European therapeutic trials in ANCA-associated systemic vasculitis: disease scoring, consensus regimens and proposed clinical trials EUROPEAN COMMUNITY STUDY GROUP ON CLINICAL TRIALS IN SYSTEMIC VASCULITIS ECSYSVASTRIAL (BMHl-Cr93-1078)

AbstractObjectivesWith the emergence of Novel corona virus, hunt for finding a preventive and therapeutic treatment options has already begun at a rapid pace with faster clinical development programs. The present study was carried out to give an insight of therapeutic interventional trials registered under clinical trial registry of India (CTRI) for COVID-19 pandemic.MethodsAll trials registered under CTRI were evaluated using keyword “COVID” from its inception till 9th June 2020. Out of which, therapeutic interventional studies were chosen for further analysis. Following information was collected for each trial: type of therapeutic intervention (preventive/therapeutic), treatment given, no. of centers (single center/multicentric), type of institution (government/private), study design (randomized/single-blinded/double-blinded) and sponsors (Government/private). Microsoft Office Excel 2007 was used for tabulation and analysis.ResultsThe search yielded total of 205 trials, out of which, 127 (62%) trials were interventional trials. Out of these, 71 (56%) were AYUSH interventions, 36 (28.3%) tested drugs, 9 (7%) tested a nondrug intervention, rest were nutraceuticals and vaccines. About 66 (56%) were therapeutic trials. Majority were single-centered trials, i.e. 87 (73.7%). Trials were government funded in 57 (48.3%) studies. Majority were randomized controlled trials, i.e. 67 (56.8%). AYUSH preparations included AYUSH-64, Arsenic Album, SamshamaniVati etc.ConclusionsThe number of therapeutic interventional clinical trials was fair in India. A clear-cut need exists for an increase in both quantity and quality of clinical trials for COVID-19. Drug repurposing approach in all systems of medicine can facilitate prompt clinical decisions at lower costs than de novo drug development.

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Patients with Primary Central Nervous System Lymphoma Not Eligible for Clinical Trials: Prognostic Factors, Treatment and Outcome

Cancers ◽

10.3390/cancers13122934 ◽

2021 ◽

Vol 13 (12) ◽

pp. 2934

Author(s):

Sabine Seidel ◽

Michelle Margold ◽

Thomas Kowalski ◽

Alexander Baraniskin ◽

Roland Schroers ◽

...

Keyword(s):

Central Nervous System ◽

Clinical Trials ◽

Nervous System ◽

Prognostic Factors ◽

Central Nervous System Lymphoma ◽

Initial Response ◽

Early Response ◽

Multicenter Trial ◽

High Dose ◽

Therapeutic Trials

Patients with primary central nervous system lymphoma (PCNSL) not fulfilling inclusion criteria for clinical trials represent an underreported population. Thirty-four consecutive PCNSL patients seen at our center between 2005 and 2019 with exclusion criteria for therapeutic trials were analyzed (non-study patients) and compared with patients from the G-PCNSL-SG-1 (German PCNSL Study Group 1) study (study patients), the largest prospective multicenter trial on PCNSL, comprising 551 patients. Median follow up was 68 months (range 1–141) in non-study patients and 51 months (1–105) in study patients. Twenty-seven/34 (79.4%) non-study patients received high dose methotrexate (HDMTX), while seven/34 (20.6%) with a glomerular filtration rate (GFR) < 50 mL/min did not. Median overall survival (OS) was six months (95% confidence interval [CI] 0–21 months) in those 34 non-study patients. The 27 non-study patients treated with HDMTX were compared with 526/551 G-PCNSL-SG-1 study patients who had received HDMTX as well. Median OS was 20 months (95% CI 0–45)/21 months (95% CI 18–25) in 27 non-study/526 study patients (p = 0.766). Favorable prognostic factors in non-study patients were young age, application of HDMTX and early response on magnet resonance imaging (MRI). If HDMTX-based chemotherapy can be applied, long-term disease control is possible even in patients not qualifying for clinical trials. Initial response on early MRI might be useful for decision on treatment continuation.

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Defining the Threshold of Permissible Risk for Non-therapeutic Clinical Trials with Children in Europe

European Journal of Health Law ◽

10.1163/15718093-12341420 ◽

2017 ◽

Vol 24 (4) ◽

pp. 414-431

Author(s):

Katherine Wade*

Keyword(s):

Clinical Trials ◽

Medical Care ◽

Medicinal Products ◽

Council Of Europe ◽

Therapeutic Trials ◽

Research With Children ◽

Individual Trial ◽

The One ◽

Definition Of ◽

Trial Participants

Abstract It is important that clinical research with children is encouraged so that they are not exposed to the dangers of extrapolation from adult treatments. Clinical trials with investigational medicinal products (imps) are an important part of improving medical care for children. Both the 2001 Clinical Trials Directive and the 2014 Regulation recognise the need for such research, including the need for non-therapeutic trials with imps. However, it is also recognised that a balance must be struck between permitting tailored medical care for children as a group on the one hand, and protecting individual trial participants from harm on the other. A central issue in striking this balance relates to defining the threshold of risk which should be permitted in such research. This article provides a critical analysis of the current European law in relation to the definition of acceptable risk for non-therapeutic clinical trials with imps and makes recommendations for reform, drawing on law from the Council of Europe, as well as law from the us.

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Cooperative study for the assessment of therapeutic trials for the spinal muscular atrophies. Sensitivity of the DCN-SMA study group methodology

Muscle & Nerve ◽

10.1002/mus.880131306 ◽

1990 ◽

Vol 13 (S1) ◽

pp. S13-S15 ◽

Cited By ~ 5

Author(s):

Barry S. Russman ◽

Susan T. Iannaccone ◽

Jay D. Cook ◽

Ralph R. Buncher ◽

Frederick Samaha ◽

...

Keyword(s):

Study Group ◽

Cooperative Study ◽

Therapeutic Trials ◽

Spinal Muscular Atrophies

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Efficacy and safety of front-line therapy with fludarabine-cyclophosphamide-rituximab regimen for chronic lymphocytic leukemia outside clinical trials: the Israeli CLL Study Group experience

Haematologica ◽

10.3324/haematol.2014.115808 ◽

2015 ◽

Vol 100 (5) ◽

pp. 662-669 ◽

Cited By ~ 14

Author(s):

Y. Herishanu ◽

N. Goldschmidt ◽

O. Bairey ◽

R. Ruchlemer ◽

R. Fineman ◽

...

Keyword(s):

Clinical Trials ◽

Chronic Lymphocytic Leukemia ◽

Lymphocytic Leukemia ◽

Study Group ◽

Efficacy And Safety ◽

Front Line ◽

Line Therapy ◽

Group Experience

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Capillaroscopy as an Outcome Measure for Clinical Trials on the Peripheral Vasculopathy in SSc—Is It Useful?

International Journal of Rheumatology ◽

10.1155/2010/784947 ◽

2010 ◽

Vol 2010 ◽

pp. 1-6 ◽

Cited By ~ 15

Author(s):

Maurizio Cutolo ◽

Alberto Sulli ◽

Carmen Pizzorni ◽

Vanessa Smith

Keyword(s):

Clinical Trials ◽

Systemic Sclerosis ◽

Outcome Measure ◽

Digital Ulcers ◽

Nailfold Videocapillaroscopy ◽

Therapeutic Trials ◽

Specific Serum ◽

Pulmonary Arterial ◽

Microvascular Impairment ◽

Clinical Conditions

Peripheral microvascular impairment in systemic sclerosis (SSc) may be easily detected and scored in a safe noninvasive way by nailfold videocapillaroscopy (NVC). The paper highlights clinical conditions related to SSc in which NVC may represent an outcome measure of therapeutical interventions, by elaborating on their already assessed relationship with the NVC patterns and eventually scores. The 3 important biological/clinical conditions are: the positivity for SSc-specific serum autoantibodies, the presence of SSc skin digital ulcers (DUs) and of pulmonary arterial hypertension (PAH) SSc associated. In conclusion, to the question if capillaroscopy (NVC) may represent in SSc an outcome measure for clinical trials on the peripheral vasculopathy, based on the growing evidence and our detailed studies, the answer is positive. Recent therapeutic trials in SSc are confirming this role, and the experience is growing rapidly.

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