The future of multiple sclerosis therapies: redesigning multiple sclerosis clinical trials in a new therapeutic eraa

2005 ◽  
Vol 11 (6) ◽  
pp. 669-676 ◽  
Author(s):  
H F McFarland ◽  
S C Reingold
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Controlled Trial ◽  
International Workshop ◽  
Regulatory Approval ◽  
Ethical Considerations ◽  
New Therapies ◽  
Clinical Trial Designs ◽  
New Strategies

Due to past success in testing and gaining regulatory approval for a variety of therapies in multiple sclerosis (MS), the conduct of future clinical trials has become increasingly problematic. An international workshop has met to discuss the issues facing the MS clinical trial community and to examine possible new strategies for the design of trials. Particular focus has been placed on trials that either avoid the use of a placebo because of ethical considerations or on designs that allow new therapies to be studied more rapidly or with fewer patients than needed in a conventional placebo-controlled trial. The discussions resulting from the workshop should provide a basis for the examination and implementation of innovative clinical trial designs in MS.

Digital Technology in Clinical Trials for Multiple Sclerosis: a systematic review. (Preprint)

2020 ◽  
Author(s):  
Marcello De Angelis ◽  
Luigi Lavorgna ◽  
Antonio Carotenuto ◽  
Martina Petruzzo ◽  
Roberta Lanzillo ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Digital Technology ◽  
Clinical Trial Design ◽  
Motor Rehabilitation ◽  
Robot Assisted ◽  
Future Directions ◽  
Treatment Side Effects

BACKGROUND Clinical trials in multiple sclerosis (MS) have leveraged the use of digital technology to overcome limitations in treatment and disease monitoring. OBJECTIVE To review the use of digital technology in concluded and ongoing MS clinical trials. METHODS In March 2020, we searched for “multiple sclerosis” and “trial” on pubmed.gov and clinicaltrials.gov using “app”, “digital”, “electronic”, “internet” and “mobile” as additional search words, separately. Overall, we included thirty-five studies. RESULTS Digital technology is part of clinical trial interventions to deliver psychotherapy and motor rehabilitation, with exergames, e-training, and robot-assisted exercises. Also, digital technology has become increasingly used to standardise previously existing outcome measures, with automatic acquisitions, reduced inconsistencies, and improved detection of symptoms. Some trials have been developing new patient-centred outcome measures for the detection of symptoms and of treatment side effects and adherence. CONCLUSIONS We will discuss how digital technology has been changing MS clinical trial design, and possible future directions for MS and neurology research.

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

scholarly journals THE DESIGN OF CLINICAL TRIALS FOR NEW THERAPIES IN BLADDER CANCER. IS IT TIME FOR NEW STRATEGIES ?

1995 ◽  
Vol 2 (s2) ◽  
pp. 113-116
Author(s):  
PHM De Mulder ◽  
R. Williams ◽  
Y. Ohashi ◽  
BA Blumenstein ◽  
M De Pauzv ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Clinical Trials ◽  
New Therapies ◽  
New Strategies

Barriers to participation in breast cancer trials

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 6593-6593
Author(s):  
O. Herasme ◽  
J. Goldberg ◽  
R. Sandoval ◽  
C. Harris ◽  
Y. Ortiz-Pride ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Child Care ◽  
Cancer Patients ◽  
Controlled Trial ◽  
Demographic Factors ◽  
Trial Participation ◽  
Barriers To Participation ◽  
Cancer Trials

6593 Background: Clinical cancer trials allow investigators to test the effectiveness and safety of new cancer drugs and treatments. Historically, fewer that 5% of cancer patients have participated in clinical trials. The purpose of this study was to assess attitudes, beliefs, and practical barriers to clinical trial recruitment. Methods: Women were recruited in the Herbert Irving Comprehensive Cancer Center while waiting for routine breast screening or for oncology care in connection with a diagnosis of breast cancer. The 29-item survey questionnaire covered demographic factors, prior cancer diagnosis or risk factors, past experience with clinical trials if any, willingness to participate in different types of trials, and attitudinal and practical barriers to participation. Results: Of 329 respondents, 48.9% were non- Hispanic white, 10.9% non-Hispanic black, 34.9% Hispanic, and 5.30% other/unknown. The mean age of participants was 52.5 (SD=12.1). Of 131 (39.8%) participants reporting that they had been asked to participate in clinical trial, 82 were white, 17 black and 32 Hispanic. Of those who enrolled, 64 were white, 14 were black, and 19 Hispanic. Of those asked to participate 56/63 breast cancer patients (88.9%) and 44/68 others (64.7%) enrolled (P=0.002). Of 48 who reported that they had child care responsibilities, 33 enrolled (68.8) compared to 67/83 (80.7%) of those without such responsibilities (P=0.07). Of the total sample, 88/220 (40.0%) of those without childcare responsibilities but only 32/109 (29.4) said they would be willing to participate in a placebo-controlled trial. Respondents were twice as likely to say they would participate in a trial comparing two active agents as a placebo-controlled trial. Conclusion: Our findings suggest that being asked to participate in a clinical trial may be associated with demographic factors, and that specific circumstances, such as child care responsibilities, may also affect trial participation. Awareness of these barriers may help investigators to develop effective strategies for overcoming them and for improving trial participation overall. No significant financial relationships to disclose.

Trifluoperazine (“Stelazine”) a Controlled Clinical Trial in Chronic Schizophrenia

1961 ◽  
Vol 107 (447) ◽  
pp. 250-257 ◽  
Author(s):  
W. J. Stanley ◽  
D. Walton
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Controlled Trial ◽  
Pernicious Anaemia ◽  
Chronic Schizophrenia ◽  
Mental Illnesses ◽  
Controlled Clinical Trial ◽  
Vitamin B ◽  
Uncontrolled Trial ◽  
New Compounds

So many new compounds are now being introduced for the treatment of psychiatric disorders that it is difficult for the clinician to assess the accuracy of the claims made for them. If a form of therapy is one hundred per cent. effective in a disease which was previously one hundred per cent. fatal, for example vitamin B12 in pernicious anaemia, the question of a controlled trial does not arise. But even the manufacturers do not claim such a degree of effectiveness for drugs advocated for the treatment of mental illnesses, and in addition the natural course of such illnesses is very variable, so that it is essential that clinical trials of these drugs should be controlled. Foulds (1958) has reviewed British and American clinical trials of drugs in psychiatry during the years 1951 to 1956, and has shown that the less controlled a trial, the more likely it is to result in a favourable report on the drug being tested, presumably because of bias on the part of the clinician. Forrester (1958), however, on the basis of a completely uncontrolled trial of Stelazine on only twenty-five patients, concluded that “the amount of improvement in a few cases was not sufficient to encourage the further use of the drug”.

scholarly journals Anti cytokine therapy of secondarily-progressing multiple sclerosis (ms)

1997 ◽  
Vol XXIX (1-2) ◽  
pp. 55-58
Author(s):  
Е. I. Gusev ◽  
Т. L. Demina ◽  
А. N. Boiko ◽  
А. V. Buglak ◽  
I. А. Belyaeva ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Effect ◽  
Preliminary Test ◽  
Cytokine Therapy ◽  
Preliminary Clinical Trial

For treating active forms of secondarily-progressing multiple sclerosis a preliminary clinical trial was performed by two courses: antibody-to-cytokine course (INT, INT and TNF) and cyclosporin А-course (sandimmun). Data of preliminary test of antibodies to cytokines evidence to possibility of performing further clinical trials "antibodies to INT and to TNF (to a lesser degree)" during active forms of progressive MS. Were obtained the results, evidencing to a possible use of sandimmun in constantly progressive forms of secondarily progressing disease, when favourable clinical effect coincided with supression of cytokine IL-2 production and lowering of activeness of immunocytes.

Highly efficient clinical trial designs for reliable screening of under-performing treatments: Application to the COVID-19 Pandemic

2020 ◽  
Vol 17 (5) ◽  
pp. 483-490
Author(s):  
Steven Piantadosi
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sample Size ◽  
Trial Design ◽  
Ranking And Selection ◽  
Design Elements ◽  
Highly Efficient ◽  
Clinical Trial Designs ◽  
Under Performing ◽  
The Many

Background: The COVID-19 pandemic presents challenges for clinical trials including urgency, disrupted infrastructure, numerous therapeutic candidates, and the need for highly efficient trial and development designs. This paper presents design components and rationale for constructing highly efficient trials to screen potential COVID-19 treatments. Methods: Key trial design elements useful in this circumstance include futility hypotheses, treatment pooling, reciprocal controls, ranking and selection, and platform administration. Assuming most of the many candidates for COVID-19 treatment are likely to be ineffective, these components can be combined to facilitate very efficient comparisons of treatments. Results: Simulations indicate such designs can reliably discard underperforming treatments using sample size to treatment ratios under 30. Conclusions: Methods to create very efficient clinical trial comparisons of treatments for COVID-19 are available. Such designs might be helpful in the pandemic and should be considered for similar needs in the future.

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