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Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome

NCT04940572 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2023-11-28

No results posted yet for this study

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).

Conditions

  • Wolfram Syndrome

Interventions

DRUG

Depakine

Refer to arm description.

Sponsors & Collaborators

  • Genethon

    collaborator OTHER

  • Centre d'Etude des Cellules Souches

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-26
Primary Completion
2025-11-01
Completion
2025-12-01

Countries

  • France
  • Spain

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04940572 on ClinicalTrials.gov