E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Dermatomyositis |
Dermatomiositis |
|
E.1.1.1 | Medical condition in easily understood language |
Dermatomyositis is a rare inflammatory disease marked by muscle weakness and a distinctive severe skin rash |
Dermatomiositis es una enfermedad inflamatoria rara marcada por debilidad muscular y un sarpullido cutáneo característico y grave. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
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 | 10012503 |
E.1.2 | Term | Dermatomyositis |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
|
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 PF-06823859. |
Evaluar la seguridad y tolerabilidad a largo plazo de PF-06823859. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the long-term efficacy of PF-06823859 in adult participants with moderate to severe DM. |
Evaluar la eficacia a largo plazo de PF-06823859 en participantes adultos con DM de moderada a grave. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participants aged ≥18 and ≤80 with moderate to severe DM, that have completed the treatment period of the qualifying study. 2. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures. 3. Refer to Appendix 4 for reproductive criteria for male (Section 10.4.1) and female (Section 10.4.2) participants. 4. Capable of giving signed informed consent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol. |
Edad y sexo: 1. Participantes de ≥18 años y ≤80 años con DM de moderada a grave que hayan completado el periodo de tratamiento del estudio de cualificación. Tipo de paciente y características de la enfermedad: 2. Los participantes deben estar dispuestos y ser capaces de cumplir con todas las visitas programadas, el plan de tratamiento, las pruebas analíticas, las consideraciones sobre el estilo de vida y otros procedimientos del estudio. 3. Consulte el Apéndice 4 del protocolo C0251008 para ver los criterios sobre reproducción de los participantes masculinos y femeninos. Consentimiento informado: 4. Capaces de otorgar consentimiento informado firmado, lo que comprende el cumplimiento de los requisitos y las restricciones indicados en el FCI y en este protocolo. |
|
E.4 | Principal exclusion criteria |
Medical Conditions: 1. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study. Prior/Concomitant Therapy: 2. Current use of any prohibited concomitant medication(s) listed in Appendix 8. 3. Hematologic abnormalities defined as: ANC ≤1000/mm3; Platelets ≤25,000/mm3; Hemoglobin ≤8g/dL. 4. Hepatic dysfunction defined as: Total bilirubin ≥2x ULN (≥3x ULN for Gilbert’s disease); AST ≥2.5 x ULN; ALT ≥2.5 x ULN; For Participants with DM who experience elevated AST, ALT, LDH, aldolase, and CK, due to muscle involvement the investigator should determine whether or not these are related to the existing condition of DM or if these lab abnormalities are related to another condition. Information should be provided in the source documentation with rationale related to any lab abnormalities. 5. Baseline standard 12-lead ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results (eg, QTcF interval >450 msec, complete LBBB, signs of an acute or indeterminate age myocardial infarction, ST-T interval changes suggestive of myocardial ischemia, second or third degree AV block, or serious bradyarrhythmias or tachyarrhythmias). If the baseline uncorrected QT interval is >450 msec, this interval should be rate corrected using the Fridericia method and the resulting QTcF should be used for decision making and reporting. If QTcF exceeds 450 msec, or QRS exceeds 120 msec, the ECG should be repeated 2 more times and the average of the 3 QTcF or QRS values should be used to determine the participant’s eligibility. Computer interpreted ECGs should be overread by a physician experienced in reading ECGs before excluding a participant. 6. Participants who met discontinuation criteria at any point during the participating qualifying studies. 7. Participants with an ongoing safety event in the qualifying studies which, in the opinion of the investigator or sponsor, is an ongoing safety concern OR the participant has met safety monitoring criteria in the qualifying study that has not resolved. 8. Participants with significant protocol deviations (eg, not following the protocol, not using appropriate contraception) or have had a serious adverse event related to study drug in the previous qualifying studies. Other Exclusions: 9. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members. |
Enfermedades: 1. Otras enfermedades o alteraciones psiquiátricas, incluidos los comportamientos o ideas suicidas recientes (en el último año), o anomalías de laboratorio que puedan aumentar el riesgo de la participación en el estudio o, a juicio del investigador, hagan que el paciente no sea apto para el estudio. Tratamiento previo/concomitante: 2. Uso actual de algún(os) medicamento(s) concomitante(s) prohibido(s) enumerado(s) en el Apéndice 8 del protocolo C0251008. 3. Anomalías hematológicas definidas como: - RAN ≤1000/mm3; - plaquetas ≤25 000/mm3; - hemoglobina ≤8 g/dl. 4. Disfunción hepática definida como: - bilirrubina total ≥2 x LSN (≥3 x LSN para la enfermedad de Gilbert); - AST ≥2,5 x LSN; - ALT ≥2,5 x LSN. - En el caso de los participantes con DM que experimenten niveles elevados de AST, ALT, LDH, aldolasa y CK, debido a la afectación muscular, el investigador debe determinar si estos están o no relacionados con la afección existente de DM o si estas anomalías analíticas están relacionadas con otra afección. La información debe proporcionarse en la documentación fuente con motivos relacionados con cualquier anomalía analítica. 5. ECG estándar al inicio de 12 derivaciones que muestre anomalías clínicamente relevantes que puedan afectar a la seguridad del participante o a la interpretación de los resultados del estudio (p. ej., intervalo QTcF >450 ms, BRI completo, signos de un infarto de miocardio agudo o de edad indeterminada, cambios en el intervalo ST-T indicativos de isquemia miocárdica, bloqueo AV de segundo o tercer grado, o bradiarritmias o taquiarritmias graves). Si el intervalo QT no corregido al inicio es >450 ms, este intervalo debe corregirse con el método de Fridericia y el QTcF resultante debe usarse para la toma de decisiones y la notificación. Si el QTcF supera los 450 ms o el QRS supera los 120 ms, el ECG debe repetirse 2 veces más y la media de los 3 valores de QTcF o QRS se debe utilizar para determinar si el participante es apto. Los ECG interpretados por ordenador deben ser supervisados por un médico con experiencia en la lectura de ECG antes de excluir a los participantes. 6. Participantes que cumplieron los criterios de interrupción en cualquier momento durante los estudios de cualificación en los que participaban. 7. Los participantes con un acontecimiento de seguridad en curso en los estudios de cualificación que, en opinión del investigador o del promotor, sea una preocupación de seguridad en curso O el participante ha cumplido los criterios de supervisión de la seguridad en el estudio de cualificación que no se ha resuelto. 8. Participantes con desviaciones significativas del protocolo (p. ej., que no siguen el protocolo, no utilizan un método anticonceptivo adecuado) o que han sufrido un acontecimiento adverso grave relacionado con el fármaco del estudio en los estudios de cualificación anteriores. Otras exclusiones: 9. Empleados del centro de investigación o de Pfizer directamente implicados en la realización del estudio, empleados del centro supervisados de alguna otra manera por el investigador, y sus respectivos familiares. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of AEs, laboratory abnormalities, changes in vital signs, and ECG findings. |
Incidencia de AA, anomalías de laboratorio, cambios en las constantes vitales y hallazgos del ECG. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the trial. |
A lo largo del estudio |
|
E.5.2 | Secondary end point(s) |
- Change from baseline in CDASI activity score at Week 52 for participants who entered from Stage 2 of Protocol C0251002. - Absolute values and changes from baseline of CDASI activity and CDASI damage scores at all scheduled timepoints. - TIS score at Week 52 and at intermediate scheduled timepoints for participants who entered from Stage 3 of Protocol C0251002. - Change from baseline in the CSMs of the TIS including PhGA, PtGA, MMT-8, HAQ-DI, muscle enzymes, and MDAAT at Week 52 and at intermediate scheduled timepoints. |
- Cambio respecto al inicio en la puntuación de actividad del CDASI en la semana 52. - Valores absolutos y cambios desde el inicio en las puntuaciones de actividad y daño del CDASI en todos los momentos o puntos temporales programados. - Puntuación TIS en la semana 52 y en puntos temporales programados intermedios para los participantes que entraron desde la etapa 3 del protocolo C0251002. - Cambio con respecto al inicio en los CSM de la TIS, incluidos EGM, EGP, MMT-8, HAQ-DI, enzimas musculares y MDAAT en la semana 52 y en puntos temporales programados intermedios. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Week 52 and all other scheduled timepoints. |
Semana 52 y los otros tiempos calendarizados |
|
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 | 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) | 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 | 18 |
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 |
Australia |
Germany |
Hungary |
Italy |
Poland |
Spain |
United States |
|
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
|
LSLV |
último paciente última visita |
|
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 | 3 |
E.8.9.1 | In the Member State concerned days | 19 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 12 |