E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Sporadic Inclusion Body Myositis
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Miosite sporadica da corpi inclusi |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10066407 |
E.1.2 | Term | Inclusion body myositis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10066407 |
E.1.2 | Term | Inclusion body myositis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the long-term safety and tolerability of BYM338 |
Valutare la sicurezza e la tollerabilità a lungo termine di BYM338 |
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E.2.2 | Secondary objectives of the trial |
- To describe the long-term evolution of quadriceps muscle strength using quadriceps Quantitative Muscle Testing (QMT) - To describe physical function reported by patients using the Sporadic Inclusion Body Myositis Functional Assessment (sIFA) - To report the incidence of self-reported falls and self-reported injurious falls (falls that result in any subject injury) which are a subset of all selfreported falls - To describe physical performance using the Short Physical Performance Battery (SPPB) - To characterize muscle changes using magnetic resonance imaging (MRI) from a subset of patients - To investigate the development of immunogenicity against BYM338 |
- Descrivere i cambiamenti sul lungo periodo della forza dei quadricipiti mediante il test muscolare quantitative (QMT) - Descrivere la funzionalità fisica riferita dai pazienti mediante il Sporadic Inclusion Body Myositis Functional Assessment (sIFA) - Riportare l’incidenza delle cadute riferite dai pazienti e delle cadute e delle cadute “dannose” riferite dai pazienti (cadute che hanno portato ad un danno del paziente) - Descrivere la prestazione fisica mediante la Short Physical Performance Battery (SPPB) - Caratterizzare i cambiamenti muscolari tramite la risonanza magnetica nucleare in un sottogruppo di pazienti - Indagare lo sviluppo dell’immunogenicità verso bimagrumab |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients who completed the core study - Written informed consent must be obtained before any extension study assessment is performed - Able to communicate well with the investigator - Willing to participate for the entire duration of the extension study with commitment to follow study requirements and procedures. |
Pazienti di sesso maschile e femminile che hanno completato lo studio principale, ossia che hanno ricevuto la dose di farmaco alla Settimana 48 e hanno completato la visita di fine studio e di follow-up dello studio principale. - Consenso informato scritto ottenuto prima dell’effettuazione di qualsiasi valutazione - Pazienti in grado di comunicare bene con lo sperimentatore - Pazienti disponibili a partecipare all’intera durata dello studio di estensione con impegno a seguire tutti i requisiti e le procedure dello studio. |
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E.4 | Principal exclusion criteria |
- Women who are pregnant - Women of child-bearing potential unless they are using highly effective methods of contraception during dosing and for 6 months after the last BYM338 dose - Current use of prohibited treatments - History of severe hypersensitivity reaction in the core study - History of adverse event(s) (including those from the core study) prior to the start of study drug in the extension study that, in the judgment of the investigator, taking into account the subject's overall status, prevent the subject from entering the extension study - Clinically significant abnormal liver function tests - Any medical condition or laboratory finding which, in the opinion of the investigator may interfere with participation in the study, might confound the results of the study, or pose an additional safety risk in administering BYM338 Other protocol-defined exclusion criteria may apply. |
- Gravidanza: test di gravidanza sul siero positivo (hcG) - Pazienti di sesso femminile potenzialmente in grado di avere figli, definiti come tutti i soggetti di sesso femminile fisiologicamente in grado di iniziare una gravidanza, a meno che stiano usando metodi di contraccezione altamente efficaci durante il trattamento e per 6 mesi dopo l’ultima dose di BYM338 - Uso di farmaci concomitanti non consentiti - Anamnesi positiva per ipersensibilità severa durante lo studio principale - Storia di eventi avversi (inclusi quelli occorsi nello studio principale) prima dell’inizio del farmaco in studio nello studio di estensione, che, a giudizio dello sperimentatore, considerando lo stato generale del paziente, impedisca l’ingresso nello studio di estensione. - Anomalie significative nei parametri di funzionalità epatica, come SGOT (AST), SGPT (ALT), fosfatasi alcalina, o bilirubina (ad eccezione della sindrome di Gilbert), che avrebbero portato all’interruzione del trattamento durante lo studio principale secondo il protocollo - Qualsiasi condizione medica o anomalia negli esami di laboratorio (per esempio valori di laboratorio inaspettati o clinicamente significativi, ECG, ecc), che, a giudizio dello sperimentatore, potrebbe interferire con la partecipazione allo studio, potrebbe confonderne i risultati, o rappresentare un rischio aggiuntivo nella somministrazione di BYM338. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Safety and tolerabilty of different i.v. BYM338 doses - Change from baseline in 6 Minute Walking Distance Test (6MWD) |
- Sicurezza e tollerabilità di dose diverse di BYM338 e.v. - Test del cammino in 6 minuti (6MWD) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline, 1 and 2 years |
Basale, 1 e 2 anni |
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E.5.2 | Secondary end point(s) |
- Change from baseline in quadriceps muscle strength - Change from baseline in patient-reported physical performance - Incidence of patients with self-reported falls and self reported injurious falls - Change from baseline in physical performance - Change in muscles of the thigh - Number of patients who develop immunogenicity against BYM338 |
- Descrivere i cambiamenti sul lungo periodo della forza dei quadricipiti mediante il test muscolare quantitative (QMT) - Descrivere la funzionalità fisica riferita dai pazienti mediante il Sporadic Inclusion Body Myositis Functional Assessment (sIFA) - Riportare l’incidenza delle cadute riferite dai pazienti e delle cadute e delle cadute “dannose” riferite dai pazienti (cadute che hanno portato ad un danno del paziente) - Descrivere la prestazione fisica mediante la Short Physical Performance Battery (SPPB) - Caratterizzare i cambiamenti muscolari tramite la risonanza magnetica nucleare in un sottogruppo di pazienti - Indagare lo sviluppo dell’immunogenicità verso bimagrumab |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Change from baseline in quadriceps muscle strength: Baseline, 1 and 2 years - Change from baseline in patient-reported physical performance: Baseline, 1 and 2 years - Incidence of patients with self-reported falls and self reported injurious falls: Baseline, 1 and 2 years - Change from baseline in physical performance: Baseline, 1 and 2 years - Change in muscles of the thigh: 1 and 2 years - Number of patients who develop immunogenicity against BYM338: 2 years |
- Change from baseline in quadriceps muscle strength: Baseline, 1 and 2 years - Change from baseline in patient-reported physical performance: Baseline, 1 and 2 years - Incidence of patients with self-reported falls and self reported injurious falls: Baseline, 1 and 2 years - Change from baseline in physical performance: Baseline, 1 and 2 years - Change in muscles of the thigh: 1 and 2 years - Number of patients who develop immunogenicity against BYM338: 2 years |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Periodo di Trattamento 2 epoch dello studio in aperto senza placebo |
Treatment Period 2 epoch of the study is open-label without placebo |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Belgium |
Denmark |
France |
Italy |
Japan |
Netherlands |
Switzerland |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |