Wolfram SyndromePhase 2NCT03717909
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
A Phase 2 study testing Sodium Valproate 200Mg E/C Tablet in people with Wolfram Syndrome, run by University of Birmingham at 6 sites in 4 countries.
Trial summary
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome. 70 paediatric and adult patients were initially planned to be randomised 2:1 to receive either sodium valproate or placebo at 6 international centres. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Wolfram Syndrome
- What is being tested?
- Sodium Valproate 200Mg E/C Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 28 Dec 2018to 15 Nov 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients must meet all of the following criteria to be eligible for enrolment:
- The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
- Documented diabetes mellitus diagnosed under 16 completed years according to WHO or ADA criteria plus documented optic atrophy diagnosed under 16 completed years
- AND B) Documented functionally relevant mutations on one or both alleles of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
- The patient is aged 6 years or older and weighing at least 20kg.
- The patient's visual acuity assessed as either the right eye or left eye having a LogMAR score of 1.6 or better on an ETDRS chart, with or without corrected vision.
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- Written informed consent (and assent as required).
- Females of child bearing potential will only be included after a negative highly sensitive urine pregnancy test. If sexually active, they must agree to use a highly effective contraception measure and to pregnancy testing at each clinic follow up visit- see 4.1.1 for further information.
- Sexually active men with a female partner of childbearing potential must agree to the use of condoms and the use of a highly effective method of contraception by the female partner
- Patient willing and able to meet all protocol defined visits for the duration of the Trial
- Pregnancy
- Adequate counselling must be given to all female patients of childbearing potential regarding the risks associated with Sodium Valproate use in pregnancy because of the potential teratogenic risk to the foetus. In the UK, Treat Wolfram protocol will be following the Valproate Pregnancy Prevention programme as per UK standard practice. Other countries will follow their local procedures as dictated by their local competent authority.
- Female patients who have started their periods but are not sexually active will be given contraception advice. If under 16 years, the advice will be given to the patient and their parents or carers.
- In line with Clinical Trial Facilitation Group Guidance, a woman is considered of childbearing potential (WOCBP), i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy.
- Due to the potential teratogenic risk to the foetus, all women of childbearing potential (WOCBP) must use a highly effective method of contraception, without interruption during the entire duration of IMP treatment. A highly effective method of contraception according to the Clinical Trial facilitation Group guidance includes methods that can achieve a failure rate of less than 1% per year when used consistently and correctly. Such methods include:
- combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:
- Oral
- Intravaginal
- Transdermal
- progestogen-only hormonal contraception associated with inhibition of ovulation:
- Oral
- Injectable
- Implantable 1
- intrauterine device (IUD) 1
- intrauterine hormone-releasing system ( IUS) 1
- bilateral tubal occlusion 1
- vasectomised partner 1, 2
- sexual abstinence 3
- Contraception methods that in the context of this guidance are considered to have low user dependency.
- Vasectomised partner is a highly effective birth control method provided that partner is the sole sexual partner of the WOCBP trial participant and that the vasectomised partner has received medical assessment of the surgical success.
- In the context of this guidance sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject.
Who cannot join
- Patients who meet any of the following criteria are not eligible for this Trial:
- The patient has clinically significant non-Wolfram related CNS involvement which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
- The patient has a diagnosis of a mitochondrial myopathy
- The patient has active liver disease, has a personal or family history of liver dysfunction related to known genetic disorders, or patient has porphyria.
- The patient has received treatment with any investigational drug within the 30 days prior to Trial entry.
- The patient is currently taking sodium valproate; or has a known hypersensitivity to sodium valproate or its excipients.
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- Any other acute or chronic medical, psychiatric, social situation or laboratory result that, based on investigator's judgment, would jeopardize patient safety during trial participation, cause inability to comply with the protocol, or affect the Trial outcome.
- The patient is currently breastfeeding.
- The patient has Known urea cycle disorders.
- The patient has one of the following disorders: Lactose intolerance, the Lapp lactase deficiency, or glucose- galactose malabsorption.
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
France2 sites
CHU de Montpellier, Hopital Gui de Chauliac
Montpellier, 34295
Hôpital Européen Georges-Pompidou
Paris, 75015
United Kingdom2 sites
University Hospitals Birmingham
Birmingham, B15 2TH
Birmingham Women's and Children's Hospital
Birmingham, B4 6NH
Spain1 site
Unidad de Gestión Clínica Almería Periferia. Distrito Sanitario Almería
Almería, 04120
About this study
Official title: A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome. 70 paediatric and adult patients were initially planned to be randomised 2:1 to receive either sodium valproate or placebo at 6 international centres. 63 patients were recruited when a decision was made to stop the study recruitment in November 2022.
Read the detailed description
This phase II clinical trial is planned as a randomised, double-blind, placebo-controlled 3 year intervention Trial in 70 patients with Classical Wolfram Syndrome aged 6 years and over. The primary outcome of the Trial is considered to be clinically relevant and of sufficient magnitude to be beneficial, as a successful Trial outcome will mean that patients will retain a clinically useful degree of visual acuity and it will decline at a slower rate than in the untreated patients. The MRI Pons Volume change has been shown to correlate with changes in the Wolfram Unified Rating Scale.
Patients will be randomised to balance the individual differences across the treatment and placebo groups, therefore reducing the potential for extraneous bias. This will ensure that the treatment effect can be established without the need to account for confounding factors. The value of a placebo arm adds robustness to the Trial by removing the potential for bias from both the investigator and patient perspectives.
Investigators will be blinded to the results of the assessments. Certain assessments will be performed by subspecialists (such as ophthalmologists and neurologists), with the Principal Investigator prevented from having access to the results. This subspecialist-led treatment is in line with the current multi-disciplinary management of these patients and will not result in patients being denied access to effective treatment.
Patients and investigators will be blinded to treatment. The Trial treatment will be a tablet formulation.
This Trial involves 11 clinic visits and 7 follow up telephone calls to reduce the burden of additional travel to the patients.
- Conditions
- Wolfram Syndrome
- Being tested
- Sodium Valproate 200Mg E/C Tablet, Sodium Valproate matched placebo
- Keywords
- Wolfram Syndrome, Treat Wolfram, Sodium valproate
- Medical subject headings
- Wolfram Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sodium Valproate 200Mg E/C TabletDrug
Also called Sodium Valproate
Treatment with twice-daily oral tablet(s)
Sodium Valproate matched placeboDrug
Also called Placebo
Treatment with twice-daily oral 200mg tablet(s)
| Study group | Role | What they receive |
|---|---|---|
| Experimental Group | Experimental | Sodium Valproate 200Mg E/C Tablet (active treatment) Receives: Drug: Sodium Valproate 200Mg E/C Tablet |
| Control Group | Placebo comparator | Sodium Valproate matched placebo (inactive treatment) Receives: Drug: Sodium Valproate matched placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual acuity (VA)
Visual acuity (VA) is measured on the logMAR scale by sight tests in clinic using Early treatment diabetic retinopathy study (ETDRS) charts. Values are taken for each eye separately, both uncorrected, and corrected with glasses or contact lenses, and can range from 0, which represents perfect vision i.e. 20/20 (values of -0.1 and -0.2 are also possible representing better than perfect vision), to +2 which represents near blindness i.e. 20/2000. Increases in logMAR represent deterioration.
Measured: 36 months
Secondary outcomes (25)
Safety - adverse events
measured by adverse events frequency, type and grade according to CTCAE v4
Measured: 37 months
Tolerability - highest treatment dose
measured by dose achieved
Measured: 36 months
Tolerability - duration of treatment
measured by days of treatment
Measured: 36 months
Tolerability - dosing modifcation
measured by treatment-related dose reductions and discontinuations
Measured: 36 months
Pons Volume
a surrogate marker for neurodegeneration, measured and recorded in mm3 by standardised analysis of MRI images of the Pons, and brain substructure volumes.
Measured: 37 months (+/- 6 months)
Brainstem volume
measured by MRI as with Pons Volume
Measured: 37 months (+/- 6 months)
Retinal nerve thickness
measured by Optical Coherence Tomography
Measured: 37 months
Colour vision
measured by Hardy Rand and Rittler test
Measured: 37 months
Visual fields
measured by the local centre standard process (the same technique must be used throughout the patient participation to the study)
Measured: 37 months
Data on cataracts
measured by incidence and frequency of cataracts
Measured: 37 months
Afferent pupillary defects
measured by incidence and frequency of afferent pupillary defects. Afferent pupillary defects are recorded as present or absent.
Measured: 37 months
Strabismus
measured by incidence and frequency of strabismus. Presence or absence of strabismus will be recorded. Strabismus will also be graded for type and size.
Measured: 37 months
Nystagmus
measured by incidence and frequency of nystagmus. Presence or absence of nystagmus will be recorded. Nystagmus will also be graded for size, amplitude and direction.
Measured: 37 months
Visual evoked potentials
measured by changes in visual evoked potentials (if available)
Measured: 37 months
Sleep - sleeping habits parent report for patients under 18 years
measured by the Sleep-related Breathing disorder scale extracted from the Pediatric Sleep Questionnaire (PSQ) Parent Questionnaire 2014. This report is a Parent Report for patients under 18. This questionnaire records usual sleep habits.
Measured: 37 months
Sleep - sleeping habits, self-report
measured by the Pittsburg Sleep Quality Index (PSQI) Self-Report. This questionnaire is completed by the patient. This questionnaire records usual sleep habits during the past month.
Measured: 37 months
Balance
measured by Mini-BESTest
Measured: 37 months
Hearing
measured by pure tone audiometry
Measured: 37 months
Wolfram Unified Rating Scale
Wolfram Unified Rating Scale (WURS). Assessments are performed in five areas (physical; seizure; behavioral; capability and clinical) by scoring listed items 0-2, 0-3, 0-4, 0-5 or 0-6 depending on the scale. Totals for each category are recorded and the WURS total, summing physical and behavioral categories, is also recorded. A low score would be considered a better outcome in all areas apart from capability where a high score would be considered a better outcome. A Wolfram Syndrome history is also recorded detailing incidence and onset of listed symptoms.
Measured: 37 months
Mood
measured by Kidscreen for patients aged 8-18 or the Hospital Axiety and Depression Scale (HADS) for adult patients. Kidscreen records the patients mood and feeling in 5 areas (physical activities and health; general mood and feelings about self; family and free time; friends; school and learning). HADS records how the patient has been feeling over the past week by scoring feelings relating to anxiety or depression. A total score for Anxiety and a total score for depression is recorded. A score of 0-7 = normal; 8-10 = borderline abnormal (borderline case) and 11-21 = abnormal (case).
Measured: 36 months
Quality of life - PedsQL
measured by PedsQL questionnaire (pediatric quality of life inventory) for paediatric patients. PedsQL 8-12 and PedsQL 13-18 and respective parent reports. PedsQL records how much of a problem each situation causes the patient; each situation is scored from 0 (never a problem) to 4 (always a problem). A score of 0 would be considered a better outcome.
Measured: 37 months
Quality of life - ICIQ-FLUTS
measured by the ICIQ-FLUTS questionnaire: ICIQ-FLUTS (adult females), ICIQ-MLUTS (adult males), or ICIQ-CLUTS (children and caregiver forms). This questionnaire records urinary symptoms in three categories (filling, voiding and incontinence) from 0-4 and how much each symptom bothers the patient from 0 (not at all) to 10 (a great deal). Scores for each category are totalled. Low scores would be considered a better outcome.
Measured: 37 months
Quality of life - VQoL_C/ YP
measured by the vision related quality of life questionnaire for children and for young people. This questionnaire records how patients feel about their eyesight in relation to the listed statements. Each statement is scored from 1 (not at all true) to 4 (completely true). The score denoting a better outcome is dependent on the question.
Measured: 37 months
Quality of life - VFQ-25
measured by the National Eye Institute Visual Function Questionnaire 25 (VFQ-25). This questionnaire records information in three categories. Questions in the general health and vision category are scored 1-5 or 6 and a low score would be considered a better outcome. Questions in the difficulty with activities category are scored 1 (no difficulty at all) to 6 (stopped doing this for other reasons or not interested in doing this); a low score would be considered a better outcome. Questions in the vision problems category are scored 1 (all of the time) to 5 (none of the time); a high score would be considered a better outcome.
Measured: 37 months
Pancreatic beta cell reserve - glycated haemoglobin or equivalent
measured by percentage glycated haemoglobin or equivalent
Measured: 37 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start28 Dec 2018
- Primary completion15 Nov 2024
- Study completion15 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Wolfram SyndromeStudies 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 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Birmingham
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Timothy Barrett, PhD, MB, BS, Principal investigator, University of Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03717909
- Sponsor's protocol ID
- RG_16_211
- Status last verified
- Apr 2026
- First submitted
- 11 Oct 2018
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No prior plans. CRCTU's data sharing policy - available here https://www.birmingham.ac.uk/research/crctu/data-sharing-policy.aspx - to be followed in the event of any plan to share.
Publications
Dias RP, Brock K, Hu K, Gupta R, Martin U, Peet A, Wilson M, Yu-Wai-Man P, Wright B, Mollan S, Kulkarni A, Meunier I, Billingham L, Nagy Z, Rose H, Koks S, Zatyka M, Astuti D, Lynch T, Morrison KE, Barton D, Cronier S, Malpass R, Evans R, Malhi A, Takhar P, Lamb A, Esteban-Bueno G, Mlynarski W, Orssaud C, Roubertie A, Homer V, Barrett T. Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial. BMJ Open. 2025 Feb 26;15(2):e091495. doi: 10.1136/bmjopen-2024-091495.
PubMed 40010822
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pivotal, International, Randomised, Double-blind, Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
- ClinicalTrials.gov ID
- NCT03717909
- Acronym / sponsor ID
- RG_16_211
- Conditions
- Wolfram Syndrome
- Keywords
- Wolfram Syndrome, Treat Wolfram, Sodium valproate
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- University of BirminghamUniversity, hospital or other
- Study start
- 28 Dec 2018 (actual)
- Primary completion
- 15 Nov 2024 (actual)
- Study completion
- 15 Nov 2024 (actual)
- First posted
- 24 Oct 2018 (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.
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome. 70 paediatric and adult patients were initially planned to be randomised 2:1 to receive either sodium valproate or placebo at 6 international centres. 63 patients were recruited when a decision was made to stop the study recruitment in November 2022.
Detailed description
This phase II clinical trial is planned as a randomised, double-blind, placebo-controlled 3 year intervention Trial in 70 patients with Classical Wolfram Syndrome aged 6 years and over. The primary outcome of the Trial is considered to be clinically relevant and of sufficient magnitude to be beneficial, as a successful Trial outcome will mean that patients will retain a clinically useful degree of visual acuity and it will decline at a slower rate than in the untreated patients. The MRI Pons Volume change has been shown to correlate with changes in the Wolfram Unified Rating Scale.
Patients will be randomised to balance the individual differences across the treatment and placebo groups, therefore reducing the potential for extraneous bias. This will ensure that the treatment effect can be established without the need to account for confounding factors. The value of a placebo arm adds robustness to the Trial by removing the potential for bias from both the investigator and patient perspectives.
Investigators will be blinded to the results of the assessments. Certain assessments will be performed by subspecialists (such as ophthalmologists and neurologists), with the Principal Investigator prevented from having access to the results. This subspecialist-led treatment is in line with the current multi-disciplinary management of these patients and will not result in patients being denied access to effective treatment.
Patients and investigators will be blinded to treatment. The Trial treatment will be a tablet formulation.
This Trial involves 11 clinic visits and 7 follow up telephone calls to reduce the burden of additional travel to the patients.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Experimental Group | Experimental | Drug: Sodium Valproate 200Mg E/C TabletSodium Valproate 200Mg E/C Tablet (active treatment) |
| Control Group | Placebo comparator | Drug: Sodium Valproate matched placeboSodium Valproate matched placebo (inactive treatment) |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (31 listed)
- The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
- AND B) Documented functionally relevant mutations on one or both alleles of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
- The patient is aged 6 years or older and weighing at least 20kg.
- The patient's visual acuity assessed as either the right eye or left eye having a LogMAR score of 1.6 or better on an ETDRS chart, with or without corrected vision.
- Written informed consent (and assent as required).
Key exclusion criteria (10 listed)
- The patient has clinically significant non-Wolfram related CNS involvement which is judged by the Investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
- The patient has a diagnosis of a mitochondrial myopathy
- The patient has active liver disease, has a personal or family history of liver dysfunction related to known genetic disorders, or patient has porphyria.
- The patient has received treatment with any investigational drug within the 30 days prior to Trial entry.
- The patient is currently taking sodium valproate; or has a known hypersensitivity to sodium valproate or its excipients.
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 |
|---|---|---|
| Sodium Valproate 200Mg E/C TabletAlso: Sodium ValproateTreatment with twice-daily oral tablet(s) | Drug | Experimental Group |
| Sodium Valproate matched placeboAlso: PlaceboTreatment with twice-daily oral 200mg tablet(s) | Drug | Control Group |
Primary outcome
Visual acuity (VA)
Time frame: 36 months
Visual acuity (VA) is measured on the logMAR scale by sight tests in clinic using Early treatment diabetic retinopathy study (ETDRS) charts. Values are taken for each eye separately, both uncorrected, and corrected with glasses or contact lenses, and can range from 0, which represents perfect vision i.e. 20/20 (values of -0.1 and -0.2 are also possible representing better than perfect vision), to +2 which represents near blindness i.e. 20/2000. Increases in logMAR represent deterioration.
25 secondary outcomes
Safety - adverse events
Time frame: 37 months
Tolerability - highest treatment dose
Time frame: 36 months
Tolerability - duration of treatment
Time frame: 36 months
Tolerability - dosing modifcation
Time frame: 36 months
Pons Volume
Time frame: 37 months (+/- 6 months)
Brainstem volume
Time frame: 37 months (+/- 6 months)
Retinal nerve thickness
Time frame: 37 months
Colour vision
Time frame: 37 months
Visual fields
Time frame: 37 months
Data on cataracts
Time frame: 37 months
Afferent pupillary defects
Time frame: 37 months
Strabismus
Time frame: 37 months
Nystagmus
Time frame: 37 months
Visual evoked potentials
Time frame: 37 months
Sleep - sleeping habits parent report for patients under 18 years
Time frame: 37 months
Sleep - sleeping habits, self-report
Time frame: 37 months
Balance
Time frame: 37 months
Hearing
Time frame: 37 months
Wolfram Unified Rating Scale
Time frame: 37 months
Mood
Time frame: 36 months
Quality of life - PedsQL
Time frame: 37 months
Quality of life - ICIQ-FLUTS
Time frame: 37 months
Quality of life - VQoL_C/ YP
Time frame: 37 months
Quality of life - VFQ-25
Time frame: 37 months
Pancreatic beta cell reserve - glycated haemoglobin or equivalent
Time frame: 37 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- Derived from this study: PubMed 40010822Dias RP, Brock K, Hu K, Gupta R, Martin U, Peet A, Wilson M, Yu-Wai-Man P, Wright B, Mollan S, Kulkarni A, Meunier I, Billingham L, Nagy Z, Rose H, Koks S, Zatyka M, Astuti D, Lynch T, Morrison KE, Barton D, Cronier S, Malpass R, Evans R, Malhi A, Takhar P, Lamb A, Esteban-Bueno G, Mlynarski W, Orssaud C, Roubertie A, Homer V, Barrett T. Sodium valproate, a potential repurposed treatment for the neurodegeneration in Wolfram syndrome (TREATWOLFRAM): trial protocol for a pivotal multicentre, randomised double-blind controlled trial. BMJ Open. 2025 Feb 26;15(2):e091495. doi: 10.1136/bmjopen-2024-091495.
- Individual participant dataSharing planned: No. No prior plans. CRCTU's data sharing policy - available here https://www.birmingham.ac.uk/research/crctu/data-sharing-policy.aspx - to be followed in the event of any plan to share.
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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. This study is not recruiting at the moment.
Timothy Barrett, PhD, MB, BS principal investigator
University of Birmingham
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Sodium Valproate 200Mg E/C Tablet in people with Wolfram Syndrome, run by University of Birmingham at 6 sites in 4 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 31 inclusion and 10 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: finished, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France, United Kingdom, Poland, Spain. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Visual acuity (VA) (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03717909, which also lists 1 linked publication.
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 5 May 2026; AzTrials last refreshed its copy on 8 Nov 2026.