E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary hyperparathyroidism in patients with MEN1 |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10020707 |
E.1.2 | Term | Hyperparathyroidism primary |
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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 study the activities of Cinacalcet- HCl administered to patients with MEN1-associated primary hyperparathyroidism (plasma PTH> 7.6 pmol / ml or 70 pg / ml and total serum calcium> 10.3 mg / dl or 2.57 mmol / l) at doses of 30 mg 1-2 times / day (starting with 30 mg per day, increasing the dose to 30 mg 2 times day, up to a maximum of 40-50 mg 2 times per day, if at the next inspection calcemia was still greater than 10.3 mg / dl or 2.57 mmol / l), considering: a) reduction or normalization of the levels of PTH and calcium; b) reducing or maintaining the size of parathyroid tissue affected |
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E.2.2 | Secondary objectives of the trial |
a) to evaluate changes with respect to the baseline visit of the bone turnover markers, both serum and urine levels and bone mass (expressed as Z-score); b)to assess the changes of other hormones related to MEN1 syndrome; c) to assess the safety and tolerance of the drug |
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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 |
FARMACOGENETICA: Versione:1 Data:2008/09/29 Titolo:cinetica delle variabili alleliche del recettore sensibile al calcio Obiettivi:correlazione genotipo/fenotipo
FARMACOCINETICA/FARMACODINAMICA: Versione:1 Data:2008/09/29 Titolo:valutazione di paratormone e calcemia totale dopo la dose mattutuina del farmaco in studio Obiettivi:valutazione di paratormone e calcemia totale dopo la dose mattutuina del farmaco in studio
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E.3 | Principal inclusion criteria |
1. Both genders and age >18 years 2. Serum levels of PTH and calcium above the upper limit of the range of reference 3rd Normal blood count and normal liver function (ALT, AST and alkaline phosphatase in normal range and total bilirubin <1.5 mg/ml) and renal (normal uremic levels and creatinine clearance> 50 ml/min) 4. Performance status <2 (ECOG) 5. Written informed consent 6. Female subjects treated with stable doses of SERMs or ERT will be eligible |
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E.4 | Principal exclusion criteria |
1. Pregnancy (assessed by specific test) and lactation. The state of pregnancy during treatment involves the suspension of therapy and exit of patients from the study. 2. Creatinine clearance <50 ml/min (0.83 ml/sec) 3. Treatment with bisphosphonates or peptides of PTH, strontium ranelate or cinacalcet itself within 90 days before the baseline visit 4. Because cinacalcet inhibits cytochrome P450 2D6 (CYP2D6), we exclude patients requiring drugs that are metabolized by this enzyme and with a narrow therapeutic index, such as flecainide, thioridazine and many tricyclic antidepressants 5. Presence of insulinoma 6. Insulin dependent diabetes mellitus with poor metabolic compensation (HbA1c> 8% or <5%) 7. Any serious disease with concomitant risk of life, in particular cardiovascular diseases such as heart disease of any type, uncontrolled hypertension, inastable angina and severe coronary disease 8. Patients included in a clinical treatment protocols in the last 6 months 9. Known hypersensitivity to cinacalcet |
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E.5 End points |
E.5.1 | Primary end point(s) |
Currently, there is no drug prevention or cure of the MEN1-associated primary hyperparathyroidism and the study preposes to assess whether cinacalcet can be considered as an alternative treatment for patients MEN1. |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
controllo della crescita del tessuto paratiroideo e di altre secrezioni neuroendocrine |
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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) | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 4 |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |