Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Synd… (NCT03717909) | Clinical Trial Compass
CompletedPhase 2
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
France, Poland, Spain63 participantsStarted 2018-12-28
Plain-language 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. 63 patients were recruited when a decision was made to stop the study recruitment in November 2022.
Who can participate
Age range6 Years
SexALL
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Inclusion criteria
✓. The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
✓. 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).
✓. 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
✓. Contraception methods that in the context of this guidance are considered to have low user dependency.
Exclusion criteria
✕. 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.
✕. 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.