E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pulmonary sarcoidosis |
Sarcoïdose pulmonaire |
|
E.1.1.1 | Medical condition in easily understood language |
Pulmonary sarcoidosis |
Sarcoïdose pulmonaire |
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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 |
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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 |
To assess the efficacy of efzofitimod in patients with pulmonary sarcoidosis |
Evaluer l’efficacité de l’efzofitimod chez des patients atteints de sarcoïdose pulmonaire |
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E.2.2 | Secondary objectives of the trial |
To assess the safety and tolerability of efzofitimod in patients with pulmonary sarcoidosis |
Evaluer la sécurité et la tolérance de l’efzofitimod chez des patients atteints de sarcoïdose pulmonaire |
|
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
|
- Diagnostic confirmé de sarcoïdose pulmonaire depuis au moins 6 mois, définie par les critères suivants : diagnostic documenté de sarcoïdose histologiquement prouvée par biopsie tissulaire et preuve d’atteinte pulmonaire parenchymateuse documentée par les examens radiologiques historiques
- Mise en évidence d’une sarcoïdose pulmonaire symptomatique, démontrée par les critères suivants : échelle de dyspnée modifiée du MRC (Medical Research Council) de grade d’au moins 1 et score KSQ-Poumon ≤ 70
- Les patients doivent recevoir un traitement par corticoïdes oraux (CO) ≥ 3 mois avec une dose de départ entre ≥ 7,5 et ≤ 25 mg/jour
- Poids corporel ≥ 40 kg et < 160 kg
|
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E.4 | Principal exclusion criteria |
- Treatment with > 1 oral immunosuppressant therapy
- Treatment with biological immunomodulators, such as tumor necrosis factor-alpha (TNF-α) inhibitors or antifibrotics or interleukin inhibitors
- Likelihood of significant pulmonary fibrosis as shown by any 1 or more of the following: High resolution CT fibrosis > 20% at Screening; FVC % predicted < 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
|
- Traitement par > 1 agent immunosuppresseur oral
- Traitement par immunomodulateurs biologiques, tels que inhibiteurs du facteur de nécrose tumorale-alpha (TNF-α), antifibrotiques ou inhibiteurs d’interleukines
- Probabilité de fibrose pulmonaire significative démontrée par au moins 1 des critères suivants : TDM à haute résolution montrant une fibrose > 20 % à la sélection, valeur prédite CVF % < 50 % et score KSQ-Poumon < 30
- Hypertension pulmonaire cliniquement significative nécessitant un traitement par vasodilatateurs
- Sarcoïdose cardiaque, neurosarcoïdose ou sarcoïdose rénale
- Sarcoïdose cutanée et oculaire cliniquement significative
- Antécédents de symptômes addisoniens ayant empêché les tentatives précédentes de diminution progressive de CO
- Gros fumeur actif de tabac/produits contenant de la nicotine
- Antécédents de syndrome anti-synthétase (anti-Jo-1) ou positivité Jo-1 à l’inclusion
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in mean daily OCS dose post-taper |
Modification, par rapport aux valeurs initiales, de la dose quotidienne moyenne de CO après diminution progressive |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
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
|
• Taux annuel de modification de la valeur absolue de CVF • Modification en pourcentage, par rapport aux valeurs initiales, de la dose quotidienne moyenne de CO après diminution progressive • Modifications, par rapport aux valeurs initiales, du score KSQ-Poumon à la Semaine 48 |
|
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 | 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 | 5 |
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 | 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 |
Japan |
Puerto Rico |
United States |
France |
Netherlands |
Spain |
Germany |
Italy |
United Kingdom |
|
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
|
LVLS |
Dernière visite du dernier patient |
|
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 |