Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches

New study seeks biological clues for ALS and FTD diagnosis

NCT: NCT06856850 · Status: RECRUITING · Phase: N/A · Sponsor: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · Started: 2025-02-27 · Est. Completion: 2026-08

Plain English Summary

Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches is a Not Applicable clinical trial sponsored by Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta studying ALS (Amyotrophic Lateral Sclerosis), FTD, Neuropathic, Psychiatric Disorders, Idiopathic Intracranial Hypertension, Frontotemporal Dementia (FTD). This study is looking for biological markers (biosignatures) in easily accessible tissues to better diagnose and understand Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). It is for individuals diagnosed with ALS or FTD, aiming to improve diagnostic accuracy beyond current clinical assessments. Participation involves providing biological samples like skin, tears, or cerebrospinal fluid for advanced analysis. Currently, diagnosis relies heavily on symptoms; this research seeks to add biological insights to improve precision. The trial aims to enroll 230 participants.

Official Summary

Diagnosis of ALS/FTD disease spectrum is challenging because it largely relies on clinical symptoms. Identifying novel biomarkers is essential for a paradigm shift towards a more precise biological-based diagnosis. To achieve this aim, having access to proper specimens and analytical methods is crucial. Our team of experts in neurology, biology, chemistry, physics, and AI will explore ALS/FTD from novel perspectives using transcriptomics, proteomics, genomics and other innovative approaches to analyzing easily accessible tissues. The seed amplification assay (SAA) will be also exploited to detect pathological TDP-43. This project aims to create disease fingerprints useful for patient stratification and monitoring of disease progression, and to evaluate the therapeutic efficacy in clinical trials, thus overcoming the limits of clinical interpretation. Discovering new biomarkers and cellular pathways will improve the diagnosis and treatment of these devastating diseases.

Who Can Participate

Here is what you need to know about eligibility for this trial. Individuals with a confirmed diagnosis of ALS or FTD based on established clinical criteria can participate. The study does not list specific age ranges or health requirements beyond the primary diagnosis. Individuals not meeting the clinical criteria for ALS or FTD will not be eligible. This trial is studying ALS (Amyotrophic Lateral Sclerosis), FTD, Neuropathic, Psychiatric Disorders, Idiopathic Intracranial Hypertension, Frontotemporal Dementia (FTD), so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how accurately a new test can find a specific abnormal protein (TDP-43) in various body fluids and tissues, which could lead to a more precise diagnosis. The specific primary outcome measures are: Evaluation of SAA accuracy in detecting misfolded TDP-43 in CSF, skin, OM, and tears of ALS and FTD patients. (24 months). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This study does not have a traditional clinical phase designation. It may be an observational study that follows patients without intervening in their care, an expanded access or compassionate use program, or other non-interventional research. These studies contribute valuable data about disease progression, treatment patterns, and patient outcomes.

Why This Trial Matters

This trial matters because it aims to find new biological markers to improve the diagnosis and treatment of ALS and FTD, which currently rely heavily on clinical symptoms. This research targets ALS (Amyotrophic Lateral Sclerosis), FTD, Neuropathic, Psychiatric Disorders, Idiopathic Intracranial Hypertension, Frontotemporal Dementia (FTD), where improved treatment options are needed.

Investor Insight

This observational study focuses on biomarker discovery, which is a crucial first step for developing new diagnostic tools and potential therapies in a significant market for neurodegenerative disease

Is This Trial Right for Me?

Ask your doctor if your diagnosis meets the specific clinical criteria used in this study. Understand what types of biological samples will be collected and how often. Inquire about the potential benefits and risks of providing these samples. This trial is currently recruiting participants. The trial is being conducted at 4 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Inclusion Criteria:

* Clinical criteria for ALS (Brooks et al., 2000; de Carvalho M., 2008), FTD (GornoTempini et al., 2011; Rascovsky et al., 2011)

Exclusion Criteria:

* na

Trial Locations

Frequently Asked Questions

What is clinical trial NCT06856850?

NCT06856850 is a Not Applicable OBSERVATIONAL study titled "Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches." It is currently recruiting and is sponsored by Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta. The trial targets enrollment of 230 participants.

What conditions does NCT06856850 study?

This trial investigates treatments for ALS (Amyotrophic Lateral Sclerosis), FTD, Neuropathic, Psychiatric Disorders, Idiopathic Intracranial Hypertension, Frontotemporal Dementia (FTD). The primary condition under study is ALS (Amyotrophic Lateral Sclerosis).

What does Not Applicable mean for NCT06856850?

This study does not have a defined clinical phase. It may be an observational study, expanded access program, or other non-interventional research.

What is the current status of NCT06856850?

This trial is currently "Recruiting." It started on 2025-02-27. The estimated completion date is 2026-08.

Who is sponsoring NCT06856850?

NCT06856850 is sponsored by Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT06856850?

The trial aims to enroll 230 participants. The trial is currently recruiting and accepting new participants.

How is NCT06856850 designed?

This is a observational study.

What are the primary outcomes being measured in NCT06856850?

The primary outcome measures are: Evaluation of SAA accuracy in detecting misfolded TDP-43 in CSF, skin, OM, and tears of ALS and FTD patients. (24 months). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT06856850 being conducted?

This trial is being conducted at 4 sites, including Milan; Naples; Sassari; Sesto Fiorentino (Italy).

Where can I find official information about NCT06856850?

The official record for NCT06856850 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT06856850. This government database provides the most up-to-date and detailed information about the trial.

What is NCT06856850 testing in simple terms?

This study is looking for biological markers (biosignatures) in easily accessible tissues to better diagnose and understand Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). It is for individuals diagnosed with ALS or FTD, aiming to improve diagnostic accuracy beyond current clinical assessments.

Why is this trial significant?

This trial matters because it aims to find new biological markers to improve the diagnosis and treatment of ALS and FTD, which currently rely heavily on clinical symptoms.

What are the potential risks of participating in NCT06856850?

Potential risks are generally associated with sample collection, such as discomfort or minor infection from needle sticks for CSF or skin biopsies. The accuracy of the new diagnostic tests being evaluated is still under investigation. There is a possibility that the study may not find the expected biomarkers. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT06856850?

Ask your doctor if your diagnosis meets the specific clinical criteria used in this study. Understand what types of biological samples will be collected and how often. Inquire about the potential benefits and risks of providing these samples. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT06856850 signal from an investment perspective?

This observational study focuses on biomarker discovery, which is a crucial first step for developing new diagnostic tools and potential therapies in a significant market for neurodegenerative disease This is a Not Applicable trial, which is in early development stages.

What happens if the treatment in this trial doesn't work?

Participation involves providing biological samples like skin, tears, or cerebrospinal fluid for advanced analysis. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.