NCT03206190

The preSPG4 Study - Studying the Prodromal and Early Phase of SPG4

Studying the Prodromal and Early Phase of Hereditary Spastic Paraplegia Type 4 (SPG4)

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: University Hospital Tuebingen · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

Study goals 1. Prospective longitudinal data on progression in the natural course of SPG4 in presymptomatic mutation carriers prior to clinical disease onset and in early stages of disease 2. Biomarkers providing objective measures of disease activity

Conditions

• Hereditary Spastic Paraplegia
• Hereditary, Spastic Paraplegia, Autosomal Dominant

Interventions

Type	Name	Description
OTHER	SPRS Score and clinical signs	Patients will clinically characterized by using the SPRS Score and the inventory V3
BEHAVIORAL	Cognition Testing using CANTAB	Patients will be tested using the CANTAB
DIAGNOSTIC_TEST	Lumbar Puncture and blood draw	Biomaterial will be collected (not obligate) to compare e.g. Nfl levels in serum and CSF
DIAGNOSTIC_TEST	MRI	MRI will be used to reveal presymptomatic brain morphology changes (not obligate)
DIAGNOSTIC_TEST	Electrophysiology	Electrophysiological tests will be used to characterize patients better.
DIAGNOSTIC_TEST	Testing functional performance	By using the 3 minute walk, 5 stair-climb test, and 10m walking test we will try to identify and measure subclinical progression prior to disease onset
DIAGNOSTIC_TEST	Non motor symptoms	By using a number of different tests we try to identify other non-motor symptoms which might manifest prior to disease onset.

Timeline

• Start date: 2018-07-01
• Primary completion: 2029-12-01
• Completion: 2031-12-01
• First posted: 2017-07-02
• Last updated: 2022-08-23

Locations

1 site across 1 country: Germany