E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary hyperparathyroidism in patients affected with multiple endocrine neoplasia syndrome type 1 (MEN1) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10027180 |
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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 |
To evaluate the efficacy and safety profile of cinacalcet, a second generation calcimimetic, in the treatment of primary hyperparathyroidism in patients with MEN 1 in whom surgery has failed or is not indicated. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the effects of cinacalcet on renal function and complications, neuromuscular symptoms, neuropsychiatric and cognitive features, heart function, gastrointestinal symptoms and perceived quality of life. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
� Age > 18 years � Total serum calcium > 10.4 mg/dl and < 13 mg/dl (at least 2 consecutive evaluations within the last 6 months) � Ionized serum calcium > 1.32 mmol/L � PTH > 65 pg/ml (at least 2 consecutive evaluations within the last 6 months) � 25(OH)-vitamin D > 30 ng/ml |
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E.4 | Principal exclusion criteria |
� Pregnancy � Chronic kidney disease � Chronic hepatic � Treatment with drugs metabolized by hepatic cytochrome P450 2D6 (CPY2D6), inhibitors or inducers of CYP3A4 and/or CYP1A2 � Treatment with biphosphonates or fluoride within (60 days before baseline) � Treatment with anti convulsive drugs or positive history of epilepsy or convulsions. � Secondary hyperparathyroidism � Other causes of hypercalcemia |
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate the efficacy and safety profile of cinacalcet, a second generation calcimimetic, in the treatment of primary HPT in patients with MEN 1 in whom surgery has failed or is not indicated, according to the NHI Consensus. The primary objective will be to assess the ability of the drug to reduce serum calcium, urinary calcium and PTH as well as to reduce/normalize bone metabolism alterations. |
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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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | Yes |
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.8.3 |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |