Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome (NCT04940572) | Clinical Trial Compass
UnknownPhase 2
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
France, Spain23 participantsStarted 2021-11-26
Plain-language summary
Open label, phase II study non randomized single group assignment of 20 evaluable patients 13 years and older, over 37,5 kg body-weight, with sensorineural hearing loss of at least 20 dB at 8 kHz in high frequency average (HFA), and with documented genetic mutations in the WFS1 gene and with at least one other major documented clinical symptom pertaining to Wolfram syndrome (i.e. diabetes mellitus, diabetes insipidus, optic atrophy). Every patients will receive over three years a treatment by VPA (Depakine chrono).
Who can participate
Age range13 Years
SexALL
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Inclusion criteria
✓. The patient has a definite diagnosis of Wolfram syndrome, as determined by the following:
✓. The patient has sensorineural hearing loss of at least 20 db at 8 kHz in HFA
✓. The patient is 13 years of age or older, and has a body-weight over 37.5 kg
✓. Written informed consent for the principal study
✓. Women of childbearing potential who are prescribed with sodium valproate must use effective contraception without interruption during the entire duration of treatment and at least 90 days after last administration . These patients will be provided with comprehensive information on pregnancy prevention and will be referred for contraceptive advice if they are not using effective contraception. At least one effective method of contraception (preferably a user independent form such as an intra-uterine device or implant) or two complementary forms of contraception including a barrier method should be used.
✓. Women with childbearing potential are required to have a confirmed negative blood pregnancy test before starting medication administration at baseline. Women with childbearing potential agree to repeat blood pregnancy tests during at each study visit.
✓. Sexually active men with a female partner of childbearing potential must agree to the use of condoms and the use of a effective method of contraception by the female partner.
✓. Patient willing and able to meet all protocol defined visits for the duration of the Trial.
Exclusion criteria
✕. The patient is unable or unwilling to comply with the protocol requirements
✕. The patient has received treatment with any investigational drug within the 30 days prior to the screening visit
✕. The patient is currently taking VPA
✕. The patient has an history of allergy or hypersensitivity to VPA or its excipients/ingredients
✕. The patient is known to be affected by a pathology for which the symptoms or associated treatments can alter the hearing function and/or affect the ear
✕. 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 contra-indication to VPA: mitochondrial disorders caused by mutations in the nuclear gene encoding the mitochondrial enzyme polymerase γ (POLG), e.g. Alpers-Huttenlocher Syndrome, active liver disease, personal or family history of liver dysfunction related to known genetic disorders, porphyria, lactose intolerance, the Lapp lactase deficiency, glucose- galactose malabsorption, urea cycle disorders…
✕. 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 participation