Neuroinflammation in Amyotrophic Lateral Sclerosis - Mechanisms and Therapeutic Perspectives: a Translational Pilot Study Among ALS Patients

NCT02424669 | Unknown

• 2 other identifiers: 2014-19RCAPHM15_0132
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

Amyotrophic Lateral Sclerosis (ALS) is the most common motor neuron diseases. It is considered as a rare disease with a prevalence of about 8 per 100,000 persons. Initiating in mid-life by progressive paralysis, it evolves rapidly into a generalized muscle wasting that leads irrevocably to death within 2 or 5 years of clinical onset. Since there is no cure for ALS, the management of the disease is supportive and palliative. Riluzole is the only drug that has been shown to extend survival by about three months. The identification of biomarkers sensitive to the progression of the disease might enhance the diagnostic and provide new drug targets. Dysfunction of the immune system is a pathological hallmark of ALS. Increased levels of interferon gamma (IFNgamma) were found in the serum and cerebrospinal fluid (CSF) of ALS patients. However, the cell origin as well as the pathogenic influence of this peripheral source of IFNg is unknown. Thus, IFNgamma might have a role in the pathogenic process of ALS and might be a potential biomarker of the disease.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (1)

• ALS Functional rating Scale-revised (ALS FRS-R) score

  12 months

Study Arms (3)

recently diagnosed ALS patients

EXPERIMENTAL

Other: ALS Functional rating Scale-revised (ALS FRS-R); Other: slow vital capacity; Other: Blood sample; Other: Cerebrospinal Fluid (CSF) sample

not recently diagnosed ALS patients

EXPERIMENTAL

Other: ALS Functional rating Scale-revised (ALS FRS-R); Other: slow vital capacity; Other: Blood sample; Other: Cerebrospinal Fluid (CSF) sample

patients with peripheral neuropathy, recently diagnosed

ACTIVE COMPARATOR

Other: Blood sample

Interventions

ALS Functional rating Scale-revised (ALS FRS-R) OTHER

not recently diagnosed ALS patients; recently diagnosed ALS patients

slow vital capacity OTHER

not recently diagnosed ALS patients; recently diagnosed ALS patients

Blood sample OTHER

not recently diagnosed ALS patients; patients with peripheral neuropathy, recently diagnosed; recently diagnosed ALS patients

Cerebrospinal Fluid (CSF) sample OTHER

not recently diagnosed ALS patients; recently diagnosed ALS patients

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Group 1 and Group 2:
• with sporadic ALS (without family history), recently diagnosed (onset of first symptoms \< 24 months) group 1, not recently diagnosed (onset of first symptoms \> 24 months) group 2
• Who meet the laboratory-supported probable, probable or definite form of ALS according to the El Escorial criteria
• Suffering from the spinal form of ALS
• Group 3:
• \- with an inflammatory peripheral neuropathy, or a non inflammatory peripheral neuropathy, recently diagnosed

You may not qualify if:

• Familial form of ALS
• Bulbar form and respiratory onset form of ALS
• Subjects with a clinically significant history of unstable or severe cardiac, oncologic, hepatic or renal disease, or other medically significant illness.
• Subjects with significant cognitive impairment, clinical dementia, or psychiatric illness.
• Female of childbearing potential (apart of patient using adequate contraceptive measures), pregnant or breast feeding
• Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom follow-up evaluation is not assured)
• Participation in any other clinical study within 30 days prior to the Screening Visit
• Persons deprived of freedom by judicial or administrative decision, hospitalized without their consent or for other reasons than the research, under legal protection or unable to express their consent

Sponsors & Collaborators

• Assistance Publique Hopitaux De Marseille: lead

Study Sites (1)

Assistance Publique Hôpitaux de Marseille

Marseille, 13005, France

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Blood Specimen Collection

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Specimen Handling; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Punctures; Surgical Procedures, Operative; Investigative Techniques

Study Officials

• Urielle Desalbres

  Assistance Publique Hôpitaux de Marseille

  STUDY DIRECTOR

Central Study Contacts

Joelle Micallef

CONTACT

joelle.micallef@ap-hm.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 20, 2015
• First Posted: April 23, 2015
• Study Start: May 1, 2015
• Primary Completion: November 1, 2017
• Study Completion: November 1, 2018
• Last Updated: November 17, 2015

Record last verified: 2015-11

Locations

FR (1)