Amyotrophic Lateral Sclerosis Clinical Trial
— PREV-DEMALSOfficial title:
Predict to Prevent Frontotemporal Lobar Degeneration and Amyotrophic Lateral Sclerosis
Verified date | August 2016 |
Source | Assistance Publique - Hôpitaux de Paris |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The project focuses on C9orf72, the most frequent genetic form of frontotemporal dementias (FTD, or frontotemporal lobar degeneration, FTLD) and amyotrophic lateral sclerosis (ALS). FTD is the second commonest cause of degenerative dementia in presenium after Alzheimer's disease. Behavioural and cognitive impairments progressively lead to dementia. ALS produces progressive muscle weakness leading to the death in 2 to 4 years. Since 2006, major discoveries have linked FTLD and ALS: 1. TDP-43 aggregates in neurons and 2. C9orf72 mutations is a major genetic cause in both disorders. Two major pathological subtypes are now defined in FTD, FTD-TDP and FTD-TAU. C9orf72 mutations (associated to FTD-TDP) are the most frequent genetic causes of FTD (15%), FTD-ALS (65%) and ALS (40%). FTD is difficult at an early stage; and no clinical, biological or imaging features can predict the underlying pathology in living patients. Therapeutic perspectives emerged against tau aggregation, progranulin deficit and C9orf72 expansion (antisense). Presymptomatic carriers of genetic FTD would benefit, before onset of symptoms, from these therapeutic that would delay or prevent the disease. At this step, it becomes crucial to develop markers to know how many years before symptoms, does the pathological progress begin, to treat the patients at the most early stage of the disease. Markers are also needed to predict the pathology (FTD-TDP/FTD-tau) in patients that will be eligible for trials targeting specific pathological lesion.
Status | Completed |
Enrollment | 100 |
Est. completion date | October 27, 2020 |
Est. primary completion date | October 27, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Age = 18 - Signed informed consent for genetic and clinical study - To be carrier of a C9ORF72 mutation - Diagnosis criteria of FTD or ALS - To be French-speaking - To be affiliated to the social security scheme - Absence of another intercurrent neurological pathology (vascular cerebral accident, tumor, etc.....) Inclusion criteria for asymptomatic relatives - Age = 18 - To be first degree relative of a person carrying a C9ORF72 mutation OR first degree relative of FTD or ALS deceased patient whose C9ORF72 mutation as been identified in the family - Signed informed consent for genetic and clinical study - To be French-speaking - To be affiliated to the social security scheme - Absence of proven neurologic disorders or an intercurrent neurological pathology (vascular cerebral accident, tumor, etc.....) Exclusion Criteria: - Contra-indication to perform a brain MRI (wearing of pacemaker, cardiac valve or incompatible vascular MRI surgical equipment , neurosurgery or surgery vascular equipment, surgical equipment likely to concentrate the radio frequency field, intra ocular or intra cerebral metal foreign body, claustrophobia, wearing a non-compliant radio intrauterine device ), - Inability to lie one hour without moving - PET-FDG contra-indication - Breastfeeding and pregnant women - Human chorionic gonadotrophin (Bétâ-HCG) positive determination or Positive urine pregnancy test for women of childbearing age Exclusion criteria for related asymptomatic : - Clinical proven signs of FTD, language disorder, praxis disorder, mnemic, of parkinson's syndrome or amyotrophic lateral sclerosis. - Counter-indication to perform a brain MRI (wearing of pacemaker, cardiac valve or incompatible vascular MRI surgical equipment , neurosurgery or surgery vascular equipment, surgical equipment likely to concentrate the radio frequency field, intra ocular or intra cerebral metal foreign body, claustrophobia, wearing a non-compliant radio intrauterine device ) - Severe chronic alcoholism - PET-FDG contre-indication - Inability to lie one hour without moving - Bétâ-HCG positive determination or Positive urine pregnancy test for women of childbearing age |
Country | Name | City | State |
---|---|---|---|
France | Groupe Hospitalier Pitié-Salpêtrière - Charles Foix | Paris |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique - Hôpitaux de Paris |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in behavioral assessment (20 scales) | at baseline, 16 weeks and 32weeks | ||
Primary | Change in MRI morphological criteria | at baseline, 16 weeks and 32weeks | ||
Primary | Change in cerebral perfusion by SPECT / PET | at baseline, 16 weeks and 32weeks | ||
Secondary | correlations between behavioral assessment, MRI morphological criteria, Cerebral perfusion and metabolism by SPECT / PET and transcriptome analysis | 32 weeks |
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