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62 people with a confirmed repeat of the C9ORF72 gene mutation have ALS, ALS-FTD, or FTD, or are carriers of the gene mutation and have a symptomatic family member, with a documented repeat expansion in C9ORF72 gene mutation. At enrollment and three follow-up visits to NIH to determine disease severity, design Participants will perform a structured battery of medical and neuropsychological assessments. The ALS Functional rating scale, which has been revised, will be the primary measure of motor clinical function. Changes in verbal fluency scores is the primary measure of cognitive function. The fronto-behavioral index will be the primary measure of behavioral dysfunction, as the caregiver evaluation will be the most reliable indicator of mental illness. In an exploratory manner, the relationship between primary and secondary clinical outcome measures and candidate biomarkers will be investigated to see whether candidate biomarkers are predictive of disease onset or progression.
Source link: https://clinicaltrials.gov/ct2/show/NCT01925196
For the first eight weeks, participants will be given baricitinib 2 mg per day by mouth for the first 8 weeks and baricitinib 4 mg per day by mouth for the remaining 16 weeks. Participants will be eligible if their CSF biomarker meets threshold requirements and meets other eligibility criteria, but not both eligibility requirements will be considered. All enrolled participants must have received their first dose of recombinant zoster vaccine within four years before starting therapy. Baricitinib, an FDA-approved treatment for rheumatoid arthritis, restored inflammatory biomarkers and neural cell death in a human neural cell culture model of inflammatory-mediated death in a dose-dependent manner. Baricitinib was one of the leading drugs to reverse AD's behavior, according to independent research of gene expression profiles of AD brains, coded DRIAD. The trial will determine whether baricitinib, 2 mg/day or both enters the cerebrospinal fluid and achieves therapeutic tolerance, as well as whether it reduces inflammation biomarkers in patients at risk of or with ALS. If this Phase I/II trial proves that baricitinib is safe in AD and ALS patients and achieves therapeutic standards in the CSF as determined by drug concentration and pharmacokinetic biomarkers, a Phase III clinical trial powered to evaluate clinical outcomes in AD patients, ALS patients, or both would be warranted.
Source link: https://clinicaltrials.gov/ct2/show/NCT05189106
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