Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease
Public ClinicalTrials.gov record NCT07882290. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label, Single-arm, Multicenter, Efficacy and Safety Study to Investigate Batten-1 (Miglustat Oral Liquid Formulation) for the Treatment of Pediatric Subjects With CLN3 Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT07882290
- Recruitment status
- Not yet recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 27 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 2026 (Estimated)
- Primary completion
- Jun 2028 (Estimated)
- Completion
- Jun 2028 (Estimated)
- Last update posted
- Oct 20, 2026
2026 – 2028
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | — |
| Kennedy Krieger Institute, Clinical Trials Unit | Baltimore | Maryland | 21205 | — |
| Texas Children Hospital | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 7 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07882290, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 20, 2026 · Synced Oct 20, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT07882290 live on ClinicalTrials.gov.