E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
musculoskeletal health in children with cystic fibrosis |
muskuloskeletálního zdraví u dětí s cystickou fibrózou |
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E.1.1.1 | Medical condition in easily understood language |
musculoskeletal health in children with cystic fibrosis |
muskuloskeletálního zdraví u dětí s cystickou fibrózou |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10050713 |
E.1.2 | Term | Vitamin D |
E.1.2 | System Organ Class | 10022891 - Investigations |
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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 |
Maximum recommended doses of vitamin D given over two years to children with CF will normalize bone metabolism parameters and improve bone density and skeletal muscle force. |
Maximální doporučené dávky vitaminu D podávané během dvou let dětem s CF normalizují parametry kostního metabolismu a zlepší hustotu kostí a sílu kosterního svalstva. |
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E.2.2 | Secondary objectives of the trial |
Not Applicable |
Not Applicable |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Diagnosis of CF Age 8-16 years Able and willing to sign the informed consent and participate in the study
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Diagnóza CF Věk 8-16 let Schopnost a ochota podepsat informovaný souhlas a zúčastnit se studie |
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E.4 | Principal exclusion criteria |
Lung transplantation Bisphosphonate treatment Underlying disease that affects the bone metabolism other than CF Hypersensitivity to the IMP or additive substance (as per SMPC) Hypercalcaemia Renal osteodystrofy with hyperfosphatemia Other according to SMPC
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Transplantace plic Léčba bisfosfonáty Základní onemocnění, které ovlivňuje metabolismus kostí jiné než CF Hypersenzitivita na IMP nebo přídatnou látku (podle SMPC) Hyperkalcémie Renální osteodystrofie s hyperfosfatemie Jiné podle SMPC |
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E.5 End points |
E.5.1 | Primary end point(s) |
The planned examinations will make it possible to assess the effectiveness of vitamin D substitution, possibly adjust the dose, and further assess the degree of risk of fractures, or to start treatment for osteoporosis. |
Plánovaná vyšetření umožní posoudit účinnost substituce vitaminem D, eventuálně upravit dávku, a dále zhodnotit míru rizika vzniku zlomenin, případně zahájit léčbu osteoporózy. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Taking a blood sample from which the current state of bone metabolism will be determined, X-ray examination of the spine (side image) to rule out asymptomatic vertebral body fractures, examination of bone density to assess bone quality and examination of muscle strength and performance.
Vein blood sampling and urine samples will be performed at study initiation and at 3, 6, 12, 18, and 24-month intervals thereafter. An X-ray lateral image of the spine will be taken at the start of the study and then after 24 months Bone density and strength tests will be performed at the start of the study, after 12 and 24 months Examination of muscle strength and performance will take place at the start of the study, after 12 and 24 months |
Odběr vzorku krve, ze kterého bude zjištěn aktuální stav kostního metabolizmu, rentgenové vyšetření páteře (boční snímek) k vyloučení asymptomatických zlomenin obratlových těl, vyšetření kostní denzity k posouzení kvality kostní tkáně a vyšetření svalové síly a výkonu.
Odběr krve ze žíly a vzorek moči bude proveden při zahájení studie a poté v odstupech 3, 6, 12, 18 a 24 měsíců Rentgenový bočný snímek páteře bude proveden při zahájení studie a poté po 24 měsících Vyšetření kostní denzity a pevnosti bude provedeno při zahájení studie, po 12 a 24 měsících Vyšetření svalové síly a výkonu proběhne při zahájení studie, po 12 a 24 měsících |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
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.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | No |
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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LVLS |
Poslední vizita posledního pacienta |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |