HSP90 inhibitor RGRN‐305 for oral treatment of plaque type psoriasis: efficacy, safety and biomarker results in an open‐label proof‐of‐concept study

2021 ◽  
Author(s):  
A. Bregnhøj ◽  
K.K.H. Thuesen ◽  
T. Emmanuel ◽  
T. Litman ◽  
C.L. Grek ◽  
...  
Keyword(s):  
Oral Treatment ◽  
Hsp90 Inhibitor ◽  
Proof Of Concept ◽  
Open Label ◽  
Concept Study ◽  
Plaque Type

scholarly journals Tofacitinib as a Steroid-Sparing Therapy in Pulmonary Sarcoidosis, an Open-Label Prospective Proof-of-Concept Study

Lung ◽  
2021 ◽  
Author(s):  
Marcia A. Friedman ◽  
Brian Le ◽  
Janelle Stevens ◽  
Julianna Desmarais ◽  
Daniel Seifer ◽  
...  
Keyword(s):  
Pulmonary Sarcoidosis ◽  
Proof Of Concept ◽  
Open Label ◽  
Concept Study ◽  
Steroid Sparing

Efficacy and safety of pregabalin in refractory focal epilepsy with and without comorbid anxiety disorders — Results of an open-label, parallel group, investigator-initiated, proof-of-concept study

2013 ◽  
Vol 29 (2) ◽  
pp. 298-304 ◽  
Author(s):  
Christian Brandt ◽  
Martin Schoendienst ◽  
Monika Trentowska ◽  
Mario Schrecke ◽  
Nora Fueratsch ◽  
...  
Keyword(s):  
Anxiety Disorders ◽  
Focal Epilepsy ◽  
Proof Of Concept ◽  
Efficacy And Safety ◽  
Open Label ◽  
Concept Study ◽  
Comorbid Anxiety ◽  
Parallel Group

scholarly journals Autologous Bone Marrow Mononuclear Cell Therapy for Autism: An Open Label Proof of Concept Study

2013 ◽  
Vol 2013 ◽  
pp. 1-13 ◽  
Author(s):  
Alok Sharma ◽  
Nandini Gokulchandran ◽  
Hemangi Sane ◽  
Anjana Nagrajan ◽  
Amruta Paranjape ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Cellular Therapy ◽  
Autologous Bone Marrow ◽  
Bone Marrow Mononuclear Cell ◽  
Clinical Effects ◽  
Proof Of Concept ◽  
Open Label ◽  
Global Improvement ◽  
Concept Study ◽  
Autologous Bone

Cellular therapy is an emerging therapeutic modality with a great potential for the treatment of autism. Recent findings show that the major underlying pathogenetic mechanisms of autism are hypoperfusion and immune alterations in the brain. So conceptually, cellular therapy which facilitates counteractive processes of improving perfusion by angiogenesis and balancing inflammation by immune regulation would exhibit beneficial clinical effects in patients with autism. This is an open label proof of concept study of autologous bone marrow mononuclear cells (BMMNCs) intrathecal transplantation in 32 patients with autism followed by multidisciplinary therapies. All patients were followed up for 26 months (mean 12.7). Outcome measures used were ISAA, CGI, and FIM/Wee-FIM scales. Positron Emission Tomography-Computed Tomography (PET-CT) scan recorded objective changes. Out of 32 patients, a total of 29 (91%) patients improved on total ISAA scores and 20 patients (62%) showed decreased severity on CGI-I. The difference between pre- and postscores was statistically significant (P<0.001) on Wilcoxon matched-pairs signed rank test. On CGI-II 96% of patients showed global improvement. The efficacy was measured on CGI-III efficacy index. Few adverse events including seizures in three patients were controlled with medications. The encouraging results of this leading clinical study provide future directions for application of cellular therapy in autism.

scholarly journals Selective targeting of angiopoietin-like 3 (ANGPTL3) with vupanorsen for the treatment of patients with familial partial lipodystrophy (FPLD): results of a proof-of-concept study

2021 ◽  
Vol 20 (1) ◽  
Author(s):  
Maria C. Foss-Freitas ◽  
Baris Akinci ◽  
Adam Neidert ◽  
Victoria J. Bartlett ◽  
Eunju Hurh ◽  
...  
Keyword(s):  
Subcutaneous Fat ◽  
Area Under The Curve ◽  
Density Lipoprotein ◽  
Resistance Index ◽  
Lipoprotein Cholesterol ◽  
Proof Of Concept ◽  
Open Label ◽  
Partial Lipodystrophy ◽  
Concept Study ◽  
Familial Partial Lipodystrophy

Abstract Background Familial partial lipodystrophy (FPLD) is a rare disease characterized by selective loss of peripheral subcutaneous fat, associated with dyslipidemia and diabetes mellitus. Reductions in circulating levels of ANGPTL3 are associated with lower triglyceride and other atherogenic lipids, making it an attractive target for treatment of FPLD patients. This proof-of-concept study was conducted to assess the efficacy and safety of targeting ANGPTL3 with vupanorsen in patients with FPLD. Methods This was an open-label study. Four patients with FPLD (two with pathogenic variants in LMNA gene, and two with no causative genetic variant), diabetes (HbA1c ≥ 7.0 % and ≤ 12 %), hypertriglyceridemia (≥ 500 mg/dL), and hepatic steatosis (hepatic fat fraction, HFF ≥ 6.4 %) were included. Patients received vupanorsen subcutaneously at a dose of 20 mg weekly for 26 weeks. The primary endpoint was the percent change from baseline in fasting triglycerides at Week 27. Other endpoints analyzed at the same time point included changes in ANGPTL3, fasting lipids and lipoproteins, insulin secretion/sensitivity, postprandial lipids, and glycemic changes in response to a mixed meal test, HFF measured by MRI, and body composition measured by dual-energy absorptiometry (DEXA). Results Baseline mean ± SD fasting triglyceride level was 9.24 ± 4.9 mmol/L (817.8 ± 431.9 mg/dL). Treatment resulted in reduction in fasting levels of triglycerides by 59.9 %, ANGPTL3 by 54.7 %, and in several other lipoproteins/lipids, including very low-density lipoprotein cholesterol by 53.5 %, non-high-density lipoprotein cholesterol by 20.9 %, and free fatty acids (FFA) by 41.7 %. The area under the curve for postprandial triglycerides, FFA, and glucose was reduced by 60 %, 32 %, and 14 %, respectively. Treatment with vupanorsen also resulted in 55 % reduction in adipose tissue insulin resistance index, while other insulin sensitivity indices and HbA1c levels were not changed. Additional investigations into HFF and DEXA parameters suggested dynamic changes in fat partitioning during treatment. Adverse events observed were related to common serious complications associated with diabetes and FPLD. Vupanorsen was well tolerated, and there was no effect on platelet count. Conclusions Although limited, these results suggest that targeting ANGPTL3 with vupanorsen could address several metabolic abnormalities in patients with FPLD.

Abatacept in IgG4-related disease: a prospective, open-label, single-arm, single-centre, proof-of-concept study

2021 ◽  
Author(s):  
Mark A Matza ◽  
Cory A Perugino ◽  
Liam Harvey ◽  
Ana D Fernandes ◽  
Zachary S Wallace ◽  
...  
Keyword(s):  
Proof Of Concept ◽  
Single Centre ◽  
Open Label ◽  
Concept Study ◽  
Related Disease ◽  
Igg4 Related Disease

5.20 Efficacy of Transcranial Near-Infrared Light Treatment in ASD: Interim Analysis of an Open-Label Proof of Concept Study of a Novel Approach

2018 ◽  
Vol 57 (10) ◽  
pp. S233-S234
Author(s):  
Tolga Atilla Ceranoglu ◽  
Barbora Hoskova ◽  
Paolo Cassano ◽  
Joseph Biederman ◽  
Gagan Joshi
Keyword(s):  
Interim Analysis ◽  
Near Infrared ◽  
Light Treatment ◽  
Infrared Light ◽  
Proof Of Concept ◽  
Open Label ◽  
Near Infrared Light ◽  
Concept Study ◽  
Novel Approach
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