E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic pulmonary fibrosis |
Fibrosis Pulmonar Idiopática |
|
E.1.1.1 | Medical condition in easily understood language |
Lung scarring of unknown origin |
Cicatrización pulmonar de origen desconocido |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10021240 |
E.1.2 | Term | Idiopathic pulmonary fibrosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
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 |
To evaluate, in comparison with placebo, the efficacy of 2 dose levels of SAR156597 administered subcutaneously during 52 weeks on lung function of patients with IPF. |
Evaluar, en comparación con un placebo, la eficacia de 2 niveles de dosis/regímenes de SAR156597 administrados por vía subcutánea durante 52 semanas sobre la función pulmonar en pacientes con FPI |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the efficacy of 2 dose levels of SAR156597 compared to placebo on IPF disease progression. To evaluate the safety of 2 dose levels of SAR156597 compared to placebo in patients with IPF. |
Evaluar la eficacia de 2 niveles de dosis/regímenes de SAR156597 en comparación con placebo sobre la progresión de la enfermedad de FPI Evaluar la seguridad de 2 niveles de dosis/regímenes de SAR156597 en comparación con placebo en pacientes con FPI |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Adult male or female patients. Documented diagnosis of IPF according to the current American Thoracic Society/European Respiratory Society/Japanese Respiratory Society/ American Latin Thoracic Association (ATS/ERS/JRS/ALAT) guidelines. Signed written informed consent. |
Pacientes de sexo masculino o femenino que otorguen su consentimiento Diagnóstico documentado de FPI según las directrices actuales de la ATS, es decir: ? Exclusión de otras causas conocidas de enfermedad pulmonar intersticial (p. ej., exposiciones ambientales domésticas y ocupacionales, conectivopatías y toxicidades farmacológicas); Y ? Presencia de un patrón habitual de neumonía intersticial en la tomografía axial computarizada de alta resolución (TACAR) en pacientes no sometidos a biopsia pulmonar quirúrgica; O ? Combinaciones específicas de patrones en TACAR y biopsia quirúrgica en pacientes sometidos a biopsia pulmonar quirúrgica. HIP/CI firmado |
|
E.4 | Principal exclusion criteria |
Age ?40 years. IPF disease diagnosis >5 years. Forced vital capacity (FVC) <40% of predicted value. Carbon monoxide diffusing lung capacity (DLco) corrected for hemoglobin <30% of predicted value. Severe chronic obstructive bronchitis as characterized by forced expiratory volume in 1 second /forced vital capacity (FEV1/FVC) <0.70. Need for 24 hrs of oxygen therapy or oxygen saturation <88% after 10 minutes breathing ambient air at rest. Known diagnosis of significant respiratory disorders other than IPF. Pulmonary artery hypertension requiring a specific treatment. Currently listed and/or anticipated to be listed for lung transplantation within the next 6 months (on an active list). History of vasculitis or connective tissue disorders. Known human immunodeficiency virus (HIV) or chronic viral hepatitis. Patients with active tuberculosis or incompletely treated latent tuberculosis infection. Use of any cytotoxic/immunosuppressive agent including but not limited to azathioprine, cyclophosphamide, methotrexate, and cyclosporine within 4 weeks prior to screening. Use of any cytokine modulators (etanercept, adalimumab, efalizumab, infliximab, golimumab, certolizumab, rituximab) within 12 weeks or 5 half-lives of screening (24 weeks for rituximab and 24 months for alefacept). |
? Edad ? 40 años ? Diagnóstico de la FPI > 5 años ? Capacidad vital forzada (CVF< 40 % del valor previsto ? Capacidad de difusión pulmonar del monóxido de carbono (DLCO) corregida para la hemoglobina < 30 % del valor previsto ? Bronquitis crónica obstructiva severa caracterizada por una ratio FEV1 CVF < 0,70 ? Necesidad de oxigenoterapia durante 24 horas o saturación de oxígeno < 88 % después de 10 minutos respirando aire ambiental en reposo ? Diagnóstico conocido de trastornos respiratorios significativos (p. ej., asma, tuberculosis, sarcoidosis, aspergilosis o fibrosis quística) distintos de la FPI ? Hipertensión arterial pulmonar que requiera tratamiento específico ? Persona en lista de espera y/o que se prevea que vaya a entrar en lista de espera para trasplante de pulmón en los 6 meses siguientes (lista activa) ? Antecedentes de vasculitis, de trastornos del tejido conjuntivo o anticuerpos frente al citoplasma de los neutrófilos (ANCA positivos Antecedentes de infarto de miocardio en los 6 meses anteriores a la selección |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Mean absolute change from baseline in percent predicted FVC at 52 weeks |
Cambio absoluto respecto al momento basal en el % de CVF previsto a las 52 semanas |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Proportion of patients with disease progression Number of deaths (all causes) |
Progresión de la enfermedad definida por el número de los eventos siguientes: descenso del % absoluto de CVF previsto ? 10 %, descenso del % absoluto de DLCO previsto ? 15 %, trasplante de pulmón o muerte a las 52 semanas |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | No |
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 | 3 |
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 | 33 |
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 |
Canada |
Chile |
Colombia |
Czech Republic |
Denmark |
France |
Germany |
Greece |
Israel |
Italy |
Korea, Republic of |
Mexico |
Spain |
Turkey |
United Kingdom |
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
|
LPLV |
Última visita del último paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 28 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 28 |