The preSPG4 Study - Studying the Prodromal and Early Phase of SPG4
NCT ID: NCT03206190
Last Updated: 2022-08-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
NA
200 participants
INTERVENTIONAL
2018-07-01
2031-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Studying Non-motor Symptoms in HSP
NCT03204773
Spinal Cord Stimulation Therapy for Hereditary Spastic Paraplegias Patients
NCT05196178
The Effects of Spasticity on Glucose Metabolism in Individuals With Spinal Cord Injury
NCT03859960
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
NCT06839469
Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting
NCT02235090
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
PARALLEL
DIAGNOSTIC
TRIPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Mutation carrier
The participants will be tested genetically if they carry a disease causing mutation or not. Depending on their genetic test result they will at the end of the study divided into two groups. The clinician will be blinded throughout the entire study to the genetic results.
SPRS Score and clinical signs
Patients will clinically characterized by using the SPRS Score and the inventory V3
Cognition Testing using CANTAB
Patients will be tested using the CANTAB
Lumbar Puncture and blood draw
Biomaterial will be collected (not obligate) to compare e.g. Nfl levels in serum and CSF
MRI
MRI will be used to reveal presymptomatic brain morphology changes (not obligate)
Electrophysiology
Electrophysiological tests will be used to characterize patients better.
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
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.
Non-mutation carrier
The participants will be tested genetically if they carry a disease causing mutation or not. Depending on their genetic test result they will at the end of the study divided into two groups. The clinician will be blinded throughout the entire study to the genetic results.
SPRS Score and clinical signs
Patients will clinically characterized by using the SPRS Score and the inventory V3
Cognition Testing using CANTAB
Patients will be tested using the CANTAB
Lumbar Puncture and blood draw
Biomaterial will be collected (not obligate) to compare e.g. Nfl levels in serum and CSF
MRI
MRI will be used to reveal presymptomatic brain morphology changes (not obligate)
Electrophysiology
Electrophysiological tests will be used to characterize patients better.
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
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.
Known-mutation carriers but presymptomatic
In a third arm (open arm) we will also include positive predictive tested participants which know that they are carrying a known mutation but are at inclusion into the study asymptomatic (according to the inclusion / exclusion criteria).
SPRS Score and clinical signs
Patients will clinically characterized by using the SPRS Score and the inventory V3
Cognition Testing using CANTAB
Patients will be tested using the CANTAB
Lumbar Puncture and blood draw
Biomaterial will be collected (not obligate) to compare e.g. Nfl levels in serum and CSF
MRI
MRI will be used to reveal presymptomatic brain morphology changes (not obligate)
Electrophysiology
Electrophysiological tests will be used to characterize patients better.
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
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.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
SPRS Score and clinical signs
Patients will clinically characterized by using the SPRS Score and the inventory V3
Cognition Testing using CANTAB
Patients will be tested using the CANTAB
Lumbar Puncture and blood draw
Biomaterial will be collected (not obligate) to compare e.g. Nfl levels in serum and CSF
MRI
MRI will be used to reveal presymptomatic brain morphology changes (not obligate)
Electrophysiology
Electrophysiological tests will be used to characterize patients better.
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
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.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Age 18 to 70 years
* Written, informed consent (patient)
Exclusion Criteria
* Manifest spastic gait (subclinical signs like increased deep tendon reflexes, positive Babinski sign are allowed)
* Participation in interventional trials
18 Years
70 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
University Hospital Tuebingen
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Prof. Dr. Ludger Schöls
Prinicipal Investigator
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Ludger Schöls, Prof.
Role: PRINCIPAL_INVESTIGATOR
Head of Department
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University Hospital Tübingen, Center for Neurology
Tübingen, , Germany
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
References
Explore related publications, articles, or registry entries linked to this study.
Rattay TW, Volker M, Rautenberg M, Kessler C, Wurster I, Winter N, Haack TB, Lindig T, Hengel H, Synofzik M, Schule R, Martus P, Schols L. The prodromal phase of hereditary spastic paraplegia type 4: the preSPG4 cohort study. Brain. 2023 Mar 1;146(3):1093-1102. doi: 10.1093/brain/awac155.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
preSPG4
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.