A Phase 3 interventional study of Miglustat oral liquid formulation (250 mg/mL) in CLN3, sponsored by Beyond Batten Disease Foundation. Not yet recruiting at 10 sites in 8 countries. Open to participants aged 4 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-20.
Sponsored by Beyond Batten Disease Foundation · Phase 3, Interventional, and Treatment
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:
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:
Batten-1 (miglustat oral liquid formulation) may impact the progression of CLN3 disease via mechanisms similar to those described for the treatment of the related neurodegenerative lysosomal storage disorders, Gaucher disease and NPC disease. The dose recommendation follows the European Zavesca label for pediatric and adult Niemann Pick C (NPC) disease, adapted to a weight-based administration of 15mg/kg with a Tres In Dies (TID) dose regimen. Given the concentration of miglustat in the liquid formulation of 250 mg/mL, a volume of 0,02 mL, - the minimal quantity measurable with the pipette - represents an increment of dose of 5 mg, which will allow an adaptation of dose per kilogram (e.g., the lowest daily target dose planned to be administered will be 225 mg/d in children weighting 15 kg as a mean at 4 years old. For children with a weight below 15 kg, the same dose adjustment will be applied).
To mitigate gastro-intestinal events observed with miglustat, this study will include a dose-titration period of 6 dose levels across 8 weeks if the drug is well tolerated. If needed for tolerability reasons, a dose may be repeated for 1 or more weeks prior to increasing. Treatment with miglustat will begin at a dose of half the individual weight-based dose per intake (i.e., 1/6 of the weight-based daily target dose). Subjects will initiate miglustat on Day 1 of Week 1. If the initial dose of miglustat is well tolerated, then the dose will be increased weekly until the target dose of 15 mg/kg is reached, without exceeding the maximal recommended dose according to the subject-related body weight. A low-carbohydrate diet will be started prior to initiation of drug administration, after consent has been obtained, to also improve gastrointestinal tolerability of miglustat and potentially lessen the magnitude of weight loss in reducing diarrhoea.
Once the dose titration is completed, subjects will continue their maximal dose during the maintenance period (the remainder of the 52-week treatment duration, 44 weeks). Temporary or permanent dose reductions of miglustat performed for tolerability as per the investigator's decision, in particular for gastro-intestinal events, are permitted at any time during the maintenance period of the study and must be documented, as well as any re-escalation attempt.
A post-treatment Follow-up Period will consist of an End-of-Study visit approximately 30 days after the last dose, at which safety assessments will be performed.
Approximately 27 subjects are expected to enroll in the study. Subject participation is approximately 56 weeks (Treatment duration 52 weeks after first treatment administration, follow-up end of study visit after approximately 4 weeks).
Beyond Batten Disease Foundation is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Genetically confirmed diagnosis of syndromic CLN3 disease at screening with EITHER:
There is no restriction on the specific CLN3 mutations for eligibility to enroll in the study. The mutations will be recorded in the electronic case report form (eCRF) for potential use in determining if CLN3 genotype is associated with tolerability or efficacy of Batten-1 therapy.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Batten-1 (miglustat oral liquid formulation) dosed at 250 mg/ml
Drug: Miglustat oral liquid formulation (250 mg/mL)
Batten-1 will be administered at a dose of 15 mg/kg/day up to 600 mg /day T.I.D. during 52 weeks. A dose titration will be performed during the first 8 weeks of the treatment to reduce potential gastro-intestinal events that may occur at beginning of treatment.
Also known as: Batten-1
Best Corrected Visual Acuity (BCVA)
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.
Time frame: BCVA will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation.
Change from baseline in Unified Batten Disease Rating Scale (UBDRS) subscales
* 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).
Time frame: UBDRS subscales will be evaluated at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation
Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL)
Modified Hamburg Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) scores: (motor function, max: 3=normal, min: 0=immobile)
Time frame: JNCL Scores will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation
General Health Status
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.
Time frame: General Health Status questionnaire will be evaluated at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
The 23-item PedsQL 4.0 Generic Core Scales
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).
Time frame: The 23-item PedsQL 4.0 Generic Core Scales is assessed at baseline, Week 26, Week 52 and in case of premature treatment discontinuation.
Safety assessment
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
Time frame: 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.
PK Assessment Cmax
PK Assessment Maximum Plasma Concentration Cmax in ng/mL
Time frame: Pk assessment Cmax will be conducted at Day 1 and Week 26.
PK Assessment Tmax
PK Assessment Time to Maximum Plasma Concentration Cmax in hour
Time frame: Pk assessment Tmax will be conducted at Day 1 And Week 26.
PK Assessment AUC 0-t, AUC 0-∞
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
Time frame: Pk assessment AUC will be conducted at Day 1 and Week 26.
PK Assessment AUC 0-8hr (equivalent to AUC 0-Tau)
Area Under Curve (0-8hr equivalent to AUC 0-Tau) in h\*ng/mL
Time frame: Pk assessment Tmax will be conducted at Week 26.
PK Assessment T1/2
Elimination half-life in hour
Time frame: Pk assessment T1/2 will be conducted at Day 1
Body weight
Body weight will be measured in kilograms
Time frame: Body weight will be measured at screening, baseline, on a weekly basis from Week 2 to Week 8, and on Weeks 9,11, 13, 17, 21, 26, 30, 34, 38, 42, 48 and 52, and in case of premature treatment discontinuation.
Height
Height will be measured in centimeters
Time frame: Height will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
ECG heart rate
12-lead electrocardiogram heart rate in beats / minute
Time frame: ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
ECG QT interval
12-lead electrocardiogram QT interval (msec)
Time frame: ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
ECG PR interval
12-lead electrocardiogram PR interval (msec)
Time frame: ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
ECG QRS interval
12-lead electrocardiogram QRS interval (msec)
Time frame: ECG will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests haematology
Hemoglobin (g/L)
Time frame: Hemoglobin will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests haematology
Hematocrit (%) Differential white blood cell count (%)
Time frame: Hematocrit and differential white blood cell count will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests haematology
Erythrocytes (Red Blood Cells Count (RBC): x 10¹²/L) Leukocytes (White Blood Cell Count (WBC): × 10⁹/L) Thrombocytes (Platelets: × 10⁹/L)
Time frame: Erythrocytes, leucocytes and thrombocytes will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests chemistry
Creatinine (µmol/L) Total bilirubin (µmol/L)
Time frame: Creatinine and total bilirubin will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests chemistry
Alkaline phosphatase (IU/L) Aspartate aminotransferase (IU/L) Alanine aminotransferase (IU/L) Gamma glutamyl transpeptidase (IU/L) Creatine kinase (IU/L)
Time frame: Blood enzymes will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests chemistry
Sodium (mEq/L) Potassium (mEq/L) Chlorides (mEq/L) Carbon dioxide (mEq/L)
Time frame: Blood ions will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Lab tests chemistry
urea (mmol/L)
Time frame: Urea will be measured at baseline, Week 26, Week 52 and in case of premature treatment discontinuation
Plan to share: No — No plan to perform individual participant data sharing due to GDPR regulation
No publications or documents are linked to this record.
Start is estimated for Dec 2026; primary completion for Jun 2028, 18 months later.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Beyond Batten Disease Foundation