Trial reportNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2014
Results from a pilot study on amiodarone administration in monogenic frontotemporal dementia with granulin mutation.
Trial report in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
16 citing papers in PubMed, 33 citations in OpenAlex.
- Targeting Granulin Haploinsufficiency in Frontotemporal Dementia: From Genetic Mechanisms to Therapeutics.International journal of molecular sciences · 2025Review
- The role of endolysosomal progranulin and TMEM106B in neurodegenerative diseases.Molecular neurodegeneration · 2025Review
- Progranulin's Protective Mechanisms and Therapeutic Potential in Cardiovascular Disease.Cells · 2025Review
- Benzoxazole-derivatives enhance progranulin expression and reverse the aberrant lysosomal proteome caused by GRN haploinsufficiency.Nature communications · 2024Article
- Pharmacotherapy for Frontotemporal Dementia.CNS drugs · 2021Review
- Development of disease-modifying drugs for frontotemporal dementia spectrum disorders.Nature reviews. Neurology · 2020Review
- Neuroinflammation in frontotemporal dementia.Nature reviews. Neurology · 2019Review
- Age- and stress-associated C. elegans granulins impair lysosomal function and induce a compensatory HLH-30/TFEB transcriptional response.PLoS genetics · 2019Article
- Thalamo-cortical network hyperconnectivity in preclinical progranulin mutation carriers.NeuroImage. Clinical · 2019Article
- Precision Medicine for Frontotemporal Dementia.Frontiers in psychiatry · 2019Review
- Progranulin: Functions and neurologic correlations.Neurology · 2018Article
- An 8-week, open-label, dose-finding study of nimodipine for the treatment of progranulin insufficiency fromAlzheimer's & dementia (New York, N. Y.) · 2017Article
- Therapy and clinical trials in frontotemporal dementia: past, present, and future.Journal of neurochemistry · 2016Review
- Progranulin as a candidate biomarker for therapeutic trial in patients with ALS and FTLD.Journal of neural transmission (Vienna, Austria : 1996) · 2016Article
- Lateral Temporal Lobe: An Early Imaging Marker of the Presymptomatic GRN Disease?Journal of Alzheimer's disease : JAD · 2015Article
- Wild-type bone marrow transplant partially reverses neuroinflammation in progranulin-deficient mice.Laboratory investigation; a journal of technical methods and pathology · 2014Article
Corrections and comments
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Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Frontotemporal dementia (FTD) is one of the most important neurodegenerative conditions and Granulin (GRN) is one of the major genes associated to the disease. FTD-GRN patients are still orphan for any evidence-based target-therapy approach. Interestingly, it has been recently found that alkalizing agents rescued haploinsufficiency in cellular models expressing FTD-GRN mutations. We set up a pilot phase II clinical trial in five FTD patients with GRN Thr272s(g.1977_1980delCACT) mutation, to determine if amiodarone (200 mg/day) may (1) reverse progranulin deficiency and (2) delay disease progression. Each patient was scheduled for 7 study visits over 12 months period. We assessed GRN levels at baseline and after amiodarone administration during the treatment course. Somatic and neurologic examinations, along with cognitive and behavioral assessment were recorded as well. No significant effect on peripheral GRN levels was observed. In treated FTD, disease course did not differ when compared with a group of untreated FTD-GRN patients. This is the first trial targeting progranulin rescue in FTD-GRN patients using amiodarone. Despite the negative findings, it may be interesting to extend this attempt to a larger sample of subjects and to other alkalizing agents to restore granulin haploinsufficiency.
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