E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
les patients ayant un ostéome ostéoïde douloureux, La population étudiée concernera les enfants, les adolescents et les jeunes adultes. L'ostéome ostéoïde est rare avant l'âge de cinq ans et après 40 ans. |
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E.1.1.1 | Medical condition in easily understood language |
we investigate children, adolescents and young adults with an osteoid osteoma, a small benign bone tumor. Osteoid osteoma are unusual before the age of five years and after forty years |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10031249 |
E.1.2 | Term | Osteoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 demonstrate that in patients with OO, treatment with three intravenous cycles of 4mg of zoledronic acid (Zoledronic acid) administered monthly, is non-inferior to treatment with percutaneous thermal ablation on the efficacy measured by the percentage of pain relief between baseline and end of treatment. |
Montrer la non-infériorité de trois perfusions d'acide zolédronique en terme d'efficacité par rapport à une intervention de destruction tumorale percutanée pour le traitement d'un ostéome ostéoïde, mesurée par le pourcentage de variation de la douleur |
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E.2.2 | Secondary objectives of the trial |
To demonstrate that in patients with OO, treatment with three intravenous cycles of 4mg of zoledronic acid (Zoledronic acid) administered monthly, is non-inferior to treatment with percutaneous thermal ablation on the efficacy measured by the percentage of pain relief between baseline and end of treatment. |
a) Objectifs secondaires d'efficacité - Evaluer la douleur, l'impression de changement, la consommation d'antalgiques et d'AINS. - Etudier l'évolution de la minéralisation du nidus. - Etudier l'évolution de l'œdème péri lésionnel. - Documenter la cinétique de l'efficacité du traitement de l'acide zolédronique. b) Objectifs secondaires de sécurité - Comparer la tolérance et les effets secondaires des perfusions d'acide zolédronique et de la destruction percutanée.
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E.2.3 | Trial contains a sub-study | No |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
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E.3 | Principal inclusion criteria |
- Age superior or equal to 10 years - Patient with a typical osteoid osteoma diagnosed on clinical and radiological criteria, validated by a binomial clinician / radiologist Both radiological exams should not be older than 3 months prior to inclusion visit (if not, to be repeated before treatment). - OO never treated or recurrent OO. - OO percutaneously accessible - Pain intensity is superior or equal to 40 mm on a VAS at inclusion visit. - Written informed consent signed by the patient or his representative (for minors, agreement of the child and signature of the two mandatory parents). - Patient affiliated to the social security.
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- Patient âgé de 10 ans et plus (accord des deux parents nécessaire jusqu’aux 18 ans de l’enfant) - Patient ayant un ostéome ostéoïde typique diagnostiqué sur des critères et radiologique, validé par un binôme clinicien/radiologue. Si les examens radiologiques datent de plus de 3 mois lors de l’inclusion, ils devront être refaits avant le traitement. - OO jamais traité ou récidive d’un ostéome ostéoïde traité antérieurement - OO accessible par voie percutanée - Douleur osseuse cotée à 40 ou plus sur une échelle visuelle analogique de 0 à 100 le jour de la visite d’inclusion. - Patient étant affilié à un régime de la sécurité sociale ou ayant droit ou CMU. - Patient qui accepte de signer un consentement écrit après explication orale. Pour les mineurs signatures des deux parents obligatoires et accord de l’enfant.
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E.4 | Principal exclusion criteria |
- Patients with other diseases or receiving treatment that may impact on bone tissue or its metabolism. - Patients suffering from renal failure (i.e. lower than 60 ml/min according to the Cowcroft equation). - Patients with severe hepatic impairment, which may alter the rate of alkaline phosphatase, such as hepatitis, sclerosing cholangitis, primary biliary cirrhosis, hepatic cirrhosis. - Patients with a history of iritis or uveitis. - Patient with untreated rickets or osteomalacia. - Patient with untreated dental infection or planed dental surgery during the study period. - Patient with untreated infection of the external auditory canal (ex: furuncle, eczema superinfection) - Patient already treated by bisphosphonates. - Patients with hypersensitivity to the active substance, to other bisphosphonates or to any of the excipients (List of excipients: mannitol (E421), sodium citrate (E331), water for injections). - Pregnant or breastfeeding women, or women of child bearing potential (women following menarche and until post-menopause) planning pregnancy during the course of the study (a highly effective contraceptive measure is required during the period of treatment.) - Patient enrolled in another biomedical research protocol and during the whole study
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- Patient ayant d’autres maladies ou recevant des traitements pouvant retentir sur le tissu osseux ou son métabolisme. - Patient ayant une insuffisance rénale (définie sur une clairance de la créatinine, calculée par la formule de Cowcroft, inférieure à 60 ml/mn). - Patient ayant une insuffisance hépatique sévère, qui pourrait modifier le taux de phosphatases alcalines, telle qu’hépatite, cholangite sclérosante, cirrhose biliaire primitive, cirrhose hépatique. - Patient avec infection dentaire non traitée ou une chirurgie dentaire planifiée au cours de la période d'étude. - Patient avec une infection non traitée du conduit auditif externe (exemple : furoncle, surinfection d'un eczéma). - Patient ayant des antécédents d’iritis ou d’uvéite. - Patient ayant un rachitisme ou une ostéomalacie non traité. - Patient déjà traité par des bisphosphonates, utilisation antérieure de bisphosphonates ou de fluor. - Patient ayant une hypersensibilité connue au principe actif, à d’autres bisphosphonates ou à l’un des excipients (Liste des excipients : mannitol (E421), citrate de sodium (E331), eau pour préparations injectables).
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E.5 End points |
E.5.1 | Primary end point(s) |
Success of treatment: Success is defined as a percentage of decrease in bone pain measured on a visual analog scale (VAS) between baseline (inclusion visit) and the end of treatment larger or equal to 70%. End of treatment is set at one month after the last administration of bisphosphonates and three months after the percutaneous thermal destruction (ie 4 months after inclusion visit = V4 visit, for both groups). |
Le critère d'évaluation principal est le pourcentage de diminution de la douleur entre la visite d'inclusion (avant début du traitement) et la fin du traitement en visite V4 (4 mois +/- 5 jours après inclusion) ce qui correspond à 3 mois (+/- 5jours) après le geste percutané et 1 mois (+/- 5 jours) après la dernière perfusion de bisphosphonates, c'est à dire à trois mois de la mise en route du traitement dans les deux bras. Le traitement par bisphosphonates sera jugé non-inférieur au traitement percutané si le pourcentage de diminution de la douleur osseuse est égal ou supérieur à 70%.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Success of treatment: Success is defined as a percentage of decrease in bone pain measured on a visual analog scale (VAS) between baseline (inclusion visit) and the end of treatment larger or equal to 70%. End of treatment is set at one month after the last administration of bisphosphonates and three months after the percutaneous thermal destruction (ie 4 months after inclusion visit = V4 visit, for both groups). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
•Pain, assessed by Visual Analog Scale (VAS, annex 2) as an average over the past 48 hours, at V1 (1 month +/- 5 days), V2 (2 months +/- 5 days), V3 (3 months +/- 5 days), V7 visits (16 months +/- 1 month) and, in case of additional infusions, at V5 (7 months +/- 15 days) and V6 (10 months +/- 15 days) •Patient'Global Impression of change (PGIC) from V2 to V7 visits (PµGIC, annex 3). •Consumption of analgesic and NSAIDs:
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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 | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
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.6.13.1 | Other scope of the trial description |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | No |
E.8.2.3.1 | Comparator description |
Destruction tumorale percutanée |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 14 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 0 |