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PhenotYpe Research for ALS ModIfyer Discovery.


- candidate number14516
- NTR NumberNTR3902
- ISRCTNISRCTN wordt niet meer aangevraagd.
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR13-mrt-2013
- Secondary IDs40-42900-98-1008 ZonMw
- Public TitlePhenotYpe Research for ALS ModIfyer Discovery.
- Scientific TitlePhenotYpe Research for ALS ModIfyer Discovery.
- ACRONYMPYRAMID
- hypothesisFocusing on disease-modifying rather than etiologic mechanisms may accelerate the development of novel therapies and complement current research initiatives on causative mechanisms and curative therapies.
- Healt Condition(s) or Problem(s) studiedAmyotrophic Lateral Sclerosis (ALS)
- Inclusion criteriaAll patients with ALS.
- Exclusion criteriaN/A
- mec approval receivedyes
- multicenter trialyes
- randomisedno
- groupParallel
- TypeSingle arm
- Studytypeobservational
- planned startdate 1-jun-2013
- planned closingdate1-jun-2016
- Target number of participants600
- InterventionsN/A
- Primary outcome1. Report on key molecular drivers in transcriptomic data related to disease progression (month 35);
2. Report on key molecular drivers in methylation data related to disease progression (month 35);
3. Report on key molecular drivers in proteomic data related to disease progression (month 35);
4. Report on longitudinal MRI and MUNIX changes related to disease progression (month 35).
- Secondary outcome1. Tender document for appropriate SMEs that describes most promising therapeutic targets, based on differential co-expression, co-methylation and co-translation reports;
2. Tender document for appropriate SMEs that describes most promising molecular disease progression markers based on those markers that best discriminate between fast and slow progressors.
- TimepointsMonth 35.
- Trial web siteN/A
- statusplanned
- CONTACT FOR PUBLIC QUERIES Petra Berk
- CONTACT for SCIENTIFIC QUERIESMD. PhD. Jan H. Veldink
- Sponsor/Initiator University Medical Center Utrecht (UMCU)
- Funding
(Source(s) of Monetary or Material Support)
E-Rare-2 Call for Proposals 2012
- PublicationsN/A
- Brief summaryAmyotrophic lateral sclerosis is a relentlessly progressive neurodegenerative disorder of motor neurons, leading to death of the patient on average only 36 months after disease onset. ALS is characterized by heterogeneity at the clinical and genetic level. Causative mechanisms of motor neuron degeneration are studied in several European studies, in particular Euro-MOTOR. Notwithstanding these efforts, ALS causative mechanisms remain only partially understood and therapeutic strategies based on these mechanistic insights are largely ineffective. The only drug available- Riluzole- extends the lifespan of ALS by 6 months, presumably by targeting disease modifying rather than disease causing targets. Therefore, we hypothesize that targeting disease modifying factors is important to accelerate the development of novel, alternative therapeutics, that will complement the search for causative mechanisms and therapies already covered in other projects. In this project, using unbiased genetic, proteomic, epigenetic and gene-expression techniques, we aim to identify modifiers of disease progression. We want to exploit the phenotypic heterogeneity of patients by comparing ALS patients with very fast disease progression to patients with slow disease progression. Using this original study design we aim to identify the factors that protect a subset of patients from an aggressive disease. Data collection and sample management will be done according to harmonized European ALS sampling and data management standards developed through the EU funded project SOPHIA. In addition, the detailed prospective clinical monitoring of patients will allow us reliably delineate the parameters that predict fast disease progression already in patients early in the disease. Insight into disease modifying factors will enhance our understanding of motor neuron degeneration and reveal potential therapeutic strategies.
- Main changes (audit trail)
- RECORD13-mrt-2013 - 1-apr-2013


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