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5 results on '"Goyal, N.A."'

1. Efficacy and safety of Bimagrumab in sporadic inclusion body myositis

Author

Amato, A.A., Hanna, M.G., Machado, P.M., Badrising, U.A., Chinoy, H., Benveniste, O., Karanam, A.K., Wu, M., Tankó, L.B., Schubert-Tennigkeit, A.A., Papanicolaou, D.A., Lloyd, T.E., Needham, M., Liang, C., Reardon, K.A., de Visser, M., Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Van den Bergh, P., Baets, J., De Bleecker, J.L., Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Aoki, M., Katsuno, M., Morihata, H., Murata, K., Nodera, H., Nishino, I., Romano, C.D., Williams, V.S.L., Vissing, J., Zhang Auberson, L., Amato, A.A., Hanna, M.G., Machado, P.M., Badrising, U.A., Chinoy, H., Benveniste, O., Karanam, A.K., Wu, M., Tankó, L.B., Schubert-Tennigkeit, A.A., Papanicolaou, D.A., Lloyd, T.E., Needham, M., Liang, C., Reardon, K.A., de Visser, M., Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Van den Bergh, P., Baets, J., De Bleecker, J.L., Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Aoki, M., Katsuno, M., Morihata, H., Murata, K., Nodera, H., Nishino, I., Romano, C.D., Williams, V.S.L., Vissing, J., and Zhang Auberson, L.

Abstract

Objective To assess long-term (2 years) effects of bimagrumab in participants with sporadic inclusion body myositis (sIBM). Methods Participants (aged 36–85 years) who completed the core study (RESILIENT [Efficacy and Safety of Bimagrumab/BYM338 at 52 Weeks on Physical Function, Muscle Strength, Mobility in sIBM Patients]) were invited to join an extension study. Individuals continued on the same treatment as in the core study (10 mg/kg, 3 mg/kg, 1 mg/kg bimagrumab or matching placebo administered as IV infusions every 4 weeks). The co–primary outcome measures were 6-minute walk distance (6MWD) and safety. Results Between November 2015 and February 2017, 211 participants entered double-blind placebo-controlled period of the extension study. Mean change in 6MWD from baseline was highly variable across treatment groups, but indicated progressive deterioration from weeks 24–104 in all treatment groups. Overall, 91.0% (n = 142) of participants in the pooled bimagrumab group and 89.1% (n = 49) in the placebo group had ≥1 treatment-emergent adverse event (AE). Falls were slightly higher in the bimagrumab 3 mg/kg group vs 10 mg/kg, 1 mg/kg, and placebo groups (69.2% [n = 36 of 52] vs 56.6% [n = 30 of 53], 58.8% [n = 30 of 51], and 61.8% [n = 34 of 55], respectively). The most frequently reported AEs in the pooled bimagrumab group were diarrhea 14.7% (n = 23), involuntary muscle contractions 9.6% (n = 15), and rash 5.1% (n = 8). Incidence of serious AEs was comparable between the pooled bimagrumab and the placebo group (18.6% [n = 29] vs 14.5% [n = 8], respectively). Conclusion Extended treatment with bimagrumab up to 2 years produced a good safety profile and was well-tolerated, but did not provide clinical benefits in terms of improvement in mobility. The extension study was terminated early due to core study not meeting its primary endpoint.

Published
2021

2. A Phase 2, Double-Blind, randomized, Dose-Ranging trial Of Reldesemtiv in patients with ALS

Author

Shefner, J.M., Andrews, J.A., Genge, A., Jackson, C., Lechtzin, N., Miller, T.M., Cockroft, B.M., Meng, L., Wei, J., Wolff, A.A., Malik, F.I., Bodkin, C., Brooks, B.R., Caress, J., Dionne, A., Fee, D., Goutman, S.A., Goyal, N.A., Hardiman, O., Hayat, G., Heiman-Patterson, T., Heitzman, D., Henderson, R.D., Johnston, W., Karam, C., Kiernan, M.C., Kolb, S.J., Korngut, L., Ladha, S., Matte, G., Mora, J.S., Needham, M., Oskarsson, B., Pattee, G.L., Pioro, E.P., Pulley, M., Quan, D., Rezania, K., Schellenberg, K.L., Schultz, D., Shoesmith, C., Simmons, Z., Statland, J., Sultan, S., Swenson, A., Berg, L.H.V.D., Vu, T., Vucic, S., Weiss, M., Whyte-Rayson, A., Wymer, J., Zinman, L., Rudnicki, S.A., Shefner, J.M., Andrews, J.A., Genge, A., Jackson, C., Lechtzin, N., Miller, T.M., Cockroft, B.M., Meng, L., Wei, J., Wolff, A.A., Malik, F.I., Bodkin, C., Brooks, B.R., Caress, J., Dionne, A., Fee, D., Goutman, S.A., Goyal, N.A., Hardiman, O., Hayat, G., Heiman-Patterson, T., Heitzman, D., Henderson, R.D., Johnston, W., Karam, C., Kiernan, M.C., Kolb, S.J., Korngut, L., Ladha, S., Matte, G., Mora, J.S., Needham, M., Oskarsson, B., Pattee, G.L., Pioro, E.P., Pulley, M., Quan, D., Rezania, K., Schellenberg, K.L., Schultz, D., Shoesmith, C., Simmons, Z., Statland, J., Sultan, S., Swenson, A., Berg, L.H.V.D., Vu, T., Vucic, S., Weiss, M., Whyte-Rayson, A., Wymer, J., Zinman, L., and Rudnicki, S.A.

Abstract

To evaluate safety, dose response, and preliminary efficacy of reldesemtiv over 12 weeks in patients with amyotrophic lateral sclerosis (ALS). Methods: Patients (≤2 years since diagnosis) with slow upright vital capacity (SVC) of ≥60% were randomized 1:1:1:1 to reldesemtiv 150, 300, or 450 mg twice daily (bid) or placebo; active treatment was 12 weeks with 4-week follow-up. Primary endpoint was change in percent predicted SVC at 12 weeks; secondary measures included ALS Functional Rating Scale-Revised (ALSFRS-R) and muscle strength mega-score. Results: Patients (N = 458) were enrolled; 85% completed 12-week treatment. The primary analysis failed to reach statistical significance (p = 0.11); secondary endpoints showed no statistically significant effects (ALSFRS-R, p = 0.09; muscle strength mega-score, p = 0.31). Post hoc analyses pooling all active reldesemtiv-treated patients compared against placebo showed trends toward benefit in all endpoints (progression rate for SVC, ALSFRS-R, and muscle strength mega-score (nominal p values of 0.10, 0.01 and 0.20 respectively)). Reldesemtiv was well tolerated, with nausea and fatigue being the most common side effects. A dose-dependent decrease in estimated glomerular filtration rate was noted, and transaminase elevations were seen in approximately 5% of patients. Both hepatic and renal abnormalities trended toward resolution after study drug discontinuation. Conclusions: Although the primary efficacy analysis did not demonstrate statistical significance, there were trends favoring reldesemtiv for all three endpoints, with effect sizes generally regarded as clinically important. Tolerability was good; modest hepatic and renal abnormalities were reversible. The impact of reldesemtiv on patients with ALS should be assessed in a pivotal Phase 3 trial. (ClinicalTrials.gov Identifier: NCT03160898)

Published
2020

4. Safety and efficacy of intravenous bimagrumab in inclusion body myositis (RESILIENT): a randomised, double-blind, placebo-controlled phase 2b trial

Author

Hanna, M.G., Badrising, U.A., Benveniste, O., Lloyd, T.E., Needham, M., Chinoy, H., Aoki, M., Machado, P.M., Liang, C., Reardon, K.A., Visser, M. de, Ascherman, D.P., Barohn, R.J., Dimachkie, M.M., Miller, J.A.L., Kissel, J.T., Oskarsson, B., Joyce, N.C., Bergh, P. van den, Baets, J., Bleecker, J.L. de, Karam, C., David, W.S., Mirabella, M., Nations, S.P., Jung, H.H., Pegoraro, E., Maggi, L., Rodolico, C., Filosto, M., Shaibani, A.I., Sivakumar, K., Goyal, N.A., Mori-Yoshimura, M., Yamashita, S., Suzuki, N., Katsuno, M., Murata, K., Nodera, H., Romano, C.D., Williams, V.S.L., Vissing, J., Auberson, L.Z., Wu, M., Vera, A. de, Papanicolaou, D.A., Amato, A.A., Nishino, I., RESILIENT Study Grp, Neurology, ANS - Neuroinfection & -inflammation, UCL - SSS/IONS/NEUR - Clinical Neuroscience, UCL - (SLuc) Service de neurologie, and RESILIENT Study Grp

Subjects
Adult, Male, 0301 basic medicine, medicine.medical_specialty, Myostatin, Guidelines, Population, Guidelines, Antibodies, Monoclonal, Humanized, Placebo, Myositis, Inclusion Body, law.invention, 03 medical and health sciences, 0302 clinical medicine, Double-Blind Method, Randomized controlled trial, law, Internal medicine, medicine, Humans, education, Adverse effect, Bimagrumab, Aged, Aged, 80 and over, education.field_of_study, business.industry, Repeated measures design, Middle Aged, Myostatin, Settore MED/26 - NEUROLOGIA, Treatment Outcome, 030104 developmental biology, Blood chemistry, Tolerability, Female, Human medicine, Neurology (clinical), business, 030217 neurology & neurosurgery
Abstract

Background Inclusion body myositis is an idiopathic inflammatory myopathy and the most common myopathy affecting people older than 50 years. To date, there are no effective drug treatments. We aimed to assess the safety, efficacy, and tolerability of bimagrumab-a fully human monoclonal antibody-in individuals with inclusion body myositis. Methods We did a multicentre, double-blind, placebo-controlled study (RESILIENT) at 38 academic clinical sites in Australia, Europe, Japan, and the USA. Individuals (aged 3685 years) were eligible for the study if they met modified 2010 Medical Research Council criteria for inclusion body myositis. We randomly assigned participants (1:1:1:1) using a blocked randomisation schedule (block size of four) to either bimagrumab (10 mg/kg, 3 mg/kg, or 1 mg/kg) or placebo matched in appearance to bimagrumab, administered as intravenous infusions every 4 weeks for at least 48 weeks. All study participants, the funder, investigators, site personnel, and people doing assessments were masked to treatment assignment. The primary outcome measure was 6-min walking distance (6MWD), which was assessed at week 52 in the primary analysis population and analysed by intention-to-treat principles. We used a multivariate normal repeated measures model to analyse data for 6MWD. Safety was assessed by recording adverse events and by electrocardiography, echocardiography, haematological testing, urinalysis, and blood chemistry. This trial is registered with ClinicalTrials.gov, number NCT01925209; this report represents the final analysis. Findings Between Sept 26, 2013, and Jan 6, 2016, 251 participants were enrolled to the study, of whom 63 were assigned to each bimagrumab group and 62 were allocated to the placebo group. At week 52, 6MWD change from baseline did not differ between any bimagrumab dose and placebo (least squares mean treatment difference for bimagrumab 10 mg/kg group, 17.6 m, SE 14.3, 99% CI -19.6 to 54.8; p=0.22; for 3 mg/kg group, 18.6 m, 14.2, -18.2 to 55.4; p=0.19; and for 1 mg/kg group, 1.3 m, 14.1, -38.0 to 35.4; p=0.93). 63 (100%) participants in each bimagrumab group and 61 (98%) of 62 in the placebo group had at least one adverse event. Falls were the most frequent adverse event (48 [76%] in the bimagrumab 10 mg/kg group, 55 [87%] in the 3 mg/kg group, 54 [86%] in the 1 mg/kg group, and 52 [84%] in the placebo group). The most frequently reported adverse events with bimagrumab were muscle spasms (32 [51%] in the bimagrumab 10 mg/kg group, 43 [68%] in the 3 mg/kg group, 25 [40%] in the 1 mg/kg group, and 13 [21%] in the placebo group) and diarrhoea (33 [52%], 28 [44%], 20 [32%], and 11 [18%], respectively). Adverse events leading to discontinuation were reported in four (6%) participants in each bimagrumab group compared with one (2%) participant in the placebo group. At least one serious adverse event was reported by 21 (33%) participants in the 10 mg/kg group, 11 (17%) in the 3 mg/kg group, 20 (32%) in the 1 mg/kg group, and 20 (32%) in the placebo group. No significant adverse cardiac effects were recorded on electrocardiography or echocardiography. Two deaths were reported during the study, one attributable to subendocardial myocardial infarction (secondary to gastrointestinal bleeding after an intentional overdose of concomitant sedatives and antidepressants) and one attributable to lung adenocarcinoma. Neither death was considered by the investigator to be related to bimagrumab. Interpretation Bimagrumab showed a good safety profile, relative to placebo, in individuals with inclusion body myositis but did not improve 6MWD. The strengths of our study are that, to the best of our knowledge, it is the largest randomised controlled trial done in people with inclusion body myositis, and it provides important natural history data over 12 months. Copyright (C) 2019 Elsevier Ltd. All rights reserved.

Published
2019

5. Predicting Outcome in Guillain-Barré Syndrome

Author

Doets, Alex Y., Lingsma, Hester F., Walgaard, Christa, Islam, Badrul, Papri, Nowshin, Davidson, Amy, Yamagishi, Yuko, Kusunoki, Susumu, Dimachkie, Mazen M., Waheed, Waqar, Kolb, Noah, Islam, Zhahirul, Mohammad, Quazi Deen, Harbo, Thomas, Sindrup, Soren H., Chavada, Govindsinh, Willison, Hugh J., Casasnovas, Carlos, Bateman, Kathleen, Miller, James A.L., van den Berg, Bianca, Verboon, Christine, Roodbol, Joyce, Leonhard, Sonja E., Benedetti, Luana, Kuwabara, Satoshi, Van den Bergh, Peter, Monges, Soledad, Marfia, Girolama A., Shahrizaila, Nortina, Galassi, Giuliana, Péréon, Yann, Bürmann, Jan, Kuitwaard, Krista, Kleyweg, Ruud P., Marchesoni, Cintia, Sedano Tous, María J., Querol, Luis, Illa, Isabel, Wang, Yuzhong, Nobile-Orazio, Eduardo, Rinaldi, Simon, Schenone, Angelo, Pardo, Julio, Vermeij, Frederique H., Lehmann, Helmar C., Granit, Volkan, Cavaletti, Guido, Gutiérrez-Gutiérrez, Gerardo, Barroso, Fabio A., Visser, Leo H., Katzberg, Hans D., Dardiotis, Efthimios, Attarian, Shahram, van der Kooi, Anneke J., Eftimov, Filip, Wirtz, Paul W., Samijn, Johnny P.A., Gilhuis, H. Jacobus, Hadden, Robert D.M., Holt, James K.L., Sheikh, Kazim A., Karafiath, Summer, Vytopil, Michal, Antonini, Giovanni, Feasby, Thomas E., Faber, Catharina G., Gijsbers, Cees J., Busby, Mark, Roberts, Rhys C., Silvestri, Nicholas J., Fazio, Raffaella, van Dijk, Gert W., Garssen, Marcel P.J., Straathof, Chiara S.M., Gorson, Kenneth C., Jacobs, Bart C., Hughes, R.A.C., Cornblath, D.R., Hartung, H.P., van Doorn, P.A., de Koning, L.C., van Woerkom, M., Mandarakas, M., MPhty, BHIthSci(Hons), Reisin, R.C., Reddel, S.W., Ripellino, P., Hsieh, S.T., Addington, J.M., Ajroud-Driss, S., Andersen, H., Badrising, U.A., Bella, I.R., Bertorini, T.E., Bhavaraju-Sanka, R., Bianco, M., Brannagan, T.H., Briani, Chiara, Butterworth, S., Chao, C.C., Chen, S., Claeys, K.G., Conti, M.E., Cosgrove, J.S., Dalakas, M.C., Dornonville de la Cour, C., Echaniz-Laguna, A., Fehmi, J., Fokke, C., Fujioka, T., Fulgenzi, E.A., García-Sobrino, T., Gilchrist, J.M., Goldstein, J.M., Goyal, N.A., Grisanti, S.G., Gutman, L., Holbech, J.V., Homedes, C., Htut, M., Jellema, K., Pascual, I. Jericó, JimenoMontero, M.C., Kaida, K., Khoshnoodi, M., Kiers, L., Kimpinski, K., Köhler, A.A., Kokubun, N., Kuwahara, M., Kwan, J.Y., Ladha, S.S., Lassen, L. Landschoff, Lawson, V., Pan, E.B. Lee, Cejas, L. Léon, Lunn, M.P.T., Magot, A., Manji, H., Infante, C. Márquez, Martín-Aguilar, L., Hernandez, E. Martinez, Mataluni, G., Mattiazzi, M.G., McDermott, C.J., Meekins, G.D., Morís de la Tassa, G., Nascimbene, C., Nowak, R.J., Osei-Bonsu, M., Pascuzzi, R.M., Prada, V., Rojas-Marcos, I., Rudnicki, S.A., Sachs, G.M., Samukawa, M., Santoro, L., Savransky, A.G., Schwindling, L., Sekiguchi, Y., Sommer, C.L., Spyropoulos, A., Stein, B., Stino, A.M., Tan, C.Y., Tankisi, H., Twydell, P.T., van Damme, P., van der Ree, T., van Koningsveld, R., Varrato, J.D., Xing, C., Zhou, L., and Zivkovic, S.

Published
2022
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