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14 results on '"Kliest, Tessa"'

4. Innovating Clinical Trials for Amyotrophic Lateral Sclerosis

Author

van Eijk, Ruben P.A., Nikolakopoulos, Stavros, Roes, Kit C.B., Kendall, Lindsay, Han, Steve S., Lavrov, Arseniy, Epstein, Noam, Kliest, Tessa, de Jongh, Adriaan D., Westeneng, Henk-Jan, Al-Chalabi, Ammar, Van Damme, Philip, Hardiman, Orla, Shaw, Pamela J., McDermott, Christopher J., Eijkemans, Marinus J.C., and van den Berg, Leonard H.

Subjects
Clinical Trials as Topic, Research Design, Risk Factors, Patient Selection, Amyotrophic Lateral Sclerosis, Disease Progression, Humans, Contemporary Issues in Practice, Education, & Research
Abstract

Development of effective treatments for amyotrophic lateral sclerosis (ALS) has been hampered by disease heterogeneity, a limited understanding of underlying pathophysiology, and methodologic design challenges. We have evaluated 2 major themes in the design of pivotal, phase 3 clinical trials for ALS-(1) patient selection and (2) analytical strategy-and discussed potential solutions with the European Medicines Agency. Several design considerations were assessed using data from 5 placebo-controlled clinical trials (n = 988), 4 population-based cohorts (n = 5,100), and 2,436 placebo-allocated patients from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. The validity of each proposed design modification was confirmed by means of simulation and illustrated for a hypothetical setting. Compared to classical trial design, the proposed design modifications reduce the sample size by 30.5% and placebo exposure time by 35.4%. By making use of prognostic survival models, one creates a potential to include a larger proportion of the population and maximize generalizability. We propose a flexible design framework that naturally adapts the trial duration when inaccurate assumptions are made at the design stage, such as enrollment or survival rate. In case of futility, the follow-up time is shortened and patient exposure to ineffective treatments or placebo is minimized. For diseases such as ALS, optimizing the use of resources, widening eligibility criteria, and minimizing exposure to futile treatments and placebo is critical to the development of effective treatments. Our proposed design modifications could circumvent important pitfalls and may serve as a blueprint for future clinical trials in this population.

Published
2021

5. Clinical trials in pediatric ALS: a TRICALS feasibility study

Author

Kliest, Tessa, Van Eijk, Ruben P.A., Al-Chalabi, Ammar, Albanese, Alberto, Andersen, Peter M., Amador, Maria Del Mar, Bråthen, Geir, Brunaud-Danel, Veronique, Brylev, Lev, Camu, William, De Carvalho, Mamede, Cereda, Cristina, Cetin, Hakan, Chaverri, Delia, Chiò, Adriano, Corcia, Philippe, Couratier, Philippe, De Marchi, Fabiola, Desnuelle, Claude, Van Es, Michael A., Esteban, JesÚs, Filosto, Massimiliano, GarcÍa Redondo, Alberto, Grosskreutz, Julian, Hanemann, Clemens O., Holmøy, Trygve, Høyer, Helle, Ingre, Caroline, Koritnik, Blaz, Kuzma-Kozakiewicz, Magdalena, Lambert, Thomas, Leigh, Peter N., Lunetta, Christian, Mandrioli, Jessica, Mcdermott, Christopher J., Meyer, Thomas, Mora, Jesus S., Petri, Susanne, Povedano, Mónica, Reviers, Evy, Riva, Nilo, Roes, Kit C.B., Rubio, Miguel Á., Salachas, François, Sarafov, Stayko, Sorarù, Gianni, Stevic, Zorica, Svenstrup, Kirsten, Møller, Anette Torvin, Turner, Martin R., Van Damme, Philip, Van Leeuwen, Lucie A.G., Varona, Luis, VÁzquez Costa, Juan F., Weber, Markus, Hardiman, Orla, Van Den Berg, Leonard H., Kliest, Tessa, Van Eijk, Ruben P.A., Al-Chalabi, Ammar, Albanese, Alberto, Andersen, Peter M., Amador, Maria Del Mar, Bråthen, Geir, Brunaud-Danel, Veronique, Brylev, Lev, Camu, William, De Carvalho, Mamede, Cereda, Cristina, Cetin, Hakan, Chaverri, Delia, Chiò, Adriano, Corcia, Philippe, Couratier, Philippe, De Marchi, Fabiola, Desnuelle, Claude, Van Es, Michael A., Esteban, JesÚs, Filosto, Massimiliano, GarcÍa Redondo, Alberto, Grosskreutz, Julian, Hanemann, Clemens O., Holmøy, Trygve, Høyer, Helle, Ingre, Caroline, Koritnik, Blaz, Kuzma-Kozakiewicz, Magdalena, Lambert, Thomas, Leigh, Peter N., Lunetta, Christian, Mandrioli, Jessica, Mcdermott, Christopher J., Meyer, Thomas, Mora, Jesus S., Petri, Susanne, Povedano, Mónica, Reviers, Evy, Riva, Nilo, Roes, Kit C.B., Rubio, Miguel Á., Salachas, François, Sarafov, Stayko, Sorarù, Gianni, Stevic, Zorica, Svenstrup, Kirsten, Møller, Anette Torvin, Turner, Martin R., Van Damme, Philip, Van Leeuwen, Lucie A.G., Varona, Luis, VÁzquez Costa, Juan F., Weber, Markus, Hardiman, Orla, and Van Den Berg, Leonard H.

Abstract

Background: Pediatric investigation plans (PIPs) describe how adult drugs can be studied in children. In 2015, PIPs for Amyotrophic Lateral Sclerosis (ALS) became mandatory for European marketing-authorization of adult treatments, unless a waiver is granted by the European Medicines Agency (EMA). Objective: To assess the feasibility of clinical studies on the effect of therapy in children (<18 years) with ALS in Europe. Methods: The EMA database was searched for submitted PIPs in ALS. A questionnaire was sent to 58 European ALS centers to collect the prevalence of pediatric ALS during the past ten years, the recruitment potential for future pediatric trials, and opinions of ALS experts concerning a waiver for ALS. Results: Four PIPs were identified; two were waived and two are planned for the future. In total, 49 (84.5%) centers responded to the questionnaire. The diagnosis of 44,858 patients with ALS was reported by 46 sites; 39 of the patients had an onset < 18 years (prevalence of 0.008 cases per 100,000 or 0.087% of all diagnosed patients). The estimated recruitment potential (47 sites) was 26 pediatric patients within five years. A majority of ALS experts (75.5%) recommend a waiver should apply for ALS due to the low prevalence of pediatric ALS. Conclusions: ALS with an onset before 18 years is extremely rare and may be a distinct entity from adult ALS. Conducting studies on the effect of disease-modifying therapy in pediatric ALS may involve lengthy recruitment periods, high costs, ethical/legal implications, challenges in trial design and limited information.

Published
2022
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6. Clinical trials in pediatric ALS: a TRICALS feasibility study

Author

Kliest, Tessa, primary, Van Eijk, Ruben P.A., additional, Al-Chalabi, Ammar, additional, Albanese, Alberto, additional, Andersen, Peter M., additional, Amador, Maria Del Mar, additional, BrÅthen, Geir, additional, Brunaud-Danel, Veronique, additional, Brylev, Lev, additional, Camu, William, additional, De Carvalho, Mamede, additional, Cereda, Cristina, additional, Cetin, Hakan, additional, Chaverri, Delia, additional, Chiò, Adriano, additional, Corcia, Philippe, additional, Couratier, Philippe, additional, De Marchi, Fabiola, additional, Desnuelle, Claude, additional, Van Es, Michael A., additional, Esteban, JesÚs, additional, Filosto, Massimiliano, additional, GarcÍa Redondo, Alberto, additional, Grosskreutz, Julian, additional, Hanemann, Clemens O., additional, HolmØy, Trygve, additional, HØyer, Helle, additional, Ingre, Caroline, additional, Koritnik, Blaz, additional, Kuzma-Kozakiewicz, Magdalena, additional, Lambert, Thomas, additional, Leigh, Peter N., additional, Lunetta, Christian, additional, Mandrioli, Jessica, additional, Mcdermott, Christopher J., additional, Meyer, Thomas, additional, Mora, Jesus S., additional, Petri, Susanne, additional, Povedano, MÓnica, additional, Reviers, Evy, additional, Riva, Nilo, additional, Roes, Kit C.B., additional, Rubio, Miguel Á., additional, Salachas, FranÇois, additional, Sarafov, Stayko, additional, SorarÙ, Gianni, additional, Stevic, Zorica, additional, Svenstrup, Kirsten, additional, MØller, Anette Torvin, additional, Turner, Martin R., additional, Van Damme, Philip, additional, Van Leeuwen, Lucie A.G., additional, Varona, Luis, additional, VÁzquez Costa, Juan F., additional, Weber, Markus, additional, Hardiman, Orla, additional, and Van Den Berg, Leonard H., additional

Published
2022
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7. Innovating Clinical Trials for Amyotrophic Lateral Sclerosis: Challenging the Established Order

Author

Projectafdeling ALS, Brain, Biostatistiek Onderzoek, JC onderzoeksprogramma Methodologie, Opleiding Neurologie, Child Health, Circulatory Health, Infection & Immunity, JC onderzoeksprogramma Infectieziekten, Neurologen, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Nikolakopoulos, Stavros, Roes, Kit C B, Kendall, Lindsay, Han, Steve S, Lavrov, Arseniy, Epstein, Noam, Kliest, Tessa, de Jongh, Adriaan D, Westeneng, Henk-Jan, Al-Chalabi, Ammar, Van Damme, Philip, Hardiman, Orla, Shaw, Pamela J, McDermott, Christopher J, Eijkemans, Marinus J C, van den Berg, Leonard H, Projectafdeling ALS, Brain, Biostatistiek Onderzoek, JC onderzoeksprogramma Methodologie, Opleiding Neurologie, Child Health, Circulatory Health, Infection & Immunity, JC onderzoeksprogramma Infectieziekten, Neurologen, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Nikolakopoulos, Stavros, Roes, Kit C B, Kendall, Lindsay, Han, Steve S, Lavrov, Arseniy, Epstein, Noam, Kliest, Tessa, de Jongh, Adriaan D, Westeneng, Henk-Jan, Al-Chalabi, Ammar, Van Damme, Philip, Hardiman, Orla, Shaw, Pamela J, McDermott, Christopher J, Eijkemans, Marinus J C, and van den Berg, Leonard H

Published
2021

8. Current trends in the clinical trial landscape for amyotrophic lateral sclerosis

Author

Projectafdeling ALS, Brain, ZL Neuromusculaire Ziekten Medisch, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Kliest, Tessa, van den Berg, Leonard H, Projectafdeling ALS, Brain, ZL Neuromusculaire Ziekten Medisch, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Kliest, Tessa, and van den Berg, Leonard H

Published
2020

9. TRICALS: creating a highway toward a cure

Author

Projectafdeling ALS, Brain, Biostatistiek Onderzoek, ZL Neuromusculaire Ziekten Medisch, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Kliest, Tessa, McDermott, Christopher J, Roes, Kit C B, Van Damme, Philip, Chio, Adriano, Weber, Markus, Ingre, Caroline, Corcia, Philippe, Povedano, Mònica, Reviers, Evy, van Es, Michael A, Al-Chalabi, Ammar, Hardiman, Orla, van den Berg, Leonard H, Projectafdeling ALS, Brain, Biostatistiek Onderzoek, ZL Neuromusculaire Ziekten Medisch, Regenerative Medicine and Stem Cells, van Eijk, Ruben P A, Kliest, Tessa, McDermott, Christopher J, Roes, Kit C B, Van Damme, Philip, Chio, Adriano, Weber, Markus, Ingre, Caroline, Corcia, Philippe, Povedano, Mònica, Reviers, Evy, van Es, Michael A, Al-Chalabi, Ammar, Hardiman, Orla, and van den Berg, Leonard H

Published
2020

10. TRICALS: creating a highway toward a cure

Author

van Eijk, Ruben P.A., primary, Kliest, Tessa, additional, McDermott, Christopher J., additional, Roes, Kit C.B., additional, Van Damme, Philip, additional, Chio, Adriano, additional, Weber, Markus, additional, Ingre, Caroline, additional, Corcia, Philippe, additional, Povedano, Mònica, additional, Reviers, Evy, additional, van Es, Michael A., additional, Al-Chalabi, Ammar, additional, Hardiman, Orla, additional, and van den Berg, Leonard H., additional

Published
2020
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11. Additive Effects of Levodopa and a Neurorestorative Diet in a Mouse Model of Parkinson's Disease

Author

Perez-Pardo, Paula, Broersen, Laus M, Kliest, Tessa, van Wijk, Nick, Attali, Amos, Garssen, Johan, Kraneveld, Aletta D, Afd Pharmacology, and Pharmacology

Subjects
dietary intervention, motor-symptoms, Parkinson’s disease, levodopa, non-motor symptoms
Abstract

Though Parkinson's disease (PD) clinical picture is generally dominated by motor impairment, non-motor symptoms, such as cognitive decline and gastrointestinal dysfunctions, may develop before motor symptoms and have major effects on quality of life. L-3,4-di-hydroxy-phenylalanine (Levodopa) is the most commonly used treatment of motor symptoms but has serious side-effects with prolonged use and does not stop the degenerative process. Moreover, gastrointestinal dysfunctions interfere with the absorption of levodopa and modify its effectiveness. Since most patients are on levodopa treatment, there is a need for combinational therapies that allow for an effective reduction of both motor and non-motor symptoms. We have recently shown that a diet containing precursors and cofactors required for membrane phospholipid synthesis, as well as prebiotic fibers, had therapeutic effects in a PD mouse model. We now investigate the effects of combined administration of the same diet together with levodopa in the rotenone model of PD. Mice were injected with rotenone or vehicle in the striatum. The dietary intervention started after full induction of motor symptoms. The effects of dietary intervention and oral treatment with different doses of levodopa were assessed weekly. Motor and cognitive functions were tested, intestinal transit was analyzed and histological examination of the brain and the colon was assessed. Our results confirm our previous findings that rotenone-induced motor and non-motor problems were alleviated by the Active diet (AD). Levodopa showed an additive beneficial effect on rotarod performance in rotenone-treated animals fed with the AD. No negative interaction effects were found between the AD and levodopa. Our findings suggest that the dietary intervention might confer additional clinical benefits on patients receiving levodopa treatment.

Published
2018

14. The gut-brain axis in Parkinson's disease: Possibilities for food-based therapies

Author

Afd Pharmacology, Pharmacology, Perez-Pardo, Paula, Kliest, Tessa, Dodiya, Hemraj B., Broersen, Laus M, Garssen, Johan, Keshavarzian, Ali, Kraneveld, Aletta D, Afd Pharmacology, Pharmacology, Perez-Pardo, Paula, Kliest, Tessa, Dodiya, Hemraj B., Broersen, Laus M, Garssen, Johan, Keshavarzian, Ali, and Kraneveld, Aletta D

Published
2017

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