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Study on the Effects of Sodium Valproate for Hearing Preservation in Patients with Wolfram Syndrome

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What is this study about?

This clinical trial is focused on studying Wolfram syndrome, a rare genetic disorder that affects various parts of the body, including the eyes, ears, and pancreas. The study will use a medication called sodium valproate, which is commonly used to treat seizures. The purpose of the study is to see if sodium valproate can help preserve hearing in patients with Wolfram syndrome.

Participants in the study will take sodium valproate daily. The study will last for three years, during which time participants will have regular check-ups to monitor their hearing and overall health. The goal is to maintain hearing levels and prevent further hearing loss. The study will also look at other aspects of health, such as vision, balance, and sleep, to see if there are any additional benefits from the treatment.

Throughout the study, participants will undergo various tests, including hearing tests and possibly MRI scans, to assess the effects of the treatment. The safety of sodium valproate will be closely monitored, and any side effects will be recorded. This study aims to provide valuable information on whether sodium valproate can be an effective treatment for preserving hearing in individuals with Wolfram syndrome.

1 joining the study

Upon joining the study, a diagnosis of Wolfram syndrome is confirmed. This includes documented diabetes or optic atrophy diagnosed before the age of 16, and relevant genetic mutations.

A baseline assessment is conducted, including a blood pregnancy test for women of childbearing potential.

2 medication administration

The medication Depakine Chrono 500 mg is administered orally as a prolonged-release tablet. The dosage is adjusted to maintain a plasma level between 40 and 100 mg/l.

Women of childbearing potential must use effective contraception during the treatment and for at least 90 days after the last dose.

3 regular assessments

Hearing is assessed regularly to ensure no significant decrease in auditory function. This involves audiometry tests.

Visual acuity and retinal nerve thickness are measured at baseline, every six months during the first year, and at the final visit.

4 safety monitoring

Safety is monitored through the incidence of adverse events and changes in laboratory safety assessments and vital signs.

Patients are evaluated for any changes in insulin or desmopressin requirements.

5 additional evaluations

Balance is assessed using the Mini-BESTest at baseline, at week 52, and at the final visit.

Sleep patterns are evaluated using specific questionnaires, with different versions for patients under 18 and adults.

6 study completion

The study is expected to conclude by December 31, 2027. Final assessments are conducted to evaluate the overall efficacy and safety of the treatment.

Who Can Join the Study?

  • The patient must have a confirmed diagnosis of Wolfram syndrome. This means they have either diabetes diagnosed before age 16 or optic atrophy (a condition affecting the eyes) diagnosed before age 16, along with specific genetic mutations.
  • The patient must have sensorineural hearing loss of at least 20 decibels (dB) at a frequency of 8 kilohertz (kHz). Sensorineural hearing loss is a type of hearing loss that occurs due to problems in the inner ear or the nerve pathways from the inner ear to the brain.
  • The patient must be 13 years of age or older and weigh more than 37.5 kilograms (about 82.7 pounds).
  • The patient must provide written informed consent to participate in the study. This means they agree to join the study after being fully informed about it.
  • Women who can become pregnant must use effective birth control throughout the study and for at least 90 days after the last dose of the study medication. They will receive information on preventing pregnancy and may be referred for advice on contraception if needed.
  • Women who can become pregnant must have a negative blood pregnancy test before starting the study medication and agree to repeat pregnancy tests at each study visit.
  • Sexually active male patients with a female partner who can become pregnant must agree to use condoms and ensure their partner uses an effective method of contraception.
  • The patient must be willing and able to attend all study visits as required by the study protocol.
  • Patients with active hearing implants that contain a magnetic system can participate in the study, but they will not have MRI scans during the study.

Who Cannot Join the Study?

  • Patients who do not have Wolfram syndrome. This is a rare genetic disorder that affects various parts of the body, including hearing.
  • Patients who do not have a hearing loss of at least 20 dB at 8 kHz. This means their hearing is not reduced enough at this specific frequency.
  • Patients who cannot maintain a plasma level of the medication between 40 and 100 mg/l. Plasma level refers to the amount of medication in the blood.
  • Patients who are not within the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not willing or able to follow the study procedures and requirements.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hôpital Européen Georges-Pompidou Paris France

Other Sites

Site Name City Country Status
Unidad de Gestión Clínica Almería Periferia La Cañada Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not recruiting
26.11.2021
Spain Spain
Not recruiting
26.11.2021

Trial locations

Investigated drugs:

Sodium Valproate is being studied for its potential to help preserve hearing in patients with Wolfram syndrome. This medication is administered daily and is being tested to see if it can prevent a significant decrease in hearing ability over a period of three years. The goal is to maintain hearing levels in patients who already have some hearing loss due to this condition.

Investigated diseases:

Wolfram Syndrome – Wolfram Syndrome is a rare genetic disorder that typically begins in childhood. It is characterized by a combination of diabetes insipidus, diabetes mellitus, optic atrophy, and deafness. The condition often starts with the onset of diabetes mellitus, followed by progressive vision loss due to optic atrophy. Hearing loss may also develop, and some individuals experience neurological symptoms such as balance issues. As the syndrome progresses, additional complications can arise, affecting various body systems. The progression and severity of symptoms can vary widely among individuals with Wolfram Syndrome.

Trial ID:
2024-514896-17-00
Protocol code:
2020-004594-43
NCT ID:
NCT04940572
Trial Phase:
Therapeutic exploratory (Phase II)

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