E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic non bacterial osteomyelitis |
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E.1.1.1 | Medical condition in easily understood language |
Chronic non bacterial osteomyelitis |
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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 | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10009081 |
E.1.2 | Term | Chronic osteomyelitis |
E.1.2 | System Organ Class | 100000004862 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
In a randomized double blind placebo controlled design in patients with chronic non bacterial osteomyelitis to investigate whether it is possible by treating with pamidronat to achieve a 1. Reduction of the inflammatory activity in the osteomyelytic boneproces measured by whole body MRIafter 9 months 2. Healing of the inflammatory activity in the osteomyelytic boneproces measured by whole body MRI after 9 months |
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E.2.2 | Secondary objectives of the trial |
1. Changes in osteomyelitic bonelesions in WB MRI 2. Changes in anterior chestwall (adults) in CT scan 3. Changes in patient selfreported pain and life quality 4. Changes in inflammatory markers in blood 5. Changes in bonemarkers in blood |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Mono-, oligo- eller multifocal inflammatory bonelesions (osteomyelitis, osteitis, osteosclerosis) 2.Diagnostic score iaccording to Jansson criteria > 39 or malignancy and infnfection excluded by biopsi 3.Symptoms > 6 weeks
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E.4 | Principal exclusion criteria |
- Age older than 65 years - Age younger than 9 years - Pregnant women or nursing (breastfeeding) mothers - Hypersensitivity to pamidronat, bisphosphonate og additives in pamidronate - Known history or current lymphoprolifereative disease - History of surgery on glandula thyroidea - Known alcohol/medical abuse - Poor dental status - Low Vitamin D- status - Liver/ kidney disease |
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E.5 End points |
E.5.1 | Primary end point(s) |
Changes in the osteomyelitc bonelesion measured by activity and healing on WB MRI |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Evaluation 9 months after first pamidronat treatment |
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E.5.2 | Secondary end point(s) |
1. WB MRI changes in osteomyelitic bonelesions (bonemarrowedema, periosteal reaction, fatinfiltration) 2. CT changes in anterior chestwall (adults) (hyperostosis, periosteal element) 3. Changes in PROM ( HAQ, SF-36, EQ5D score, CHAQ- 38, PedQL, PCQ, VAS score) 4. Changes in inflammatory markers in blood 5. Changes in bonemarkers in blood |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Secondary endpoint will be evaluated at month 3,6, 9 |
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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.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 | 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 | 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 | 2 |
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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The end of the study will occur when the last patient, last visit occurs (LPLV). The LPLV is either the date of the visit of the last patient to complete the study, or the date at which the last data point from the last patient, which is required for statistical analysis (i.e., key safety and efficacy results for decision making), is received, whichever is the later date. |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | |