Real-world Study of Nerandomilast in Patients With Idiopathic Pulmonary Fibrosis and Progressive Pulmonary Fibrosis (ReaSoN-IPF/PPF)
A Prospective, Multicenter, Observational Real-world Study of Nerandomilast Efficacy and Safety in Patients With Idiopathic Pulmonary Fibrosis or Progressive Pulmonary Fibrosis
Nerandomilast is an oral medicine that slows down the loss of functional residual capacity (FVC) in people with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). It was tested in two large clinical trials in which participants were carefully selected. In everyday practice, patients are often older, have more other illnesses, or take additional medicines, and it is not known whether the benefits seen in the trials also occur outside them.
The Real-world Study of Nerandomilast (ReaSoN-IPF/PPF) follows people with IPF or PPF who begin nerandomilast as part of their usual medical care at several medical centers in Israel. The study does not assign any treatment; the treating physician makes every decision about therapy.
The main goal is to find out whether lung function declines more slowly once nerandomilast is started. Because patients have breathing tests before they begin the medicine as well as during treatment, each patient is compared with themselves: the rate at which their FVC was declining beforehand is compared with the rate following treatment.
The study also asks how patients feel, using a questionnaire called the K-BILD, and records side effects, hospital admissions, disease exacerbations, and whether patients stop the medicine.
Participants attend their normal clinic visits. They complete the questionnaire before treatment starts and again at 6 to 12 months, and their routine lung function results are collected. No extra tests or visits are required beyond the questionnaire.
Aperçu de l'étude
Statut
Intervention / Traitement
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Doron Cohn-Schwartz, MD, PhD
- Numéro de téléphone: 972-3-6973591
- E-mail: doroncs@tlvmc.gov.il
Sauvegarde des contacts de l'étude
- Nom: Avraham Unterman, MD, MBA
- Numéro de téléphone: 972-3-6973591
- E-mail: ramiu@tlvmc.gov.il
Lieux d'étude
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-
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Ashkelon, Israël, 7830604
- Recrutement
- Barzilai Medical Center, Ben-Gurion University of the Negev
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Contact:
- Ori Wand, MD
- Numéro de téléphone: 972-8-6745465
- E-mail: oriw@bmc.gov.il
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Haifa, Israël, 3109601
- Recrutement
- Rambam Health Care Campus, Technion - Israel Institute of Technology
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Contact:
- Yaniv Dotan, MD, PhD
- Numéro de téléphone: 972-4-8542648
- E-mail: Y_dotan@rmc.gov.il
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Ramat Gan, Israël, 5265601
- Recrutement
- Sheba Medical Center, Tel-HaShomer, Tel-Aviv University
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Contact:
- Liran Levy, MD
- Numéro de téléphone: 972-3-5302745
- E-mail: Liran.Levy@sheba.health.gov.il
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Tel Aviv, Israël, 6423906
- Recrutement
- Tel-Aviv Sourasky University Medical Center, Tel-Aviv University
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Contact:
- Doron Cohn-Schwartz, MD, PhD
- Numéro de téléphone: 972-3-6973591
- E-mail: doroncs@tlvmc.gov.il
-
Contact:
- Avraham Unterman, MD, MBA
- Numéro de téléphone: 972-3-6973591
- E-mail: ramiu@tlvmc.gov.il
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-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
Méthode d'échantillonnage
Population étudiée
Consecutive adults with IPF or PPF followed at one of the participating medical centers who initiate nerandomilast as part of routine clinical care during the enrollment period. Since this is a registry-based study, patients need to be included in a prospective ILD registry in order to participate.
La description
Inclusion Criteria:
- Age 18 years or older.
- Physician-confirmed diagnosis of IPF according to current ATS/ERS/JRS/ALAT guidelines, or of PPF (fibrotic interstitial lung disease other than IPF meeting INBUILD criteria for progression).
- Initiating treatment with nerandomilast as part of routine clinical care, with the treatment decision made independently of, and prior to, study enrollment.
- Willing and able to provides written informed consent to participate in the prospective ILD registry of the respective medical center.
Exclusion Criteria:
- Prior exposure to nerandomilast, including in a clinical trial or open-label extension study.
- Concurrent participation in an interventional trial of an investigational agent for interstitial lung disease.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Modèles d'observation: COHORT
- Perspectives temporelles: PROSPECTIVE
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
IPF or PPF
Patients who are diagnosed with IPF |
Either Nerandomilast 9mg twice a day or 18mg twice a day |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Adverse events
Délai: Through study completion, an average of 9 months
|
Proportion of participants experiencing prespecified adverse events (diarrhea, weight loss, nausea, cough, upper respiratory tract infection, dyspnea, nasopharyngitis, worsening of underlying condition, depression, anxiety), reported overall and by severity. |
Through study completion, an average of 9 months
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Difference between the pre-treatment and on-treatment annualized rate of FVC decline (mL/year)
Délai: Up to 24 months before initiation and, on average, 6 months after initiation
|
Average for all participants. For each participant, the annualized slope of absolute forced vital capacity over the period preceding nerandomilast initiation (derived from at least two pre-treatment measurements obtained within the previous 24 months) is compared with the annualized slope during 12 months of treatment. Each participant serves as their own control; the outcome is the within-participant difference between the two slopes, in mL/year, with a positive value indicating attenuation of decline on treatment. |
Up to 24 months before initiation and, on average, 6 months after initiation
|
|
Difference between pre-treatment and on-treatment annualized rate of FVC decline (% predicted/year)
Délai: Up to 24 months before initiation and, on average, 6 months after initiation
|
As for outcome 2, with FVC expressed as percentage of predicted value. |
Up to 24 months before initiation and, on average, 6 months after initiation
|
|
Difference between pre-treatment and on-treatment annualized rate of DLCO decline (% predicted/year)
Délai: Up to 24 months before initiation and, on average, 6 months after initiation
|
As above, for diffusing capacity of the lungs for carbon monoxide, corrected for hemoglobin. |
Up to 24 months before initiation and, on average, 6 months after initiation
|
|
Change from baseline in K-BILD domain scores
Délai: Baseline and 6-12 months
|
Breathlessness and activities, psychological, and chest symptoms domains, each scored 0-100, higher indicating better health status. |
Baseline and 6-12 months
|
|
Change from baseline in FVC (mL and % predicted)
Délai: Baseline and >6 months
|
Absolute annualized change in forced vital capacity from the pre-treatment baseline measurement. |
Baseline and >6 months
|
|
Change from baseline in DLCO (% predicted)
Délai: Baseline and >6 months
|
Absolute annualized change in hemoglobin-corrected diffusing capacity from the pre-treatment baseline measurement. |
Baseline and >6 months
|
|
Incidence of serious adverse events
Délai: Through study completion, an average of 9 months
|
Proportion of participants with at least one serious adverse event, defined as an event that is fatal, life-threatening, requires or prolongs hospitalization, or results in persistent or clinically significant disability. |
Through study completion, an average of 9 months
|
|
Permanent discontinuation of nerandomilast
Délai: Through study completion, an average of 9 months
|
Proportion of participants permanently discontinuing nerandomilast, time to discontinuation, and reason for discontinuation. |
Through study completion, an average of 9 months
|
|
Dose reduction of nerandomilast
Délai: Through study completion, an average of 9 months
|
Proportion of participants in whom nerandomilast dose is reduced, time to dose reduction, and reason for dose reduction. |
Through study completion, an average of 9 months
|
|
Annualized incidence of acute exacerbation of interstitial lung disease
Délai: Through study completion, an average of 9 months
|
Number and proportion of participants with at least one acute exacerbation, defined according to published criteria, time to exacerbation. |
Through study completion, an average of 9 months
|
|
Annualized incidence of respiratory-cause hospitalization
Délai: Through study completion, an average of 9 months
|
Annualized incidence of respiratory-cause hospitalization |
Through study completion, an average of 9 months
|
|
Annualized all-cause mortality
Délai: Through study completion, an average of 9 months
|
Proportion of participants who die from any cause during follow-up. |
Through study completion, an average of 9 months
|
|
Annualized incidence of lung transplantation
Délai: Through study completion, an average of 9 months
|
Proportion of participants undergoing lung transplantation during follow-up. |
Through study completion, an average of 9 months
|
|
Exploratory: Outcomes stratified by diagnosis, nerandomilast dosage, and background antifibrotic therapy
Délai: Through study completion, an average of 9 months
|
Primary and key secondary outcomes reported separately for IPF versus PPF, and for background nintedanib, pirfenidone, or no background antifibrotic therapy. |
Through study completion, an average of 9 months
|
Collaborateurs et enquêteurs
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Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 0101-21-TLV-A
Plan pour les données individuelles des participants (IPD)
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Informations sur les médicaments et les dispositifs, documents d'étude
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