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122 results on '"Monnet, Annabelle"'

2. Integrating Phase 2 into Phase 3 based on an Intermediate Endpoint While Accounting for a Cure Proportion -- with an Application to the Design of a Clinical Trial in Acute Myeloid Leukemia

Author

Rufibach, Kaspar, Heinzmann, Dominik, and Monnet, Annabelle

Subjects
Statistics - Applications, 62P10
Abstract

For a trial with primary endpoint overall survival for a molecule with curative potential, statistical methods that rely on the proportional hazards assumption may underestimate the power and the time to final analysis. We show how a cure proportion model can be used to get the necessary number of events and appropriate timing via simulation. If Phase 1 results for the new drug are exceptional and/or the medical need in the target population is high, a Phase 3 trial might be initiated after Phase 1. Building in a futility interim analysis into such a pivotal trial may mitigate the uncertainty of moving directly to Phase 3. However, if cure is possible, overall survival might not be mature enough at the interim to support a futility decision. We propose to base this decision on an intermediate endpoint that is sufficiently associated with survival. Planning for such an interim can be interpreted as making a randomized Phase 2 trial a part of the pivotal trial: if stopped at the interim, the trial data would be analyzed and a decision on a subsequent Phase 3 trial would be made. If the trial continues at the interim then the Phase 3 trial is already underway. To select a futility boundary, a mechanistic simulation model that connects the intermediate endpoint and survival is proposed. We illustrate how this approach was used to design a pivotal randomized trial in acute myeloid leukemia, discuss historical data that informed the simulation model, and operational challenges when implementing it., Comment: 23 pages, 3 figures, 3 tables. All code is available on github: https://github.com/numbersman77/integratePhase2.git

Published
2019
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3. Exploratory Analysis of PASADENA Open-label Extension Evaluating the Effect of Prasinezumab on the Progression of Motor Signs and Symptoms (S30.006)

Author

Pagano, Gennaro, primary, Monnet, Annabelle, additional, Reyes, Adriana, additional, Simuni, Tanya, additional, Postuma, Ronald, additional, Pavese, Nicola, additional, Stocchi, Fabrizio, additional, Smigorski, Krzysztof, additional, Gerbaldo, Valentina, additional, Thomas, Riorge, additional, Svoboda, Hanno, additional, Fontoura, Paulo, additional, Doody, Rachelle, additional, Kerchner, Geoffrey, additional, Brundin, Patrik, additional, Bonni, Azad, additional, Marek, Kenneth, additional, and Nikolcheva, Tania, additional

Published
2024
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4. Exploratory Sensor-based Outcome Measures Show Divergent Slopes of Motor Sign Progression in Parkinson’s Disease Patients Treated with Prasinezumab

Author

Taylor, Kirsten, primary, Lipsmeier, Florian, additional, Scelsi, Marzia, additional, Volkova-Volkmar, Ekaterina, additional, Rukina, Daria, additional, Popp, Werner, additional, Lambrecht, Stefan, additional, Anzures-Cabrera, Judith, additional, Summers, David, additional, Abt, Markus, additional, Monnet, Annabelle, additional, Kilchenmann, Timothy, additional, Schjodt-Eriksen, Jens, additional, Essioux, Laurent, additional, Kustermann, Thomas, additional, Zago, Wagner, additional, Svoboda, Hanno, additional, Nikolcheva, Tania, additional, Postuma, Ronald, additional, Pagano, Gennaro, additional, and Lindemann, Michael, additional

Published
2024
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5. Murine double minute 2 inhibition alone or with cytarabine in acute myeloid leukemia: Results from an idasanutlin phase 1/1b study⋆

Author

Yee, Karen, Papayannidis, Cristina, Vey, Norbert, Dickinson, Michael J., Kelly, Kevin R., Assouline, Sarit, Kasner, Margaret, Seiter, Karen, Drummond, Mark W., Yoon, Sung-Soo, Lee, Je-Hwan, Blotner, Steven, Jukofsky, Lori, Pierceall, William E., Zhi, Jianguo, Simon, Silke, Higgins, Brian, Nichols, Gwen, Monnet, Annabelle, Muehlbauer, Susanne, Ott, Marion, Chen, Lin-Chi, and Martinelli, Giovanni

Published
2021
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6. Modeling of Parkinson’s Disease Progression and Implications for Detection of Disease Modification in Treatment Trials

Author

Ribba, Benjamin, Simuni, Tanya, Marek, Kenneth, Siderowf, Andrew, Diack, Cheikh, Pierrillas, Philippe Bernard, Monnet, Annabelle, Ricci, Benedicte, Nikolcheva, Tania, and Pagano, Gennaro

Abstract

Objectively measuring Parkinson’s disease (PD) signs and symptoms over time is critical for the successful development of treatments aimed at halting the disease progression of people with PD. To create a clinical trial simulation tool that characterizes the natural history of PD progression and enables a data-driven design of randomized controlled studies testing potential disease-modifying treatments (DMT) in early-stage PD. Data from the Parkinson’s Progression Markers Initiative (PPMI) were analyzed with nonlinear mixed-effect modeling techniques to characterize the progression of MDS-UPDRS part I (non-motor aspects of experiences of daily living), part II (motor aspects of experiences of daily living), and part III (motor signs). A clinical trial simulation tool was built from these disease models and used to predict probability of success as a function of trial design. MDS-UPDRS part III progresses approximately 3 times faster than MDS-UPDRS part II and I, with an increase of 3 versus 1 points/year. Higher amounts of symptomatic therapy is associated with slower progression of MDS-UPDRS part II and III. The modeling framework predicts that a DMT effect on MDS-UPDRS part III could precede effect on part II by approximately 2 to 3 years. Our clinical trial simulation tool predicted that in a two-year randomized controlled trial, MDS-UPDRS part III could be used to evaluate a potential novel DMT, while part II would require longer trials of a minimum duration of 3 to 5 years underscoring the need for innovative trial design approaches including novel patient-centric measures. To develop effective medicines that can slow down or stop the progression of Parkinson’s disease (PD), it is important to accurately understand how the disease worsens over time. We used data from an observational study, led by the Michael J. Fox Foundation, called the Parkinson’s Progression Markers Initiative (PPMI) to understand the natural progression of  PD. We simulated clinical trials on a computer using different scales to measure the progression of PD. We specifically looked at a physician-reported measure MDS-UPDRS part III, and at a patient-reported measure MDS-UPDRS part II of how PD symptoms worsen over time. To measure the effect of a new medicine slowing down the progression of PD using patient-reported measure MDS-UPDRS part II, we estimate that we may need to conduct a clinical trial of at least 3 to 5 years. On the other hand, to measure an effect using physician-reported measure MDS-UPDRS part III, the duration of the trial could be shorter than 2 years. We were also able to show that worsening recorded by the physician-reported measure MDS-UPDRS part III could be predictive of a later worsening recorded by the patient-reported measure MDS-UPDRS part II. We concluded that MDS-UPDRS part III may be a good endpoint for a clinical trial of a reasonable duration and that MDS-UPDRS part II could be measured in longer studies, for example, open-label extensions.

Published
2024
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7. Knowledge politics in global governance: philanthropists' knowledge-making practices in global health.

Author

Littoz-Monnet, Annabelle and Osorio Garate, Ximena

Abstract

Existing research points to the presence of philanthropists in global governance as funders of programmes and partners. Through an in-depth exploration of global health governance, we highlight that philanthropic organizations now shape governance by acting as producers of knowledge. Practicing 'knowledge philanthropism', they collect, produce and assemble the data, calculations and research which is used by International Organizations (IOs) to govern problems. In addition, philanthropies craft tools of interpretation, whether concepts, vocabularies, or concrete technological devices that embed these, which are being used for the treatment of the knowledge they themselves produce. While performing such activities, they reify their own role and enable their deeper entanglement in the knowledge machinery of global governance, fashioning data-centric activities as the solution to global health problems, and themselves as the necessary partners in this resource-intensive data collection effort. The epistemic power of philanthropists produces political effects, on health interventions and modes of governing, which deeply participate to the transformation of all matters into objects of investments for financial returns. We explore these processes in relation to global health governance, with a specific focus on medical hypertension, fashioned as a top global health priority and a necessary 'investment' by the World Health Organization (WHO) and other sites of global governance. [ABSTRACT FROM AUTHOR]

Published
2024
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11. Prasinezumab slows motor progression in rapidly progressing early-stage Parkinson’s disease

Author

Pagano, Gennaro, primary, Taylor, Kirsten, additional, Cabrera, Judith, additional, Simuni, Tanya, additional, Marek, Kenneth, additional, Postuma, Ronald, additional, Pavese, Nicola, additional, Stocchi, Fabrizio, additional, Brockmann, Kathrin, additional, Svoboda, Hanno, additional, Trundell, Dylan, additional, Monnet, Annabelle, additional, Doody, Rachelle, additional, Fontoura, Paulo, additional, Kerchner, Geoffrey, additional, Brundin, Patrik, additional, Nikolcheva, Tania, additional, and Bonni, Azad, additional

Published
2023
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14. Exploratory Delayed-Start Analysis Of PASADENA Part 3 52-Week OLE Evaluating Prasinezumab Efficacy On Motor Progression And Complications In Early-Stage PD (S32.005)

Author

Pagano, Gennaro, primary, Zanigni, Stefano, additional, Monnet, Annabelle, additional, Taylor, Kirsten, additional, Hahn, Andrea, additional, Simuni, Tanya, additional, Marek, Kenneth, additional, Postuma, Ronald, additional, Pavese, Nicola, additional, Stocchi, Fabrizio, additional, Svoboda, Hanno, additional, Fontoura, Paulo, additional, Doody, Rachelle, additional, Kerchner, Geoffrey, additional, Bonni, Azad, additional, and Nikolcheva, Tania, additional

Published
2023
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18. Trial of Prasinezumab in Early-Stage Parkinson’s Disease

Author

Pagano, Gennaro, primary, Taylor, Kirsten I., additional, Anzures-Cabrera, Judith, additional, Marchesi, Maddalena, additional, Simuni, Tanya, additional, Marek, Kenneth, additional, Postuma, Ronald B., additional, Pavese, Nicola, additional, Stocchi, Fabrizio, additional, Azulay, Jean-Philippe, additional, Mollenhauer, Brit, additional, López-Manzanares, Lydia, additional, Russell, David S., additional, Boyd, James T., additional, Nicholas, Anthony P., additional, Luquin, María R., additional, Hauser, Robert A., additional, Gasser, Thomas, additional, Poewe, Werner, additional, Ricci, Benedicte, additional, Boulay, Anne, additional, Vogt, Annamarie, additional, Boess, Frank G., additional, Dukart, Juergen, additional, D’Urso, Giulia, additional, Finch, Rebecca, additional, Zanigni, Stefano, additional, Monnet, Annabelle, additional, Pross, Nathalie, additional, Hahn, Andrea, additional, Svoboda, Hanno, additional, Britschgi, Markus, additional, Lipsmeier, Florian, additional, Volkova-Volkmar, Ekaterina, additional, Lindemann, Michael, additional, Dziadek, Sebastian, additional, Holiga, Štefan, additional, Rukina, Daria, additional, Kustermann, Thomas, additional, Kerchner, Geoffrey A., additional, Fontoura, Paulo, additional, Umbricht, Daniel, additional, Doody, Rachelle, additional, Nikolcheva, Tania, additional, and Bonni, Azad, additional

Published
2022
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22. A 104-week delayed-start analysis of PASADENA (Phase II study evaluating the safety and efficacy of prasinezumab in early Parkinson’s disease) (S16.008)

Author

Pagano, Gennaro, primary, Zanigni, Stefano, additional, Monnet, Annabelle, additional, Taylor, Kirsten I., additional, Hahn, Andrea, additional, Marek, Kenneth, additional, Postuma, Ron, additional, Pavese, Nicola, additional, Stocchi, Fabrizio, additional, Simuni, Tanya, additional, D’Urso, Giulia, additional, Pross, Nathalie, additional, Lindemann, Michael, additional, Svoboda, Hanno, additional, Fontoura, Paulo, additional, Doody, Rachelle, additional, Kerchner, Geoffrey A., additional, Bonni, Azad, additional, and Nikolcheva, Tania, additional

Published
2022
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26. Trial of Prasinezumab in Early-Stage Parkinson's Disease

Author

Pagano, Gennaro, Taylor, Kirsten I, Mollenhauer, Brit, López-Manzanares, Lydia, Russell, David S, Boyd, James T, Nicholas, Anthony P, Luquin, María R, Hauser, Robert A, Gasser, Thomas, Poewe, Werner, Ricci, Benedicte, Anzures-Cabrera, Judith, Boulay, Anne, Vogt, Annamarie, Boess, Frank G, Dukart, Juergen, D'Urso, Giulia, Finch, Rebecca, Zanigni, Stefano, Monnet, Annabelle, Pross, Nathalie, Hahn, Andrea, Marchesi, Maddalena, Svoboda, Hanno, Britschgi, Markus, Lipsmeier, Florian, Volkova-Volkmar, Ekaterina, Lindemann, Michael, Dziadek, Sebastian, Holiga, Štefan, Rukina, Daria, Kustermann, Thomas, Kerchner, Geoffrey A, Simuni, Tanya, Fontoura, Paulo, Umbricht, Daniel, Doody, Rachelle, Nikolcheva, Tania, Bonni, Azad, Investigators, PASADENA, Group, Prasinezumab Study, Marek, Kenneth, Postuma, Ronald B, Pavese, Nicola, Stocchi, Fabrizio, and Azulay, Jean-Philippe

Subjects
Dopamine Plasma Membrane Transport Proteins, Parkinson Disease, General Medicine, therapeutic use [Antibodies, Monoclonal, Humanized], Antibodies, Monoclonal, Humanized, drug therapy [Parkinson Disease], Antiparkinson Agents, Treatment Outcome, Double-Blind Method, therapeutic use [Dopamine Plasma Membrane Transport Proteins], alpha-Synuclein, Humans, ddc:610, antagonists & inhibitors [alpha-Synuclein], therapeutic use [Antiparkinson Agents]
Abstract

Aggregated α-synuclein plays an important role in the pathogenesis of Parkinson's disease. The monoclonal antibody prasinezumab, directed at aggregated α-synuclein, is being studied for its effect on Parkinson's disease.In this phase 2 trial, we randomly assigned participants with early-stage Parkinson's disease in a 1:1:1 ratio to receive intravenous placebo or prasinezumab at a dose of 1500 mg or 4500 mg every 4 weeks for 52 weeks. The primary end point was the change from baseline to week 52 in the sum of scores on parts I, II, and III of the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS; range, 0 to 236, with higher scores indicating greater impairment). Secondary end points included the dopamine transporter levels in the putamen of the hemisphere ipsilateral to the clinically more affected side of the body, as measured by 123I-ioflupane single-photon-emission computed tomography (SPECT).A total of 316 participants were enrolled; 105 were assigned to receive placebo, 105 to receive 1500 mg of prasinezumab, and 106 to receive 4500 mg of prasinezumab. The baseline mean MDS-UPDRS scores were 32.0 in the placebo group, 31.5 in the 1500-mg group, and 30.8 in the 4500-mg group, and mean (±SE) changes from baseline to 52 weeks were 9.4±1.2 in the placebo group, 7.4±1.2 in the 1500-mg group (difference vs. placebo, -2.0; 80% confidence interval [CI], -4.2 to 0.2; P = 0.24), and 8.8±1.2 in the 4500-mg group (difference vs. placebo, -0.6; 80% CI, -2.8 to 1.6; P = 0.72). There was no substantial difference between the active-treatment groups and the placebo group in dopamine transporter levels on SPECT. The results for most clinical secondary end points were similar in the active-treatment groups and the placebo group. Serious adverse events occurred in 6.7% of the participants in the 1500-mg group and in 7.5% of those in the 4500-mg group; infusion reactions occurred in 19.0% and 34.0%, respectively.Prasinezumab therapy had no meaningful effect on global or imaging measures of Parkinson's disease progression as compared with placebo and was associated with infusion reactions. (Funded by F. Hoffmann-La Roche and Prothena Biosciences; PASADENA ClinicalTrials.gov number, NCT03100149.).

Published
2022

40. Combination of Idasanutlin, Venetoclax and Obinutuzumab in Patients with Relapsed or Refractory (R/R) Non-Hodgkin Lymphoma (NHL): Results from a Phase I/II Study

Author

Kamdar, Manali, primary, Augustson, Bradley, additional, Do, Young Rok, additional, Edwards, James, additional, Hertzberg, Mark, additional, Johnston, Amanda, additional, Kim, Jin Seok, additional, Kroschinsky, Frank P., additional, Smith, Sonali M., additional, Stevens, Don A., additional, Monnet, Annabelle, additional, Zhao, Ye, additional, Nouet, Yann, additional, and Marinova, Viktoriya, additional

Published
2020
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41. The European politics of book pricing

Author

Littoz-Monnet, Annabelle

Subjects
Price fixing -- Political aspects, Political science, Regional focus/area studies, European Union -- Political aspects
Abstract

The reasons behind why and how the communitarisation of the policy debate on book prices took place in Europe are explained. It is argued that once the locus of the decision-making process had shifted de facto towards the EU level, the diversity of actors' preferences, decision rules and the nature of the status quo made it impossible for the tenants of dirigiste solutions to impose their policy preferences.

Published
2005

43. MIRROS: a randomized, placebo-controlled, Phase III trial of cytarabine ± idasanutlin in relapsed or refractory acute myeloid leukemia

Author

Montesinos, Pau, primary, Beckermann, Benjamin M, additional, Catalani, Olivier, additional, Esteve, Jordi, additional, Gamel, Katia, additional, Konopleva, Marina Y, additional, Martinelli, Giovanni, additional, Monnet, Annabelle, additional, Papayannidis, Cristina, additional, Park, Aaron, additional, Récher, Christian, additional, Rodríguez-Veiga, Rebeca, additional, Röllig, Christoph, additional, Vey, Norbert, additional, Wei, Andrew H, additional, Yoon, Sung-Soo, additional, and Fenaux, Pierre, additional

Published
2020
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