Trial · NCT07882290
Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease
NCT07882290, Phase 3, Not yet recruiting, Beyond Batten Disease Foundation
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease. Trials, record NCT07882290. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07882290
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease
- NCT number
- NCT07882290
- Status
- Not yet recruiting
- Phases
- Phase 3
- Enrollment
- 27 participants, estimated
- Lead sponsor
- Beyond Batten Disease Foundation
- Conditions
- CLN3
- Interventions
- Miglustat oral liquid formulation (250 mg/mL)
- First posted
- 2026-10-20
- Last updated
- 2026-10-20
Source ClinicalTrials.gov
Registered outcomes24
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Best Corrected Visual Acuity (BCVA) | BCVA will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation. | Visual Acuity will be measured in each eye by an experienced Visual Acuity Examiner (VAE, ophthalmologist, orthoptist, optometrist, or trained ophthalmic technician) using either: * The Early Treatment Diabetic Retinopathy Study Logarithm of the Minimum Angle of Resolution chart in subjects evaluable by this method. Each line of the LogMAR chart comprises the same number of test letters (effectively standardizing the test across letter size); letter size from line to line varies logarithmically. Adapted tests (LEA symbol tests) will be used for pre-school age subjects (below 6 years of age), * If the subject is unable to read the chart at any distance, visual acuity will be tested one foot distance, using qualitative assessment (counting fingers, hand motion, light/no light perception). Each of these conditions corresponds to a LogMAR value to be used for efficacy analysis. |
| Secondary | Change from baseline in Unified Batten Disease Rating Scale (UBDRS) subscales | UBDRS subscales will be evaluated at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation | * Unified Batten Disease Rating Scale subscale - Clinical Global Impression - Overall severity (min: 0=none, max: 5=severe) * Unified Batten Disease Rating Scale - modified Capability subscale 4 items (school, chores, play, Activities of Daily Living, min:0=normal, max: 12=most severe) * Unified Batten Disease Rating Scale modified - Physical Assessment subscale 2 items (speech clarity and gait, min: 0=normal, max: 8=most severe) * Unified Batten Disease Rating Scale - Clinical Global Impression severity subscores (seizures, cognitive function, behavior, mood, and motor, min: 1=no impairment, max: 5=severely impaired for each subscore). |
| Secondary | Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) | JNCL Scores will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation | Modified Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) scores: (motor function, max: 3=normal, min: 0=immobile) |
| Other | General Health Status | General Health Status questionnaire will be evaluated at baseline, Week 26, Week 52 and in case of premature treatment discontinuation | HEALTH UTILITIES INDEX® Mark 2 and Mark 3 40-Item Questionnaire (HUI23P2USEn.40Q). The maximal score is 1.0, corresponding to a perfect health status, the minimal score represents death. |
| Other | The 23-item PedsQL 4.0 Generic Core Scales | The 23-item PedsQL 4.0 Generic Core Scales is assessed at baseline, Week 26, Week 52 and in case of premature treatment discontinuation. | The 23-item PedsQL 4.0 Generic Core Scales is a a parent proxy-report that assesses parents' perceptions of their child's Health Related Quality Of Life. Each item is evaluated with a 5-point response scale (0=never a problem, 4=almost always a problem). |
| Other | Safety assessment | Safety assessment will be performed at each of the 4 on-site visits, during the 5 phone calls or the 13 televisits, and in case or premature treatment discontinuation. | Number of participants with treatment-emergent adverse events (TEAEs), TEAEs leading to permanent discontinuation and serious adverse events (SAEs), adverse events of special interest, with abnormal laboratory tests results, abnormal 12-lead electrocardiogram (ECG) findings, abnormal slit lamp evaluation, abnormal vital signs as well as abnormal physical, neurological and mental state examinations findings |
| Other | PK Assessment Cmax | Pk assessment Cmax will be conducted at Day 1 and Week 26. | PK Assessment Maximum Plasma Concentration Cmax in ng/mL |
| Other | PK Assessment Tmax | Pk assessment Tmax will be conducted at Day 1 And Week 26. | PK Assessment Time to Maximum Plasma Concentration Cmax in hour |
| Other | PK Assessment AUC 0-t, AUC 0-∞ | Pk assessment AUC will be conducted at Day 1 and Week 26. | Area under the concentration-time curve calculated to the last observable concentration at time t (AUC 0-t), area under the concentration-time curve extrapolated to infinity (AUC 0-∞) in h\*ng/mL |
| Other | PK Assessment AUC 0-8hr (equivalent to AUC 0-Tau) | Pk assessment Tmax will be conducted at Week 26. | Area Under Curve (0-8hr equivalent to AUC 0-Tau) in h\*ng/mL |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07882290
GET https://conductscience.org/api/trials/v1/NCT07882290/registered?page=2