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Batten-1 for Juvenile Batten Disease

Not yet recruiting at 9 trial locations
MJ
SR
Overseen BySophie Ribadeau-Dumas, M.S
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Beyond Batten Disease Foundation
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

What You Need to Know Before You Apply

What is the purpose of this trial?

The goal of this open-label, single-arm, multicenter study is to assess the efficacy and safety of Batten-1 (oral liquid formulation of miglustat) (15 mg/kg up to 600 mg/day) in approximately 27 pediatric subjects with CLN3 disease over a period of 52 weeks, using visual acuity as primary efficacy endpoint, The main questions it aims to answer are:

* Does Batten-1 slow down or stabilize the visual acuity decline in pediatric CLN3 patients ?

* What medical problems do participants have when taking Batten-1? in comparison with an external group of non-treated CLN3 disease subjects from natural history studies.

After a titration period of 8 weeks, participants will take Batten-1 every day up to 52 weeks and will visit the clinic every 24 weeks for checkups and tests.

Participants will keep a diary for potential seizure occurrence and compliance to study drug. Interventions they'll be given include:

* visual acuity assessement

* motor and capability assessement

* safety assessment including lab tests and electrocardiogram

* assessment of the effect of the body on Batten-1

* assessment of quality of life

Who Is on the Research Team?

GD

Gary D Clark, MD

Principal Investigator

Texas Children Hospital Houston, Texas, United States, 77030

PG

Paul Gissen, MD PhD

Principal Investigator

Great Ormond Street Hospital, London United Kingdom, WC1N 3JH

AS

Angela Schulz, MD

Principal Investigator

UKE - Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany, 20246

Are You a Good Fit for This Trial?

This trial is for children diagnosed with CLN3 disease (Juvenile Batten disease). Participants should be able to take oral medication and attend regular clinic visits. Those with other major health issues or who can't follow the study plan may not be eligible.

Inclusion Criteria

Ability to complete study assessments and return to clinic as scheduled
I am between 4 and 17 years old and have very poor vision in at least one eye.
My specific CLN3 mutation does not affect my eligibility.
See 3 more

Exclusion Criteria

Clinically significant abnormality in clinical laboratory values at screening precluding participation
Participation in another interventional study within 3 months before screening
Baseline LogMAR score > 1.9 for best corrected visual acuity
See 9 more

What Are the Treatments Tested in This Trial?

Interventions

  • Miglustat

Trial Overview

The study tests a liquid form of miglustat (Batten-1) taken by mouth daily for up to one year, aiming to see if it can slow down vision loss in kids with CLN3. All participants receive the drug; results are compared to data from untreated patients.

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Batten-1 treated armExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Beyond Batten Disease Foundation

Lead Sponsor

Trials
2
Recruited
20,000+

Theranexus

Industry Sponsor

Trials
5
Recruited
310+

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