An open-label, multicenter, randomized phase 3 study to evaluate the efficacy and safety of Romosozumab compared to Bisphosphonates in children and adolescents with osteogenesis imperfecta
Résumé de l'étude
This is a phase 3 study, the final phase of the drug development process for humans. Researchers will compare the study drug to a standard treatment for osteogenesis imperfecta in children (ages 5 to under 12 years) and adolescents (ages 12 to under 18 years). Neither the participants nor the study doctor can choose the study drug that participants will receive. Participants agree to be randomly assigned to a treatment group. This is like flipping a coin or drawing numbers from a hat. The participants, the study doctor, and the study staff know which study drug each participant receives. The study may last up to 15 months for each participant, including a screening phase of up to 35 days, a 12-month treatment phase, and a 3-month safety follow-up phase.
(BASEC)
Intervention étudiée
Participants assigned to the Romosozumab group will receive 5 mg/kg of Romosozumab as a subcutaneous injection every month for 12 months, with a maximum monthly dose of 210 mg. Participants assigned to the comparator group will receive bisphosphonates according to local standard of care (SOC).
All participants must receive vitamin D and calcium supplements throughout the study duration to minimize the risk of hypocalcemia (low calcium levels in the blood). The recommended daily dose is at least 30 to 50 mg/kg per day up to a maximum of 1000 mg of calcium, including dietary intake, and at least 800 IU of vitamin D.
(BASEC)
Maladie en cours d'investigation
Osteogenesis imperfecta (OI) is a genetic disorder that prevents the body from forming strong bones in children. This condition is characterized by bones that break easily. There are no approved medications for the treatment of OI in children, except for Neridronate and Pamidronate, which are approved in Italy and Japan, respectively. Currently, physicians use bisphosphonates in children with moderate to severe OI to increase bone mass. However, the risk of bone fractures cannot be clearly reduced, even for long bone fractures. There is an unmet need for other treatments with further solid scientific evidence. Romosozumab increases bone density (stronger bones) and has the potential to reduce the risk of bone fractures.
(BASEC)
-The patient has provided informed consent prior to the initiation of any study-specific activities/procedures OR -The legally authorized representative of the patient has provided informed consent if the participant is legally too young to provide informed consent, and the patient has provided written consent prior to the initiation of all study-specific activities/procedures in accordance with local regulations and/or guidelines. -Ambulatory male and female children and adolescents aged 5 to <18 years, including those ambulatory with support, as defined in the pediatric OI population. (BASEC)
Critères d'exclusion
-History of an electrophoresis pattern not consistent with OI type I, III, or IV -Known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or another metabolic bone disease -History of congenital dislocation of the radial head, interosseous membrane Calcification or exuberant callus formation (BASEC)
Lieu de l’étude
Bâle, Lausanne
(BASEC)
Sponsor
Amgen Switzerland AG Amgen Inc. USA
(BASEC)
Contact pour plus d'informations sur l'étude
Personne de contact en Suisse
Dr. med. Aline Bregou
+41 21 314 92 41
aline.bregou@clutterchuv.chCentre Hospitalier Universitaire Vaudois
(BASEC)
Informations scientifiques
non disponible
Nom du comité d'éthique approbateur (pour les études multicentriques, uniquement le comité principal)
Commission cantonale d'éthique du Vaud
(BASEC)
Date d'approbation du comité d'éthique
06.02.2024
(BASEC)
Identifiant de l'essai ICTRP
non disponible
Titre officiel (approuvé par le comité d'éthique)
A Phase 3, Open-label, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Romosozumab Compared with Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta (BASEC)
Titre académique
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Titre public
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Maladie en cours d'investigation
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Intervention étudiée
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Type d'essai
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Plan de l'étude
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Critères d'inclusion/exclusion
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Critères d'évaluation principaux et secondaires
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Date d'enregistrement
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Inclusion du premier participant
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Sponsors secondaires
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Contacts supplémentaires
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ID secondaires
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Résultats-Données individuelles des participants
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Informations complémentaires sur l'essai
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Résultats de l'essai
Résumé des résultats
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Lien vers les résultats dans le registre primaire
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