An investigator-initiated prospective, longitudinal, single center, observational study to investigate biomarkers of TDP-43 and oxidized lipid metabolism in Amyotrophic Lateral Sclerosis (ALS).

• Conditions: ALS; Lou Gehrig Disease; 10029317

• Registration Number: NL-OMON56755
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 18 June 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 70

Inclusion Criteria

- Signed informed consent
- Age above 18 years
- Diagnosis ALS

Exclusion Criteria

- Fulfilling the criteria for respiratory insufficiency (non-invasive
ventilation use >23 hours per day for 10 consecutive days or having a
tracheostomy)
- MiToS stage 2, 3 or 4
- confirmed FUS or SOD1 mutation
- Contraindication to lumbar puncture
- Any of the following medically significant conditions:
1. Neurological impairment/dysfunction or unstable psychiatric illness that in
the investigator*s opinion is likely to interfere with assessment of ALS
disease progression.
2. Clinically significant unstable medical condition other than ALS, which
would present a risk to a patient to participate in the study
3. Presence of dementia that impairs the ability of the subject to provide
informed consent, according to the PI decision.
4. Cancer that is currently under active treatment or is likely to require
treatment during the study that may alter the subject*s function and thereby
interfere with assessment of ALS disease progression.
5. Any other condition that in the investigator*s opinion would present a risk
to a patient to participate in the study, interfere with the assessment of
safety or has an increased risk of causing death during the study.

Study & Design

• Study Type: Observational invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Biomarker levels of oxidized lipid metabolism and TDP-43 pathology in blood at<br /><br>time 0, 4,8 and 12 months and CSF at time 0 and 12 months, and changes of<br /><br>biomarker levels of oxidized lipid metabolism and TDP-43 over time.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Association of biomarker levels with phenotypical characteristics over time,<br /><br>e.g. time from onset of symptoms, ALSFRS-R, MiToS stage, site of onset<br /><br>(Limb/Bulbar), respiratory function, time to ventilation/death. </p><br>