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2. Effect of the mGluR5-NAM Basimglurant on Behavior in Adolescents and Adults with Fragile X Syndrome in a Randomized, Double-Blind, Placebo-Controlled Trial: FragXis Phase 2 Results.

Author

Youssef, Eriene A, Berry-Kravis, Elizabeth, Czech, Christian, Hagerman, Randi J, Hessl, David, Wong, Chin Y, Rabbia, Michael, Deptula, Dennis, John, Amy, Kinch, Russell, Drewitt, Philip, Lindemann, Lothar, Marcinowski, Moritz, Langland, Rachel, Horn, Carsten, Fontoura, Paulo, Santarelli, Luca, Quiroz, Jorge A, and FragXis Study Group

Subjects
FragXis Study Group, Humans, Fragile X Syndrome, Imidazoles, Pyridines, RNA, Messenger, Excitatory Amino Acid Antagonists, Psychotropic Drugs, Treatment Failure, Double-Blind Method, DNA Methylation, Adolescent, Adult, Middle Aged, Female, Male, Fragile X Mental Retardation Protein, Young Adult, Receptor, Metabotropic Glutamate 5, Clinical Research, Intellectual and Developmental Disabilities (IDD), Rare Diseases, Clinical Trials and Supportive Activities, Pediatric, Behavioral and Social Science, Neurosciences, Mental Health, Brain Disorders, Depression, 6.1 Pharmaceuticals, Mental health, Psychiatry, Medical and Health Sciences, Psychology and Cognitive Sciences
Abstract

Preclinical data suggest that inhibition of the metabotropic glutamate receptor 5 (mGluR5) receptor might hold therapeutic benefits in Fragile X syndrome (FXS). Treatment of Fmr1 knockout mice with mGluR5-negative allosteric modulators (NAMs) has been reported to correct a broad range of phenotypes related to FXS. The early short-term clinical trials with mGluR5 NAMs, including basimglurant, assessing the effects in individuals with FXS, were supportive of further exploration in larger, well-controlled trials. We evaluated basimglurant, a potent and selective mGluR5 NAM, in a 12-week, double-blind, parallel-group study of 183 adults and adolescents (aged 14-50, mean 23.4 years) with FXS. Individuals with an FMR1 full mutation were randomized to placebo or one of two doses of basimglurant. The primary efficacy endpoint was the change from baseline in behavioral symptoms using the Anxiety Depression and Mood Scale (ADAMS) total score. All treatment arms showed marked behavioral improvements from baseline to week 12 with less improvement in the basimglurant 1.5 mg arm than placebo; however, basimglurant 0.5 mg was inferior to placebo in the ADAMs total score. Treatment with basimglurant was overall well-tolerated. A higher incidence of adverse events classified as psychiatric disorders were reported in patients treated with basimglurant, including three patients with hallucinations or psychosis. In this phase 2 clinical trial, basimglurant did not demonstrate improvement over placebo. Evaluation of the overall risk-benefit in younger patient populations is an important consideration for the design of potential further investigations of efficacy with this class of medications.

Published
2018

4. RAINBOWFISH: Primary Efficacy and Safety Data in Risdiplam-treated Infants with Presymptomatic Spinal Muscular Atrophy (SMA) (S37.006)

Author

Servais, Laurent, primary, Farrar, Michelle, additional, Finkel, Richard, additional, Vlodavets, Dmitry, additional, Zanoteli, Edmar, additional, Al-Muhaizea, Mohammad, additional, de Queiroz Campos Araújo, Alexandra Prufer, additional, Nelson, Leslie, additional, Jaber, Birgit, additional, Gorni, Ksenija, additional, Kletzl, Heidemarie, additional, Palfreeman, Laura, additional, Gaki, Eleni, additional, Rabbia, Michael, additional, Summers, Dave, additional, Fontoura, Paulo, additional, and Bertini, Enrico, additional

Published
2024
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7. Stride Velocity 95thCentile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy

Author

Rabbia, Michael, Guridi Ormazabal, Maitea, Staunton, Hannah, Veenstra, Klaas, Eggenspieler, Damien, Annoussamy, Mélanie, Servais, Laurent, and Strijbos, Paul

Abstract

Background: Stride Velocity 95thCentile (SV95C) is the first wearable device-derived clinical outcome assessment (COA) to receive European Medicines Agency (EMA) qualification as a primary endpoint in ambulant patients with Duchenne muscular dystrophy (DMD) aged =4 years.Objective: To compare SV95C—in its first-ever clinical trial application as a secondary endpoint—with established motor function COAs used in the trial (Four-Stair Climb [4SC] velocity, North Star Ambulatory Assessment [NSAA], and Six-Minute Walk Distance [6MWD]).Methods: SV95C was a secondary endpoint in a subset (n?=?47) of participants in the SPITFIRE/WN40227 trial of taldefgrobep alfa, which was discontinued due to lack of clinical benefit. Participants in the =48-week SV95C sub-study were 6–11 years old and received corticosteroids for =6 months pre-treatment. Pearson correlations were used to compare SV95C with the other COAs. Responsiveness and changes over time were respectively assessed via standardized response means (SRMs) based on absolute changes and mixed models for repeated measures.Results: SV95C change at Week 24 was –0.07?m/s, with limited variability (standard deviation: 0.16, n?=?27). The SRM for SV95C indicated moderate responsiveness to clinical change at the earliest timepoint (Week 12, n?=?46), while those of the other COAs did not indicate moderate responsiveness until Week 36 (6MWD, n?=?33) or Week 48 (4SC velocity, n?=?20; NSAA total score, n?=?20). Baseline correlations between SV95C and other COAs were strong (r?=?0.611–0.695). Correlations between SV95C change from baseline to Week 48 and changes in other COAs were moderate to strong (r?=?0.443–0.678).?Conclusions: Overall, SV95C demonstrated sensitivity to ambulatory decline over short intervals, low variability, and correlation with established COAs. Although the negative trial precluded demonstration of SV95C’s sensitivity to drug effect, these findings support the continued use of SV95C in DMD clinical trials.

Published
2024
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8. Evaluating the Safety and Efficacy of Crenezumab vs Placebo in Adults With Early Alzheimer Disease

Author

Ostrowitzki, Susanne, primary, Bittner, Tobias, additional, Sink, Kaycee M., additional, Mackey, Howard, additional, Rabe, Christina, additional, Honig, Lawrence S., additional, Cassetta, Emanuele, additional, Woodward, Michael, additional, Boada, Mercè, additional, van Dyck, Christopher H., additional, Grimmer, Timo, additional, Selkoe, Dennis J., additional, Schneider, Andres, additional, Blondeau, Kathleen, additional, Hu, Nan, additional, Quartino, Angelica, additional, Clayton, David, additional, Dolton, Michael, additional, Dang, Yifan, additional, Ostaszewski, Beth, additional, Sanabria-Bohórquez, Sandra M., additional, Rabbia, Michael, additional, Toth, Balazs, additional, Eichenlaub, Udo, additional, Smith, Jillian, additional, Honigberg, Lee A., additional, and Doody, Rachelle S., additional

Published
2022
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15. Effect of the dual peroxisome proliferator-activated receptor-[alpha]/[gamma] agonist aleglitazar on risk of cardiovascular disease in patients with type 2 diabetes (SYNCHRONY): a phase II, randomised, dose-ranging study

Author

Henry, Robert R., Lincoff, A Michael, Mudaliar, Sunder, Rabbia, Michael, Chognot, Cathy, and Herz, Matthias

Subjects
Cardiovascular diseases -- Risk factors, Cardiovascular diseases -- Research, Hypoglycemic agents -- Health aspects, Type 2 diabetes -- Drug therapy, Type 2 diabetes -- Complications and side effects, Type 2 diabetes -- Research
Published
2009

17. P1‐046: BASELINE CHARACTERISTICS FROM CREAD2: A PHASE III TRIAL OF CRENEZUMAB IN EARLY (PRODROMAL‐TO‐MILD) ALZHEIMER'S DISEASE

Author

Sink, Kaycee, primary, Warren, Francis, additional, Smith, Jillian, additional, Schneider, Andres, additional, Fuji, Reina N., additional, Quartino, Angelica, additional, Mackey, Howard, additional, Rabbia, Michael, additional, Yule, Susan, additional, Fontoura, Paulo, additional, Doody, Rachelle, additional, and Ostrowitzki, Susanne, additional

Published
2019
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19. Baseline Characteristics from a Phase III Trial of Crenezumab in Early (Prodromal-to-Mild) Alzheimer’s Disease (CREAD) (P4.1-002)

Author

Sink, Kaycee M., primary, Ostrowitzki, Susanne, additional, Millar, Laurie, additional, Warren, Francis, additional, Lin, Helen, additional, Smith, Jillian, additional, Schneider, Andres, additional, Fuji, Reina, additional, Quartino, Angelica, additional, Mackey, Howard, additional, Rabbia, Michael, additional, Yule, Susan, additional, Fontoura, Paulo, additional, and Doody, Rachelle, additional

Published
2019
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20. A Phase 1b/2 Study of the Anti-Myostatin Adnectin RG6206 (BMS-986089) in Ambulatory Boys with Duchenne Muscular Dystrophy: A 72-Week Treatment Update (P1.6-062)

Author

Wagner, Kathryn R., primary, Wong, Brenda L., additional, Byrne, Barry J., additional, Tian, Cuixia, additional, Jacobsen, Leslie K., additional, Tirucherai, Giridhar S., additional, Rabbia, Michael, additional, Kletzl, Heidemarie, additional, Dukart, Juergen, additional, Ong, Rose, additional, Yen, Karl, additional, Sajeev, Gautam, additional, Signorovitch, James, additional, Ward, Susan J., additional, Bechtold, Cliff, additional, and Krishnan, Michelle, additional

Published
2019
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21. O1‐02‐04: BASELINE CHARACTERICS FROM A PHASE 3 TRIAL OF CRENEZUMAB IN PRODROMAL TO MILD ALZHEIMER'S DISEASE (CREAD)

Author

Lin, Helen, primary, Ostrowitzki, Susanne, additional, Sink, Kaycee M., additional, Millar, Laurie, additional, Warren, Francis, additional, Smith, Jillian, additional, Schneider, Andres, additional, Fuji, Reina N., additional, Quartino, Angelica, additional, Mackey, Howard, additional, Rabbia, Michael A., additional, Yule, Susan, additional, Fontoura, Paulo, additional, and Doody, Rachelle, additional

Published
2018
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22. A randomized, placebo-controlled, double-blind, Phase 1b/2 study of the novel antimyostatin adnectin RG6206 (BMS-986089) in ambulatory boys with Duchenne muscular dystrophy (P5.431)

Author

Wagner, Kathryn, primary, Wong, Brenda, additional, Byrne, Barry, additional, Sweeney, Lee, additional, Jacobsen, Leslie, additional, Tirucherai, Giridhar, additional, Rabbia, Michael, additional, Buchbjerg, Jeppe, additional, Krishnan, Michelle, additional, and Bechtold, Clifford, additional

Published
2018
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24. Disease Modification in Alzheimer’s Disease: Current Thinking

Author

Liu-Seifert, Hong, Schumi, Jennifer, Miao, Xiaopeng, Tian, Ying, Rabbia, Michael, Andersen, Scott W., Wilson, Steve, Li, Wen, and Entsuah, Richard

Abstract

Alzheimer’s disease (AD) has increasingly been recognized as a huge unmet medical need. Currently, there is no approved drug to cure, prevent, or even slow down the disease. It is imperative to develop disease-modifying treatments for AD to alter the underlying disease progression. This paper reviews the most up-to-date regulatory guidance on how to demonstrate disease modification and provides an overview of available methodologies and applications to clinical trials. The intent is to assist the field with future clinical trials designed to demonstrate disease-modifying effect in AD. The methodologies may be generalizable to broader neurodegenerative diseases.

Published
2024
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26. P2‐003: Clinical Trial Design of Cread: A Randomized, Double‐Blind, Placebo‐Controlled, Parallel‐Group Phase 3 Study to Evaluate Crenezumab Treatment in Patients with Prodromal‐To‐Mild Alzheimer’s Disease

Author

Blaettler, Thomas, primary, Smith, Janice, additional, Smith, Jillian, additional, Paul, Robert, additional, Asnaghi, Veronica, additional, Fuji, Reina, additional, Quartino, Angelica, additional, Honigberg, Lee, additional, Rabbia, Michael A., additional, Yule, Susan, additional, Ostrowitzki, Susanne, additional, and Fontoura, Paulo, additional

Published
2016
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30. 531 High Dose Combination Therapy (Peginterferon Alfa-2a Plus Ribavirin) in Difficult-to-Cure Patients With Chronic Hepatitis C: Impact on Rvr, Cevr and SVR

Author

Reddy, Rajender, primary, Shiffman, Mitchell L., additional, Rodriguez-Torres, Maribel, additional, Abdurakhmanov, Djamal, additional, Bakulin, Igor G., additional, Silva, Giovanni Faria, additional, Cheinquer, Hugo, additional, Rabbia, Michael A., additional, Mckenna, Michael, additional, Tietz, Andreas, additional, and Harrison, Stephen A., additional

Published
2010
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32. Efficacy and safety of balovaptan for posttraumatic stress disorder: A randomized, placebo-controlled trial.

Author

Marler S, Rabbia M, Sanders K, Derks M, Bailey L, Vilimovskij A, Maurer J, Nordstroem AL, and Guthrie H

Abstract

Posttraumatic stress disorder (PTSD) has a significant impact on quality of life and affects more than 13 million individuals in the United States, with limited treatments available. EXUVIA (NCT05401565) was a Phase 2, randomized, placebo-controlled, double-blind trial, conducted across eight sites in the United States. The study aimed to assess the efficacy, safety, and pharmacokinetics of balovaptan, a highly selective vasopressin 1a receptor antagonist, in adults with PTSD. Between August 2022 and October 2023, a total of 57 adult participants (aged 18-60 years) were screened, and 29 participants were randomly allocated (1:1) to receive either balovaptan (13/29 [44.8%]) or placebo (16/29 [55.2%]). No meaningful differences were observed for balovaptan (-17.2 [± 10.7]) versus placebo (-15.6 [± 10.7]) as measured by the primary endpoint of change from baseline at Week 12 in Clinician-Administered PTSD Scale for Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition total symptom severity score. No meaningful differences for balovaptan versus placebo were observed at Week 12 for any secondary endpoints. Balovaptan was well tolerated with no new safety findings. The number of participants with at least one adverse event of any intensity was 9/13 (69.2%) in the balovaptan group and 7/16 (43.8%) in the placebo group. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Published
2024
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