E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Wound Healing in Uraemic Calciphylaxis |
Curación de heridas en pacientes de clacifilaxis urémica |
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E.1.1.1 | Medical condition in easily understood language |
deposition of calcium into the blood vessels |
acumulación de calcio en los vasos sanguineos |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10051714 |
E.1.2 | Term | Calciphylaxis |
E.1.2 | System Organ Class | 10027433 - Metabolism and nutrition 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 effect of SNF472 on top of standard of care on promoting wound healing and other parameters of therapeutic response in haemodialysis patients with calciphylaxis |
Para evaluar el efecto de SNF472, encima del tratamiento estándar de cuidado, en la promoción de la cicatrización de heridas y otros parámetros de la respuesta terapéutica en pacientes en hemodiálisis con calcifilaxis |
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E.2.2 | Secondary objectives of the trial |
not applicable |
no procede |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with newly diagnosed CUA (diagnosed within 3 weeks of study start) 2. Patients who signed the written informed consent to participate in this clinical trial (prior to any clinical trial related procedures being performed), after reading the Patient Information Sheet and Informed Consent Form (ICF), and who had the opportunity to discuss the clinical trial with the Investigator or designee 3. Males or females aged ?18. 4. Females of child-bearing potential should have a negative pregnancy test at baseline and use double-barrier contraception . Male subjects having sexual relationship in which pregnancy can occur should take adequate contraceptive precautions 5. Patients on maintenance haemodialysis 6. Patients with a minimum level of pain on VAS scale of 30 mm |
1. Los pacientes con diagnóstico reciente de CUA (diagnosticado 3 semanas antes de iniciar en el estudio) 2. Capaz de dar su consentimiento informado por escrito 3. Edad ? 18 años 4. Mujeres con potencial reproductor deben dar negativo en una prueba de embarazo basal y usar métodos anticonceptivos de doble barrera. Los hombres que mantengan relaciones sexuales con mujeres que puedan quedar embarazadas deben tomar precauciones anticonceptivas adecuadas 5.Pacientes de hemodiálisis crónica 6. Nivel mínimo del dolor en la escala VAS de 30 |
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E.4 | Principal exclusion criteria |
1. Current or history of bisphosphonate treatment within 12 months of entering into study 2. Patients being anticoagulated with citrate 3. Severely ill patients without reasonable expectation of survival for > 6 months according to the treating physician 4. Patients with scheduled parathyroidectomy during the run-in or study period 5. Female patients who are either intending to get pregnant or are undergoing treatment to get pregnant 6. Participation in another clinical trial with an experimental drug within 90 days before the first administration of the IMP 7. Any psychological, emotional problems, any disorders or resultant therapy that is likely to invalidate informed consent, or limit the ability of the subject to comply with the Clinical trial Protocol requirements 8. Subjects who, in the opinion of the Investigator, are considered unsuitable for any other reason |
1. Actual o antecedentes de tratamiento con bifosfonatos durante los 12 meses anteriores al estudio 2. Pacientes anticouagulados con citrato 3. Los pacientes gravemente enfermos sin expectativa razonable de supervivencia durante> 6 meses, de acuerdo con el médico tratante 4. Los pacientes con paratiroidectomía programada durante el período de ejecución del estudio 5. Mujeres que pretendan quedarse embarazadas o estén en tratamiento de fertilidad para quedarse embarazadas 6. Participación en otro ensayo clínico un un medicamento experimental en los últimos 90 días antes de la administración del medicamento en estudio 7. Cualquier problema emocional y psicológico, cualquier desorde o tratamiento que pueda invalidar el consentimiento informatdo o que limite la capacidad del sujeto a cumplir con lo que se requiere de él según el protocolo del ensayo 8. Pacientes que, en opinión del investigador, no se consideren aptos por cualquier motivo |
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E.5 End points |
E.5.1 | Primary end point(s) |
? The primary lesion is the largest one ? based on longitudinal and transverse measurements - and the one to be followed through imaging throughout the duration of the study by taking pictures of the lesion ? A maximum of 3 lesions ? including the primary one ? will be thoroughly assessed during the study ? Lesion score (Bates Jensen Assessment tool will be performed during Weeks 1, 2, then 4, 6, 8, 10, 12 and EOS (FU) visit |
? La lesión/úlcera principal es la más extensa y la que ha de seguirse a través de imágenes en toda la duración del estudio ? Un máximo de 3 lesiones - incluyendo la lesión principal - se evaluará a fondo durante el estudio ? La puntuación de la lesión realizado en la semana 1, 2, luego 4, 6, 8, 10, 12 y la visita final del estudio (FU) (según herramienta Bates Jensen Assessment) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weeks 1, 2, then 4, 6, 8, 10, 12 and EOS (FU) visit |
Semanas 1, 2 y posteriorment 4, 6, 8, 10, 12 y visistas de fin de estudio / seguimiento |
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E.5.2 | Secondary end point(s) |
? Wound infection, both for the primary and the secondary lesions ? Systemic infection ? Surgical treatment of the wound ? Hospitalisation defined as being admitted for >24 hours ? Course of the secondary lesions defined clinically as for the primary ? Change in pain from week 0 to 12 according to 100 mm Visual Analog Pain Scale (VAS) - Pain scale done at Screening, weeks 1, 2, 4, 6, 8, 10, 12 and EOS (FU) visit ? Change in dose and type of pain medication assessed as a continuous variable. Name, dose and number of pills captured. ? Wound QOL score done at Screening and weeks 1, 6 and 12 ? Death |
? Presencia o ausencia de infección de la herida, tanto para las lesiones principales y las secundarias ? Infección sistémica ? Tratamiento quirúrgico de las lesiones ? Hospitalización, definidas como ingreso de más de 24 horas ? Curso de las lesiones secundarias ? Cambio en el dolor de la semana 0 a 12 según la escala visual analógica (VAS) de dolor de 100mm -Escala de dolor realizada en las semanas 1, 2, 4, 6, 8, 10, 12 y visita final de estudio (FU) ? Cambio en la dosis y el tipo de medicamento para el dolor evaluado como una variable continua. Nombre, dosis y el número de píldoras capturados. ? CVRS hecho en las semanas 1, 6 y 12 ? Muerte |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. Pain Score: at Screening, weeks 1, 2, 4, 6, 8, 10, 12 and EOS (FU) visit 2. Wound QoL Score: at Screening and weeks 1, 6 and 12 |
1. Puntuación del dolor: en visitas de selección, semana 1, 2, 4, 6, 8, 10, 12 y visita final de estudio 2. Cuestionario de calidad de vida: visitas de selección y semanas 1, 6 y 12 |
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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 | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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 | 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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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.8.3 |
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 | 4 |
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 |
Germany |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
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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LVLS |
Ultima visita del último paciente |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |