of efficacy in the treatment of CLN2 and fulfills an unmet medical need. The primary serious risks.

7. thelancet.com thelancet.com/journals/laneur/article/PIIS1474-4422(23)00384-8/abstract

Safety and efficacy of cerliponase alfa in children with ... Cerliponase alfa over a mean treatment period of more than 5 years was seen to confer a clinically meaningful slowing of decline of motor and language function ...