E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
cerebral palsy, osteopenia, fracture |
cerebral parese, osteopeni, fraktur |
|
E.1.1.1 | Medical condition in easily understood language |
physical impairment, low bone density, broken bone |
spastisk lammelse, lav knogletæthed, knoglebrud |
|
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 | 10049088 |
E.1.2 | Term | Osteopenia |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.1 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10034157 |
E.1.2 | Term | Pathological fractures and complications |
E.1.2 | System Organ Class | 100000005035 |
|
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 | 10049904 |
E.1.2 | Term | Osteoporosis prophylaxis |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021740 |
E.1.2 | Term | Infantile cerebral palsy |
E.1.2 | System Organ Class | 100000012650 |
|
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 | 10006002 |
E.1.2 | Term | Bone pain |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10013522 |
E.1.2 | Term | Disuse osteoporosis |
E.1.2 | System Organ Class | 100000018618 |
|
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 evaluate the effect of 12 months treatment with i.v. zoledronic acid on bone mineralization in children with non-ambulatory cerebral palsy. |
Undersøge virkningen af 12 måneders behandling med i.v. zoledronsyre på knoglemineraliseringen hos børn med cerebral parese uden gangfunktion. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the effect of 12 months treatment with i.v. zoledronic acid on fractures, pain and quality of life in children with non-ambulatory cerebral palsy. |
Undersøge virkningen af 12 måneders behandling med i.v. zoledronsyre på frakturer, smerter og livskvalitet hos børn med cerebral parese uden gangfunktion. |
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Sub-study title: Bone microstructure and response to zoledronat in children with cerebral palsy. Date and version: Jan 9 2017, version 7 Objective: To evaluate the effect of 12 months treatment with i.v. zoledronic acid on bone microstructure in children with non-ambulatory cerebral palsy. |
Substudie: Effekten af zoledronat på knoglemikrostrukturen hos børn med cerebral parese. Dato og version: 9. jan 2017, version 7 Undersøge virkningen af zoledronsyre på knoglernes mikrostruktur hos børn med cerebral parese uden gangfunktion. |
|
E.3 | Principal inclusion criteria |
Child age 5-17 years Cerebral palsy GMFCS level 4 or 5 Bone Mass Density z-score below -1 |
Barn 5-17 år Cerebral parese GMFCS level 4 eller 5 Bone Mass Density z-score under -1 |
|
E.4 | Principal exclusion criteria |
Previous treatment with any disphosphonate Inability to receive medication via i.v. route Hydroxy-vitamin D level below 50 Bone metabolic disease Liver/kidney disease |
Tidligere behandling med bisfosfonat Umuligt at give i.v. medicin OH-D-vitamin under 50 Knoglemetabolisk sygdom lever- eller nyresygdom |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Bone Mass Density z-score change |
Bone Mass Density z-score ændring |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Pain score change Well-being score change Fracture occurence |
Smerte score ændring Velbefindende score ændring Frakturforekomst |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
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.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 | Yes |
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 | 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 | Yes |
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
| 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 | 6 |
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
|
LVLS |
Sidste besøg af sidste deltager |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |