- ICH GCP
- Registre américain des essais cliniques
- Essai clinique NCT01543191
A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF
Aperçu de l'étude
Type d'étude
Inscription (Réel)
Phase
- La phase 1
Contacts et emplacements
Lieux d'étude
-
-
Northern Ireland
-
Belfast, Northern Ireland, Royaume-Uni, BT9 6AD
- Celerion
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
- Males or non-pregnant, non-lactating healthy female.
- Age 18 to 55 years of age (inclusive) on day of signing informed consent.
- Subject has a BMI of 17 or greater.
- Subject has a confirmed diagnosis of cystic fibrosis documented by a compatible clinical or radiographic presentation, and definitive accepted lab and clinical criteria (e.g., sweat chloride > 60 mEq/L or 2 disease causing CFTR mutations) OR is regularly attending a Consultant led clinic where the diagnosis of Cystic Fibrosis is not in doubt.
- Subject has an FEV1 ≥ 45% of predicted at screening.
- Subject has an oxygen saturation >92% on room air as determined by pulse oximetry at screening.
- Subject is a non-smoker or ex-smoker has not smoked for at least six months prior to screening.
- Subject is otherwise medically stable without clinically significant abnormal screening results that depart from their usual baseline level of health as a subject with CF.
- Must be willing and able to communicate in English and participate in the whole study.
- Must provide written informed consent.
Exclusion Criteria:
- Subject has unstable lung disease, as defined by a change in medical regimen during the preceding 2-weeks, or a significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse), or in the opinion of the Investigator, has had a decline in pulmonary status within the last year not considered a part of the usual, chronic progression of CF lung disease and part of their baseline health condition as a subject with CF.
- Subject has had an exacerbation of respiratory symptoms within the past 30 days that required initiation of a new or altered respiratory therapy.
- Subject had either an upper or lower respiratory illness within the 30 days prior to dosing days, or has symptoms from such an illness that have not resolved.
- Subject has a history of lung transplantation.
- Females of child bearing potential who are pregnant, or lactating. Females who are sexually active and either not surgically sterile or not willing to use an acceptable form of contraception
- Clinically significant abnormal biochemistry, haematology or urinalysis not consistent with CF as judged by the Investigator
- Failure to satisfy the Investigator of fitness to participate for any other reason.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Randomisé
- Modèle interventionnel: Affectation croisée
- Masquage: Tripler
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Comparateur placebo: Placebo
|
low dose and high dose for 3 doses, BID
|
|
Expérimental: PUR118
|
low dose and high dose for 3 doses, BID
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Safety and Tolerability
Délai: 4-6 weeks
|
Changes in clinical signs and symptoms from physical examination and chest examination, changes in clinical safety laboratory tests, vital signs, spirometry, lung clearance index, 24-hour sputum weight and volume, oxygen saturation, ECG, telemetry, and adverse events.
|
4-6 weeks
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Pharmacodynamics
Délai: 36 hours
|
To assess impact of PUR118 on mobilization and expectorated volume of airway mucus secretions. To evaluate impact of PUR118 on measurements of lung function, including ventilation inhomogeneity, by both spirometry and measurement of the lung clearance index (LCI). To evaluate an impact of PUR118 on sputum, serum and plasma biomarkers related to airway inflammation. |
36 hours
|
Collaborateurs et enquêteurs
Parrainer
Collaborateurs
Les enquêteurs
- Chercheur principal: Stephen Smith, MD, Celerion
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude
Achèvement primaire (Réel)
Achèvement de l'étude (Réel)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Estimation)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Estimation)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- 601-0009
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
produit fabriqué et exporté des États-Unis.
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