E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
EOSINOPHILIC ESOPHAGITIS |
ESOFAGITIS EOSINOFÍLICA |
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E.1.1.1 | Medical condition in easily understood language |
Eosinophilic esophagitis (EoE) is a chronic disease that affects food intake and quality of life. |
La esofagitis eosinofílica (EEo) es una enfermedad crónica que afecta a la ingesta de comida y a la calidad de vida. |
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E.1.1.2 | Therapeutic area | Body processes [G] - Digestive System and Oral Physiological Phenomena [G10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064220 |
E.1.2 | Term | Eosinophilic esophagitis |
E.1.2 | System Organ Class | 100000004856 |
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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 CC-93538 in subjects with eosinophilic esophagitis (EoE). |
Evaluar la seguridad y la tolerabilidad a largo plazo de CC-93538 en sujetos con esofagitis eosinofílica (EEo). |
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E.2.2 | Secondary objectives of the trial |
To characterize the immunogenicity profile of CC-93538 following long-term treatment. |
Determinar el perfil de inmunogenicidad de CC-93538 después del tratamiento a largo plazo. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Title: Endolumenal Functional Lumen Imaging Probe (EndoFLIP TM) Sub-study Objective: An optional sub-study will be performed at a subset of clinical sites utilizing an Endolumenal Functional Lumen Imaging Probe (EndoFLIP) to evaluate the effects of CC-93538 on esophageal distensibility. |
Título: Subestudio de sonda de imagen funcional endoluminal (EndoFLIP TM) Objetivo: Se realizará un subestudio opcional en un subgrupo de centros clínicos que utilicen una sonda de imagen funcional endoluminal (EndoFLIP) para evaluar los efectos de CC-93538 sobre la distensibilidad esofágica. |
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E.3 | Principal inclusion criteria |
• Previously participated in prior clinical study CC-93538-EE-001 and either: - completed both the Induction and Maintenance phases; or - completed the Induction Phase, however, did not qualify for the Maintenance Phase, including having a severe EoE flare requiring endoscopic intervention or rescue therapy. • Demonstrated compliance with required investigational product dosing during the prior study. • Did not permanently discontinue investigational product in the prior study and/or did not experience any clinically significant adverse events related to Investigational Product that would preclude further dosing. • Females of childbearing potential must have a negative pregnancy test prior to the first dose of open-label CC-93538 and agree to practice a highly effective method of contraception (as defined in the prior study) until 5 months after the last dose of open-label CC-93538. |
• Participación previa en el estudio clínico CC-93538-EE-001 y cumplir una de las dos condiciones siguientes: - haber completado las fases de inducción y mantenimiento; o - haber completado la fase de inducción, pero no cumplir los requisitos para la fase de mantenimiento, incluida una exacerbación grave de la esofagitis eosinofílica con necesidad de intervención endoscópica o tratamiento de rescate. • Cumplimiento demostrado de la administración obligatoria del producto en investigación durante el estudio anterior. • No haber suspendido permanentemente el producto en investigación en el estudio anterior y/o no haber experimentado ningún acontecimiento adverso de importancia clínica relacionado con el producto en investigación que impida continuar la administración. • Las mujeres en edad fértil deberán tener una prueba de embarazo negativa antes de la primera dosis de CC-93538 en régimen abierto y comprometerse a utilizar un método anticonceptivo muy eficaz (según la definición del estudio anterior) hasta 5 meses después de la última dosis de CC-93538 en régimen abierto. |
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E.4 | Principal exclusion criteria |
• Clinical or endoscopic evidence of other diseases or conditions that may affect or confound the histologic, endoscopic, or clinical symptom evaluation for this study. • Active Helicobacter pylori infection or esophageal varices. • Evidence of immunosuppression, or of having received systemic immunosuppressive or immunomodulating drugs within 5 drug half-lives prior to open-label extension study (OLE) Day 1. Use of these agents is prohibited during the study. • Treatment with oral or sublingual immunotherapy within 6 months of OLE Day 1. Use of these agents is prohibited during the study. • Received an investigational product, other than that administered in CC-93538-EE-001, within 5 half-lives prior to OLE Day 1 (includes investigational product received during an interventional trial for COVID-19). Those vaccinated with an investigational COVID-19 vaccine during CC-93538-EE-001 are not eligible, unless allowed following a discussion with the Clinical Trial Physician. • Received a live attenuated vaccine within one month prior to OLE Day 1; or anticipates the need for a live attenuated vaccine at any time throughout the course of this study. • Any disease that would affect the conduct of the protocol or interpretation of the study results, or would put a patient at risk by participating in the study (e.g. colitis, celiac disease, Mendelian disorder associated with EoE, severe uncontrolled asthma, infection causing eosinophilia, hypereosinophilic syndrome, or cardiovascular condition, or neurologic or psychiatric illness that could compromise the subject's ability to accurately document symptoms of EoE; newly diagnosed malignancy, lymphoproliferative disease, or clinically significant laboratory abnormality). • Active or ongoing infections including parasitic/helminthic infections, viral hepatitis, tuberculosis, or HIV. • Has had idiopathic anaphylaxis or major immunologic reaction to an immunoglobulin-G containing agent; or any known hypersensitivity to any ingredient in CC-93538. • Females who are pregnant or lactating |
• Pruebas clínicas o endoscópicas de otras enfermedades que pudieran afectar a la evaluación histológica, endoscópica o clínica de los síntomas de este estudio. • Infección activa por Helicobacter pylori o varices esofágicas. • Signos de inmunodepresión o haber recibido inmunodepresores o inmunomoduladores sistémicos en las 5 semividas previas al día 1 del estudio de extensión abierto (ERA). El uso de estos fármacos está prohibido durante el estudio. • Tratamiento con inmunoterapia oral o sublingual en los 6 meses previos al día 1 de la extensión abierta. El uso de estos fármacos está prohibido durante el estudio. • Haber recibido un producto en investigación, distinto del administrado en CC-93538-EE-001, en el plazo de 5 semividas antes del día 1 de la extensión abierta (incluye el producto en investigación recibido durante un ensayo intervencionista de COVID-19). Los pacientes vacunados con una vacuna experimental contra la COVID-19 durante el estudio CC-93538-EE-001 no podrán participar, a menos que se permita después de una conversación con el médico del ensayo clínico. • Recepción de una vacuna de microorganismos vivos atenuados en el mes previo al día 1 de la extensión abierta o previsión de la necesidad de una vacuna de microorganismos vivos atenuados en cualquier momento durante el transcurso de este estudio. • Cualquier enfermedad que afecte a la realización del protocolo o a la interpretación de los resultados del estudio o que suponga un riesgo para el paciente si participa en el estudio (p. ej., colitis, enfermedad celíaca, trastorno mendeliano asociado a esofagitis eosinofílica, asma grave no controlada, infección causante de eosinofilia, síndrome hipereosinofílico o trastorno cardiovascular o enfermedad neurológica o psiquiátrica que pueda comprometer la capacidad del paciente para documentar con exactitud los síntomas de esofagitis eosinofílica, neoplasia maligna recién diagnosticada, enfermedad linfoproliferativa o anomalía analítica de importancia clínica). • Infecciones activas o en curso, incluidas las infestaciones parasitarias o helmínticas, la hepatitis viral, la tuberculosis o el VIH. • Haber experimentado anafilaxia idiopática o reacción inmunológica importante a un fármaco que contiene inmunoglobulina G; o cualquier hipersensibilidad conocida a cualquier componente de CC-93538. • Embarazo o lactancia en las mujeres. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability of long-term treatment with CC-93538 evaluated by the incidence, severity, and relationship to CC-93538 of adverse events (AEs), serious adverse events (SAEs), clinical laboratory abnormalities, changes in vital signs, and physical examination abnormalities. |
La seguridad y la tolerabilidad del tratamiento a largo plazo con CC-93538 en función de la incidencia y la intensidad de los acontecimientos adversos (AA), los acontecimientos adversos graves (AAG), las anomalías analíticas, las variaciones de las constantes vitales y las anomalías de la exploración física, y su relación con CC-93538 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline through Safety Follow-up Visit |
Desde el momento basal hasta la visita de seguimiento de la seguridad |
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E.5.2 | Secondary end point(s) |
Immunogenicity of CC-93538 assessed through the incidence of anti-drug antibodies, including neutralizing antibodies when warranted. |
La inmunogenicidad de CC-93538 en función de la incidencia de anticuerpos contra el fármaco, incluidos los anticuerpos neutralizantes cuando proceda |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline through Safety Follow-up Visit |
Desde el momento basal hasta la visita de seguimiento de la seguridad |
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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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 64 |
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 | No |
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 |
Argentina |
Australia |
Canada |
Israel |
Japan |
United States |
Austria |
Belgium |
Germany |
Italy |
Poland |
Portugal |
United Kingdom |
Spain |
Switzerland |
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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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The End of Study is defined as either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary, and/or exploratory analysis, as pre-specified in the protocol, whichever is the later date. |
El final del estudio se define como la fecha de la última visita del último sujeto que finalice el seguimiento posterior al tratamiento, o la fecha de recepción de los últimos datos correspondientes al último sujeto que sean necesarios para los análisis principales, secundarios o exploratorios, tal como se haya especificado en el protocolo, lo que ocurra más tarde. |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |