Traitement de l'insuffisance surrénalienne chez les enfants
Une étude ouverte de phase 3 d'Infacort® chez les nouveau-nés, les nourrissons et les enfants de moins de 6 ans atteints d'insuffisance surrénalienne
The study will be conducted in a total of 24 subjects before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism.
The study will consist of three consecutive cohorts. Cohort 1 will include 12 subjects aged between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2 years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be enrolled (Cohort 3).
The decision to continue after each cohort will be based on the recommendation of an Independent Data Monitoring Committee (IDMC).
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
This is a Phase 3, open label, single centre study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency. The study will be conducted in a total of 24 subjects, before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism. Due to the rare occurrence of adrenal insufficiency it is expected that for the third cohort of this study (neonates) only subjects with CAH will be recruited.
The study will consist of three consecutive parts. Cohort 1 will include 12 subjects aged between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2 years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be enrolled (Cohort 3).
The decision to continue after each cohort will be based on the recommendation of an Independent Data Monitoring Committee (IDMC).
The study will consist of a screening visit (Visit 1 performed as a minimum the day before the intake of study drug), one treatment visit (Visit 2, Day 1), a follow-up visit (Visit 3) one to three days after intake of study drug (Day 2, Day 3 or Day 4) and a follow-up telephone call (Visit 4) 7 - 10 days after intake of study drug. Study completion evaluation will be performed at Visit 3. Parents/ carers will have at least 1 night to consider participation of their child before completing written informed consent. Children aged 3 - 6 years will be informed about their involvement in the study in the presence of their parents/carers.
All subjects will receive their standard treatment including fludrocortisone other than the dose of hydrocortisone that is to be substituted by Infacort®.
Subjects who meet the eligibility criteria at screening (Visit 1) will attend for Visit 2 at a suitable time before the next planned dose of hydrocortisone is due. Subjects may have insertion of an intravenous cannula (with suitable local anaesthesia) allowing blood samples to be taken as well as their routine clinical samples (where required) prior to their next dose of hydrocortisone given as Infacort®. If a cannula is not used, direct venous sampling may be carried out instead. After all planned study procedures have been completed the subjects will go home and will return one to three days later for the follow-up assessments (Visit 3). A follow-up telephone call (Visit 4) 7 - 10 days after intake of study drug will be undertaken.
Type d'étude
Inscription (Réel)
Phase
- Phase 3
Contacts et emplacements
Lieux d'étude
-
-
-
Berlin, Allemagne, 13353
- Charité-Universitätsmedizin Berlin, CVK
-
-
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
- Male and female children less than 6 years of age.
- A diagnosis of adrenal insufficiency as confirmed by an inappropriately low cortisol usually with other supporting tests.
- Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
- Adequately hydrated and nourished.
- Ability of parents/carers to understand and give written Informed Consent
Exclusion Criteria:
- Clinically evident acute adrenal insufficiency (adrenal crisis).
- Inability of the child to take oral therapy.
- Concomitant therapy (other than that required to treat adrenal insufficiency, Vitamin D, Fluoride, Thyroxine and growth hormone).
- Subjects with clinical signs of acute infection or fever on Day 1.
- Any surgical or medical condition which in the opinion of the investigator may place the subject at higher risk from his/her participation in the study.
- Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons.
- Subjects who are dependent on the investigator or the sponsor.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
|---|---|
|
Expérimental: Infacort
Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg). The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose. |
dry granule formulation of hydrocortisone |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Serum Cortisol Concentration up to 240 Minutes
Délai: 240 minutes
|
The primary endpoint will be the maximum levels of serum cortisol concentration up to 240 minutes after intake of study drug as determined by the central laboratory. |
240 minutes
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Serum Cortisol Concentration up to 6 Hours
Délai: 240 minutes
|
Serum cortisol concentration 240 minutes after intake of study drug as determined by the central laboratory |
240 minutes
|
|
Subject Assessment of Taste of the Product
Délai: 1 minute
|
Palatability of the investigational product as determined by parent/carer responses to the following questions: Question 1: My child found swallowing easy. Question 2: My child showed a positive reaction after Infacort was given. Question 3: I would be happy to give my child Infacort in the future. Question 4: Overall, I would prefer Infacort for my child over the usual hydrocortisone medication. |
1 minute
|
|
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE)
Délai: 7-10 days
|
Incidence of serious adverse events (SAEs) and adverse events (AE). |
7-10 days
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Chercheur principal: Susanna Wiegand, MD, Charité-Universitätsmedizin Berlin, CVK
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 (Estimé)
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
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- Infacort-003
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .