Clinical trials: Wolfram Syndrome
Wolfram Syndrome
I-Tracking Neurodegeneration in Early Wolfram Syndrome
Wolfram syndrome (WFS; OMIM #222300) is a rare autosomal recessive disease clinically defined in 1938 as the combination of childhood-onset insulin dependent diabetes, optic nerve atrophy, diabetes insipidus and deafness.
- Trial name
- I-TRACK (NCT03951298)
- Where
- United States
- Volunteers
- 128 people
- Sponsor
- Washington University School of Medicine
Wolfram Syndrome, Diabetes Mellitus
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration.
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Trial name
- NCT02829268
- Testing
- dantrolene sodium
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Washington University School of Medicine
Diabetes Mellitus, Iron Metabolism Disorders
Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy
Patients who are genetically diagnosed with the recently reported and rare Wolfram syndrome type 2 ( WFS2) and have the degenerative and symptomatic disease including signs such as diabetes, platelet aggregation defect or visual…
- Conditions
- Diabetes Mellitus, Iron Metabolism Disorders, Gastroduodenal Ulcer, Optic Atrophy
- Trial name
- NCT02882477
- Testing
- Deferiprone, Acetylcysteine, Sitagliptin and Metformin
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Hadassah Medical Organization
Wolfram Syndrome 1, Optic Atrophies, Hereditary
Does Recessive Optic Atrophy Due to WFS1 Exist?
All patients with Wolfram syndrome and recessive optic atrophy due to a mutation of the WFS1 from a single Center were included in a retrospective study.
- Trial name
- NCT07336966
- Testing
- analyse study
- Volunteers
- 45 people
- Sponsor
- Hôpital Necker-Enfants Malades
Type 1 Diabetes, Diabetes Insipidus
Tracking Neurodegeneration in Early Wolfram Syndrome
The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive…
- Conditions
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Trial name
- TRACK (NCT02455414)
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Wolfram Syndrome, Fecal Incontinence
Mechanisms of Fecal Incontinence in Wolfram Syndrome
In order to clarify the mechanism of fecal incontinence in in Wolfram syndrome and to characterize its risk factors, the examiner will reviewed the files of patients followed in a Reference Center for Rare Diseases in…
- Trial name
- NCT07313085
- Testing
- Retrospective case-control analysis
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hôpital Necker-Enfants Malades
Optical Coherence Tomography (OCT) of the Retina, Mutation
Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome
Looking for the pthogenicity of mutations of WFS1 gene for patients with mutation of the two alleles but a dominant phenotype
- Trial name
- VUS in Wolfram (NCT07485413)
- Where
- France
- Volunteers
- 45 people
- Sponsor
- Hôpital Necker-Enfants Malades
Diabetes Mellitus, Type 1, Monogenic Diabetes
Monogenic Diabetes Misdiagnosed as Type 1
The study has two aims: 1. To (1a) determine the frequency of monogenic diabetes misdiagnosed as type 1 diabetes (T1D) and (2) to define an algorithm for case selection. 2.
- Conditions
- Diabetes Mellitus, Type 1, Monogenic Diabetes, Neonatal Diabetes, Maturity-onset Diabetes in the Young (MODY)
- Trial name
- ADDAM (NCT03988764)
- Testing
- None AHT
- Where
- Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Wolfram Syndrome
Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1
Wolfram syndrome (WFS:OMIM 222300) is a group of inherited disorders that usually appear in childhood and cause diabetes, optic atrophy leading to loss of vision, deafness and often diabetes insipidus.
- Trial name
- NCT05659368
- Testing
- Tirzepatide
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Ospedale San Raffaele
Wolfram Syndrome, Diabetes Mellitus
Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study
In this study, the investigators hypothesize that studying monogenic variants with strong effect associated with severe insulin deficiency of Wolfram syndrome will provide important insights into the more complex type 1 and type…
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Deafness
- Trial name
- NCT02841553
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- Washington University School of Medicine
Wolfram Syndrome
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
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)…
- Trial name
- AUDIOWOLF (NCT04940572)
- Testing
- Depakine
- Where
- 2 sites in France, Spain
- Volunteers
- 23 people
- Sponsor
- Centre d'Etude des Cellules Souches
Wolfram Syndrome
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
This trial aims to investigate the efficacy, safety and tolerability of sodium valproate in the treatment of patients with Wolfram syndrome.
- Trial name
- NCT03717909
- Testing
- Sodium Valproate 200Mg E/C Tablet
- Where
- 6 sites in France, Poland and 2 more
- Volunteers
- 63 people
- Sponsor
- University of Birmingham
Diabetes Mellitus Associated With Genetic Syndrome, Wolfram Syndrome
GLP Analogs for Diabetes in Wolfram Syndrome Patients
Wolfram syndrome, also referred to as DIDMOAD (diabetes insipidus, diabetes mellitus, optic atrophy and deafness) is a genetic syndrome characterized by beta-cell dysfunction and apoptosis leading to diabetes, neurodegeneration…
- Trial name
- NCT01302327
- Testing
- Exenatide
- Sponsor
- Hadassah Medical Organization
Wolfram Syndrome
AMX0035 in Adult Patients With Wolfram Syndrome
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
- Trial name
- NCT05676034
- Testing
- AMX0035
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Amylyx Pharmaceuticals Inc.