scholarly journals Open‐Label Pilot Study of Interferon Gamma ‐ 1b in Patients with Non‐infantile Osteopetrosis

JBMR Plus ◽  
2022 ◽  
Author(s):  
Andrew Nguyen ◽  
Weston P. Miller ◽  
Ashish Gupta ◽  
Troy C. Lund ◽  
Daniel Schiferl ◽  
...  
Keyword(s):  
Pilot Study ◽  
Interferon Gamma ◽  
Open Label

Open-label pilot study of interferon gamma-1b in Friedreich ataxia

2014 ◽  
Vol 132 (1) ◽  
pp. 7-15 ◽  
Author(s):  
L. Seyer ◽  
N. Greeley ◽  
D. Foerster ◽  
C. Strawser ◽  
S. Gelbard ◽  
...  
Keyword(s):  
Pilot Study ◽  
Interferon Gamma ◽  
Friedreich Ataxia ◽  
Open Label

Evaluating Thrombin Inhibition in Alzheimer’s Disease (The BEACON Trial): Protocol for a Pilot Study (Preprint)

2019 ◽  
Author(s):  
Cláudia Yang Santos ◽  
Christine Getter ◽  
John Stoukides ◽  
Brian Ott ◽  
Stephen Salloway ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Pilot Study ◽  
Cognitive Performance ◽  
Thrombin Inhibitor ◽  
Placebo Control ◽  
Open Label ◽  
Double Blind ◽  
Thrombin Inhibition ◽  
Approved Drugs

BACKGROUND The precise mechanisms whereby cardiovascular risk factors increase the risk of Alzheimer’s disease (AD) have not been delineated. We reported that microvessels isolated from AD brains overexpress a diverse array of neurotoxic and inflammatory proteins, which is consistent with the process of vascular activation. In pre-clinical studies using AD animal models we showed that a vascular activation inhibitor reduced vascular-derived neuroinflammation and improved cognitive performance. Thrombin is a key mediator of cerebrovascular activation in AD. OBJECTIVE This study aims to investigate the safety and potential efficacy of the direct thrombin inhibitor dabigatran, in patients with mild cognitive impairment (MCI) or mild AD to decrease vascular-derived neuroinflammation and improve cognitive performance. METHODS Participants will be enrolled then evaluated quarterly throughout the 24-month study. This is a 24-month randomized-control, double-blind, placebo-controlled, multicenter, delayed-start, pilot study evaluating thrombin inhibition in people with biomarker-confirmed MCI probably due to AD or mild AD. 40 - 60 participants will be recruited between 50 - 85 years old. In the initial 9-months of study, either dabigatran or placebo will be orally administered to patients at a dose of 150 mg per day. After 9 months of the placebo-control (Phase I), the placebo arm will cross-over to an active, open-label (Phase II) where all patients will be treated with a 150 mg daily dose of dabigatran orally for an additional 12 months. A 3-month non-treatment follow-up period will assess duration of effects. RESULTS Beginning in July 2019, and concluding in August 2022, this study is expected to publish final results in January 2023. CONCLUSIONS BEACON is a first-in-kind randomized clinical trial targeting thrombin activation in AD therapeutics. This trial will stimulate translational investigations of an FDA-approved drugs in a newly defined therapeutic areas. CLINICALTRIAL Clinicaltrials.gov NCT03752294

An open-label, single-center pilot study to test the effects of an amino acid mixture in older patients admitted to internal medicine wards

Nutrition ◽  
2020 ◽  
Vol 69 ◽  
pp. 110588 ◽  
Author(s):  
Francesco Bellanti ◽  
Aurelio Lo Buglio ◽  
Elena Di Stasio ◽  
Giorgia di Bello ◽  
Rosanna Tamborra ◽  
...  
Keyword(s):  
Internal Medicine ◽  
Amino Acid ◽  
Pilot Study ◽  
Older Patients ◽  
Amino Acid Mixture ◽  
Acid Mixture ◽  
Single Center ◽  
Open Label ◽  
Internal Medicine Wards

scholarly journals Effects of bumetanide on neurodevelopmental impairments in patients with tuberous sclerosis complex: an open-label pilot study

2020 ◽  
Vol 11 (1) ◽  
Author(s):  
Dorinde M. van Andel ◽  
Jan J. Sprengers ◽  
Bob Oranje ◽  
Floortje E. Scheepers ◽  
Floor E. Jansen ◽  
...  
Keyword(s):  
Pilot Study ◽  
Tuberous Sclerosis ◽  
Tuberous Sclerosis Complex ◽  
Open Label
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