E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Nefropatía crónica Chronic Kidney Disease |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064848 |
E.1.2 | Term | Chronic kidney disease |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
- Evaluar la eficacia y la seguridad del tratamiento episódico de la anemia ferropénica (AF) con ferumoxitol. - To evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with ferumoxytol. |
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E.2.2 | Secondary objectives of the trial |
Evaluar el patrón de recidivas de la AF. - To assess the pattern of recurrence of IDA. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Sujetos que hayan completado la participación en los estudios FER-CKD-251 o FER-CKD-252 en las 4 últimas semanas 2.Las mujeres en edad fértil que sean sexualmente activas deben utilizar un método anticonceptivo eficaz y aceptar seguir utilizándolo hasta la finalización del estudio 3.El sujeto y/o el tutor legal son capaces de comprender y cumplir los requisitos del protocolo y están disponibles durante todo el estudio 4.El sujeto y/o el tutor legal han sido informados de la naturaleza experimental de este estudio y han otorgado su consentimiento informado por escrito de forma voluntaria y, si procede, el niño/adolescente ha proporcionado el "asentimiento" atendiendo a la Health Insurance Portability and Accountability Act (HIPAA) o la protección de pacientes de conformidad con las normas institucionales, locales y nacionales de protección de datos de salud personales.
1. Subjects who have completed participation in study FER CKD 251 or FER CKD-252 within the past 4 weeks 2. Female subjects of childbearing potential who are sexually active must be on an effective method of birth control and agree to remain on birth control until completion of the study 3. Subject and/or legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study 4. Subject and/or legal guardian has been informed of the investigational nature of this study and has given voluntary written informed consent, and, if appropriate, child/adolescent has provided ?assent? and Health Insurance Portability and Accountability Act (HIPAA) or patient protection authorization in accordance with institutional, local, and national personal health data protection guidelines |
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E.4 | Principal exclusion criteria |
1.Haber experimentado un acontecimiento adverso grave (AAG) relacionado con el tratamiento con hierro intravenoso (IV) en los estudios FER-CKD-251 o FER-CKD-252. 2.Hemoglobina < o igual 7,0 g/dl. 3.Intervención de cirugía mayor o invasiva en las 4 semanas previas a la selección o durante el período de selección. 4.Aparición de una neoplasia maligna durante la participación en los estudios FER-CKD-251 o FER-CKD-252 o antes del reclutamiento en este estudio (salvo cáncer de piel distinto del melanoma o carcinoma in situ que sea extirpable). 5.Haber recibido un fármaco en investigación durante la participación en los estudios FER-CKD-251 o FER-CKD-252 o antes del reclutamiento en este estudio, aparte del especificado en los protocolos de los estudios. 6.Mujeres que estén embarazadas o que tengan intención de quedarse embarazadas o con un resultado positivo en una prueba de embarazo en suero u orina. 7.Cualquier otro trastorno o enfermedad de carácter médico o psiquiátrico clínicamente importante (p. ej., hipertensión no controlada, psicosis) o responsabilidad del sujeto que, en opinión del investigador, pueda afectar a la capacidad del sujeto (y/o su tutor legal) de seguir el protocolo, interferir en la evaluación del producto experimental o ser una contraindicación para la participación del sujeto en el estudio.
1. Experienced a serious adverse event (SAE) related to intravenous (IV) iron therapy in study FER CKD 251 or FER-CKD-252 2. Hemoglobin ?7 g/dL 3. Major surgery or invasive intervention within 4 weeks prior to Screening or during the Screening Period 4. Development of an active malignancy during participation in study FER CKD 251 or FER-CKD-252 or prior to enrollment in this study (except nonmelanoma skin cancer or carcinoma in situ that is excisable) 5. Received an investigational agent during participation in study FER CKD 251 or FER-CKD-252 or prior to enrollment in this study, other than as specified by the study protocols 6. Female subjects who are pregnant or intend to become pregnant or have a positive serum or urine pregnancy test 7. Development of any other clinically significant medical or psychiatric disease or condition (eg, uncontrolled hypertension, psychosis) or subject responsibility that, in the Investigator?s opinion, may interfere with a subject?s (and/or legal guardian?s) ability to adhere to the protocol, interfere with assessment of the investigational product, or serve as a contraindication to the subject?s participation in the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
Criterio de valoración principal: -Variación media de la hemoglobina entre el momento basal y la semana 5 después del primer ciclo de ferumoxitol. Otros criterios de valoración de la eficacia: -Variación media de la hemoglobina entre el momento basal y la semana 7 después del primer ciclo de ferumoxitol. -Proporción de sujetos con un aumento de la hemoglobina > o igual 1,0 g/dl durante el período comprendido entre el momento basal y las semanas 5 y 7 después de cada ciclo de ferumoxitol. -Proporción de sujetos con un aumento de la hemoglobina > o igual 12,0 g/dl durante el período comprendido entre el momento basal y las semanas 5 y 7 después de cada ciclo de ferumoxitol. -Variación media de la SATT desde el momento basal hasta las semanas 5 y 7 después del primer ciclo de ferumoxitol. -Proporción de sujetos que requieran el inicio de FEE o un aumento > 20% de la dosis durante el estudio. -Proporción de sujetos que reciban transfusiones de eritrocitos o de sangre completa durante el estudio. -Variación media de otros marcadores de los depósitos de hierro (p. ej., ferritina sérica y hierro sérico) entre el momento basal y las semanas 5 y 7 después del primer ciclo de ferumoxitol. -Frecuencia del tratamiento con ferumoxitol. Criterios de valoración de la seguridad: -Acontecimientos adversos de interés especial (AAIE) (hipotensión e hipersensibilidad). -AAG. -AA intensos. -AA cardiovasculares (infarto de miocardio no mortal, insuficiencia cardíaca, hipertensión moderada a grave y hospitalización por cualquier causa cardiovascular). -AA que obliguen a suspender la medicación del estudio. -Todos los AA, las constantes vitales (presión arterial, frecuencia cardiaca, frecuencia respiratoria) y la temperatura corporal, y los parámetros analíticos habituales (hematología, bioquímica, perfil del hierro y análisis de orina). PRIMARY ENDPOINT: ? Mean change in hemoglobin from Baseline to Week 5 following the first course of ferumoxytol ADDITIONAL EFFICACY ENDPOINTS: ? Mean change in hemoglobin from Baseline to Week 7 following the first course of ferumoxytol ? Proportion of subjects with an increase in hemoglobin ?1.0 g/dL during the period from Baseline to Week 5 and Week 7 following each course of ferumoxytol ? Proportion of subjects with an increase in hemoglobin to ?12.0 g/dL during the period from Baseline to Week 5 and Week 7 following each course of ferumoxytol ? Mean change in TSAT from Baseline to Week 5 and Week 7 following the first course of ferumoxytol ? Proportion of subjects requiring initiation of ESA or >20% increase in dose during the study ? Proportion of subjects receiving red blood cell (RBC) or whole blood transfusions during the study ? Mean change in other markers of iron stores (eg, serum ferritin and serum iron) from Baseline to Week 5 and Week 7 following the first course of ferumoxytol ? Frequency of treatment with ferumoxytol
Additional analyses of efficacy endpoints will also be performed based on age cohort, CKD status (stage of CKD, dialysis modality, transplant status), ESA use, and treatment course (first vs subsequent courses of treatment).
SAFETY ENDPOINTS ? Adverse events of special interest (AESI) (hypotension and hypersensitivity) ? SAEs ? Severe AEs ? Cardiovascular AEs (nonfatal myocardial infarction, heart failure, moderate to severe hypertension, and hospitalization due to any cardiovascular cause) ? AEs leading to study drug discontinuation ? All AEs, vital signs (blood pressure, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry, iron panel, and urinalysis) |
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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 | No |
E.6.7 | Pharmacodynamic | No |
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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 |
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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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Última visita del último paciente que participa en el ensayo. 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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |