Eight At One Stroke: Attention Gangliosidoses A Registry Study for Patients With Gangliosidoses

Status: Unknown
Location: See location...
Study Type: Observational
SUMMARY

The clinical project Eight At One Stroke: Attention Gangliosidoses represents a clinical registry for recording the clinical manifestation and the disease progression of gangliosidoses. The intention of this project is to better understand the manifestation and progression of gangliosidoses and to raise awareness of these disorders in the public health service. The patients or their families, respectively, will be integrated in the study in order to measure Patient Outcome and to objectify the psychosocial burden for the patient and his family. The study has a retrospective and a prospective part. It is planned to transfer the data of the study into a continuous registry.

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Biochemically and/or genetically affirmed diagnosis of a gangliosidosis

• The patient or respectively the parents or the caregiver (for children or older underage patients) have given written informed consent

Locations
Other Locations
Germany
SphinCS Lyso gemeinnützige UG (haftungsbeschränkt)
RECRUITING
Hochheim Am Main
Contact Information
Primary
Eugen Mengel, MD
info@sphincs.de
0496146904820
Backup
Yasmina Amraoui, MD
info@sphincs.de
0496146904820
Time Frame
Start Date: 2020-06-08
Completion Date: 2025-06-07
Participants
Target number of participants: 40
Treatments
GM1-Gangliosidosis - Sialidosis
Confirmed diagnosis of:~* GM1-Gangliosidosis Morquio B Variant~* Sialidosis~* Galactosialidosis
GM2-Gangliosidoses
Confirmed diagnosis of:~* Tay-Sachs Disease, incl. B1-Variante~* Sandhoff Disease~* GM2-Activator-Deficiency
Sponsors
Leads: SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)

This content was sourced from clinicaltrials.gov

Similar Clinical Trials