E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary sarcoidosis |
Sarcoidosi polmonare |
|
E.1.1.1 | Medical condition in easily understood language |
Pulmonary sarcoidosis |
Sarcoidosi polmonare |
|
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 | 10037430 |
E.1.2 | Term | Pulmonary sarcoidosis |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal 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 assess the efficacy of efzofitimod in patients with pulmonary sarcoidosis |
Valutare l’’efficacia di efzofitimod in pazienti con sarcoidosi polmonare |
|
E.2.2 | Secondary objectives of the trial |
To assess the safety and tolerability of efzofitimod in patients with pulmonary sarcoidosis |
Valutare la sicurezza e tollerabilità di efzofitimod in pazienti con sarcoidosi polmonare |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Confirmed diagnosis of pulmonary sarcoidosis for at least 6 months, defined by the following criteria: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence - Evidence of symptomatic pulmonary sarcoidosis, as demonstrated by the following criteria: Modified Medical Research Council (MRC) dyspnea scale grade of at least 1 and KSQ-Lung score <=70 - Patients must be receiving treatment with OCS of >= 3 months with a starting dose between >= 7.5 and <= 25 mg/day. - Body weight >= 40 kg and < 160 kg |
- Diagnosi confermata di sarcoidosi polmonare da almeno 6 mesi, definita dai seguenti criteri: diagnosi documentata di sarcoidosi istologicamente provata mediante biopsia tissutale ed evidenza documentata di coinvolgimento polmonare parenchimale mediante evidenza radiologica anamnestica - Evidenza di sarcoidosi polmonare sintomatica, come dimostrato dai seguenti criteri: punteggio di almeno 1 nella scala modificata della dispnea secondo il Consiglio per la ricerca medica (MRC) e punteggio KSQ-Lung <=70 - I pazienti devono ricevere un trattamento con OCS di >= 3 mesi con una dose iniziale compresa tra >= 7,5 e <= 25 mg/die. - Peso corporeo >= 40 kg e <160 kg |
|
E.4 | Principal exclusion criteria |
- Treatment with > 1 oral immunosuppressant therapy - Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-a) inhibitors or antifibrotics or interleukin inhibitors - Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: CT fibrosis > 20% within the last 12 months; FVC % predicted (FVCPP)< 50% and KSQ-Lung score < 30 - Clinically significant pulmonary hypertension requiring treatment with vasodilators - Patients with cardiac sarcoidosis, neurosarcoidosis, or renal sarcoidosis - Clinically significant cutaneous and ocular sarcoidosis - History of Addisonian symptoms that precluded previous OCS taper attempts - Is an active, heavy smoker of tobacco/nicotine-containing products - History of anti-synthetase syndrome or Jo-1 positive at baseline |
- Trattamento con > 1 terapia immunosoppressiva orale - Trattamento con immunomodulatori biologici, come inibitori del fattore di necrosi tumorale-alfa (TNF-a) o antifibrotici o inibitori dell'interleuchina - Probabilità di fibrosi polmonare significativa come mostrato da uno o più dei seguenti: fibrosi alla TC > 20% entro gli ultimi 12 mesi; % di FVC prevista (FVCPP) < 50% e punteggio KSQ-Lung < 30 - Ipertensione polmonare clinicamente significativa che richiede un trattamento con vasodilatatori - Pazienti con sarcoidosi cardiaca, neurosarcoidosi o sarcoidosi renale - Sarcoidosi cutanea e oculare clinicamente significativa - Anamnesi di sintomi addisoniani che precludevano precedenti tentativi di riduzione graduale dell'OCS - Essere un attivo e forte fumatore di prodotti contenenti tabacco/nicotina - Anamnesi di sindrome anti-sintetasi o Jo-1 positivo al basale |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in mean daily OCS dose post-taper |
Variazione rispetto al basale nella dose media giornaliera di OCS post-riduzione graduale |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
At week 48 |
Alla settimana 48 |
|
E.5.2 | Secondary end point(s) |
• Annual rate of change in absolute value of FVC • Percent change from baseline in mean daily OCS post-taper • Change from baseline in KSQ-Lung score at Week 48 |
• Tasso annuale di variazione del valore assoluto di FVC • Variazione percentuale rispetto al basale nella dose media giornaliera di OCS post-riduzione graduale • Variazione dal basale nel punteggio KSQ-Lung alla Settimana 48 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
At week 48 |
Alla settimana 48 |
|
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 | 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) | 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 | 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 | 30 |
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 |
Puerto Rico |
France |
Germany |
Italy |
Japan |
Netherlands |
Spain |
United Kingdom |
United States |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |