CLN3Phase 3NCT07882290
Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease
A Phase 3 study testing Miglustat oral liquid formulation (250 mg/mL) in people with CLN3, run by Beyond Batten Disease Foundation at 10 sites in 8 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 4 to 17 years
- Conditions the study is for
- CLN3
- What is being tested?
- Miglustat oral liquid formulation (250 mg/mL)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 10 sitesin 8 countries
- Study dates
- Dec 2026to Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female subjects ≥ 4 years and <18 years of age with a best corrected visual acuity ≤ 1.9 LogMAR (counting fingers) in at least one eye at baseline
- Parent/legally authorized representative who have signed and dated informed consent on behalf of the subject; subjects older than 7 years of age may sign and date an assent or give a verbal assent in case of vision loss.
- Genetically confirmed diagnosis of syndromic CLN3 disease at screening with EITHER:
- Two pathogenic/likely pathogenic variants in the CLN3 gene according to the American College of Medical Genetics and Genomics and Association for Molecular Pathology standards and guidelines (Richards et al. 2015), OR
- One confirmed pathogenic variant AND one variant of unknown significance, PLUS secondary confirmation with evidence of characteristic inclusions on electron microscopy, or vacuolated lymphocytes as an alternative AND a characteristic clinical course suggestive of CLN3 syndromic disease according to the age.
- 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.
- Male and female subjects of child-bearing potential that are sexually active must use a highly effective method of contraception with a Pearl-Index < 1 % and must continue for the duration of the trial (and for 30 days after the end of treatment).
- Can complete study assessments (subject [e.g., absence of cataracts at baseline that preclude visual acuity assessment] and/or caregiver [compliance to study procedures]) and return to the clinic as scheduled.
Who cannot join
- Subjects with non-syndromic variants reported to be associated with a vision-only phenotype or subjects above 8 years with clinical presentation at baseline of vision-only phenotype.
- Have been evaluated at baseline visit with a LogMAR score > 1.9 for best corrected visual acuity.
- Have any ocular conditions that in the opinion of the investigator might affect visual assessments (e.g. cataract, glaucoma, diabetes, amblyopia, etc…).
- Have received gene therapy or stem cell therapy intended to modify the course of CLN3 disease.
- Have participated in another interventional study during the last three months before screening.
- Have been exposed to miglustat within 1 week prior to baseline, or is scheduled to receive an investigational device or drug (other than test product) during the study.
Show all 12Show fewer
- Have a medical condition that, in the opinion of the investigator, would interfere with the efficacy or safety assessments or increase the subject's risk of adverse events (AEs), including any active malignancy of any type except non-melanoma skin cancer.
- Have a clinically significant abnormality in their clinical laboratory values (hematology, chemistry) at screening that would preclude their participation in the study.
- Have a cardiac impairment including repolarization disturbances (prolonged QTc), ventricular hypertrophy and sinus node dysfunction with severe bradycardia and other conduction issues.
- Have a known allergy or hypersensitivity to miglustat, or any component of the Investigational Medicinal Product (IMP) formulation.
- Have a severe renal disease (estimated Glomerular Filtration Rate (eGFR) < 30 mL/min).
- Have a medical history of HIV, hepatitis B, hepatitis C, or positive results at screening.
Trial locations and contacts
10 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
Sophie Ribadeau-Dumas, M.S contact
10 sites in 8 countries
United States3 sites
Children's Hospital Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
MD
Kourtney K Santucci, MD
Kennedy Krieger Institute, Clinical Trials Unit
Baltimore, Maryland, 21205
MD
researchtrials@kennedykrieger.org
Texas Children Hospital
Houston, Texas, 77030
MD
Gary D Clark, MD
Denmark1 site
Department of Paediatric and Adolescent Medicine Aarhus University Hospital
Aarhus, 8200
Jane Hagelskjær Knudsen, +45 3071 5242, janeknen@rm.dk
Mette Moller Handrup, MD
France1 site
Service de Neurologie Hôpital Pediatrique Armand Trousseau
Paris, Île-de-France Region, 75012
Khaled Alasaadalwekyan, 01 85 55 08 69, khaled.alasaadalwekyan@aphp.fr
Benedicte Heron, MD
Germany1 site
UKE - Universitätsklinikum Hamburg-Eppendorf
Hamburg, 20246
MD
Angela Schulz, MD
Netherlands1 site
UMCU-WKZ, Polikliniek Metabole Ziekten
Utrecht
Lotte van Dam, +31 88 75 540 70, C.H.J.vanDam-6@umcutrecht.nl
Sabine Michel, +31 88 75 540 70, S.Michel-2@umcutrecht.nl
Sweden1 site
Sahlgrenska University Hospital, Queen Silvia Children's Hospital
Gothenburg, 41685
Jenny Gustafsson, +46 (0) 722 035 707, jenny.gustafsson@vgregion.se
Niklas Darin, MD PhD
United Kingdom1 site
Great Ormond Street, London
London, WC1N 3JH
0207 762 6892
Paul Gissen, MD PhD
About this study
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
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.
Read more
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
Read the detailed description
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).
- Conditions
- CLN3
- Being tested
- Miglustat oral liquid formulation (250 mg/mL)
- Keywords
- CLN3, Miglustat, Pediatric subjects, Best corrected visual acuity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Miglustat oral liquid formulation (250 mg/mL)Drug
Also called Batten-1
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.
| Study group | Role | What they receive |
|---|---|---|
| Batten-1 treated arm | Experimental | Batten-1 (miglustat oral liquid formulation) dosed at 250 mg/ml Receives: Drug: Miglustat oral liquid formulation (250 mg/mL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: BCVA will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation.
Secondary outcomes (2)
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).
Measured: 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)
Measured: JNCL Scores will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2026
- Study startDec 2026 (estimated)
- Primary completionJun 2028 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CLN3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beyond Batten Disease Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- Theranexus
- Responsible party
- Sponsor
- Investigators
- Gary D Clark, MD, Principal investigator, Texas Children Hospital Houston, Texas, United States, 77030Paul Gissen, MD PhD, Principal investigator, Great Ormond Street Hospital, London United Kingdom, WC1N 3JHAngela Schulz, MD, Principal investigator, UKE - Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany, 20246
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07882290
- Sponsor's protocol ID
- Batten-1-02
- Other IDs
- 2023-510533-29-00, 2023
- Status last verified
- Oct 2026
- First submitted
- 21 Jul 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: No plan to perform individual participant data sharing due to GDPR regulation
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07882290
- Acronym / sponsor ID
- Batten-1-02
- Conditions
- CLN3
- Keywords
- CLN3, Miglustat, Pediatric subjects, Best corrected visual acuity
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- Beyond Batten Disease FoundationUniversity, hospital or other
- Collaborators
- Theranexus
- Study start
- Dec 2026 (estimated)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 20 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Batten-1 treated arm | Experimental | Drug: Miglustat oral liquid formulation (250 mg/mL)Batten-1 (miglustat oral liquid formulation) dosed at 250 mg/ml |
Who may qualify
- Ages
- 4 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Male or female subjects ≥ 4 years and <18 years of age with a best corrected visual acuity ≤ 1.9 LogMAR (counting fingers) in at least one eye at baseline
- Parent/legally authorized representative who have signed and dated informed consent on behalf of the subject; subjects older than 7 years of age may sign and date an assent or give a verbal assent in case of vision loss.
- 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.
- Male and female subjects of child-bearing potential that are sexually active must use a highly effective method of contraception with a Pearl-Index < 1 % and must continue for the duration of the trial (and for 30 days after the end of treatment).
Key exclusion criteria (12 listed)
- Subjects with non-syndromic variants reported to be associated with a vision-only phenotype or subjects above 8 years with clinical presentation at baseline of vision-only phenotype.
- Have been evaluated at baseline visit with a LogMAR score > 1.9 for best corrected visual acuity.
- Have any ocular conditions that in the opinion of the investigator might affect visual assessments (e.g. cataract, glaucoma, diabetes, amblyopia, etc…).
- Have received gene therapy or stem cell therapy intended to modify the course of CLN3 disease.
- Have participated in another interventional study during the last three months before screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Miglustat oral liquid formulation (250 mg/mL)Also: Batten-1Batten-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. | Drug | Batten-1 treated arm |
Primary outcome
Best Corrected Visual Acuity (BCVA)
Time frame: 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.
2 secondary outcomes
Change from baseline in Unified Batten Disease Rating Scale (UBDRS) subscales
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)
Time frame: JNCL Scores will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Individual participant dataSharing planned: No. No plan to perform individual participant data sharing due to GDPR regulation
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sophie Ribadeau-Dumas, M.S contact
Gary D Clark, MD principal investigator
Texas Children Hospital Houston, Texas, United States, 77030
Paul Gissen, MD PhD principal investigator
Great Ormond Street Hospital, London United Kingdom, WC1N 3JH
Angela Schulz, MD principal investigator
UKE - Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany, 20246
10 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Miglustat oral liquid formulation (250 mg/mL) in people with CLN3, run by Beyond Batten Disease Foundation at 10 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Denmark, France, Germany, Italy and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Marie J Sebille, MD, PhD (+33688941976, marie.sebille@THXPharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Best Corrected Visual Acuity (BCVA) (time frame: BCVA will be measured at baseline, 26 weeks, 52 weeks and in case of premature treatment discontinuation.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.