EUCTR2020-004594-43-FR进行中（未招募）1 期
AUDIOWOLF: A phase II, open-label, efficacy study of daily administration of sodium valproate in patients clinically affected by Wolfram syndrome due to monogenic mutation - AUDIOWOLF
CECS/I-Stem0 个研究点目标入组 23 人开始时间: 2021年3月11日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 23
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Patients must meet all of the following criteria to be eligible for enrolment:
- •1. The patient has a definite diagnosis of Wolfram syndrome, as determined by the following:
- •a. Documented diabetes mellitus diagnosed under 16 completed years according to WHO or ADA criteria OR 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
- •2. The patient has sensorineural hearing loss of at least 20 db at 8 kHz in HFA
- •3. The patient is 13 years of age or older, and has a body-weight over 37.5 kg
- •4. Written informed consent for the principal study
- •5. 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 .
- •6. 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.
- •7. 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 23
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 11
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from the study for any of the following reasons:
- •1. The patient is unable or unwilling to comply with the protocol requirements
- •2. The patient has received treatment with any investigational drug within the 30 days prior to the screening visit
- •3. The patient is currently taking VPA
- •4. The patient has an history of allergy or hypersensitivity to VPA or its excipients/ingredients
- •5. 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
- •6. 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
- •7. The patient has a contra-indication to VPA: mitochondrial disorders caused by mutations in the nuclear gene encoding the mitochondrial enzyme polymerase ? (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…
- •8. 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
- •9. The patient has a known history of central apnea
- •10. An unwillingness on the part of male patients to use highly effective form of birth control if engaging in sexual intercourse with a woman who could become pregnant from the time of the first dose of study medication until completion of follow-up procedures.
- •11. An unwillingness on the part of female patients to use highly effective form of birth control if engaging in sexual intercourse and to have frequent pregnancy tests during treatment and until completion of follow-up procedures.
- •12. The patient is currently pregnant or breastfeeding
- •13. The patient has swallowing disorders
研究者
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