E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary hyperparathyroidism (PHPT) is a common disease, characterized by a high-normal calcium concentration and an inappropriately increased parathyroid hormone (PTH) level |
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E.1.1.1 | Medical condition in easily understood language |
Primary hyperparathyroidism (PHPT) is a common disease, characterized by a high-normal calcium concentration and an inappropriately increased parathyroid hormone (PTH) level |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036693 |
E.1.2 | Term | Primary hyperparathyroidism |
E.1.2 | System Organ Class | 100000004860 |
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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 |
To determine the relevance of short-term calcimimetics treatment in primary hyperparathyroidism for decision of parathyroid surgery |
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E.2.2 | Secondary objectives of the trial |
•To assess the feasibility of the study design
•To assess if the ionized calcium level will be normalized during four weeks of treatment with Mimpara 30 mgx1
•To assess the safety during Mimpara treatment
•To compare changes in quality of life, psychological health, cognitive function and muscle strength between patients with PHPT randomized to treatment with Mimpara (M+) or not (M-) preoperatively
•To compare changes in quality of life, psychological health, cognitive function and muscle strength during treatment with Mimpara (M+) with postoperative changes
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Primary hyperparathyroidism, scheduled for parathyroid surgery
•Woman (part A only)
•Age > 40
•Understand the purpose of the study, able to participate in all tests included in the study model
•Signed informed consent
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E.4 | Principal exclusion criteria |
•Pregnancy
•Breast feeding
•Fertile woman not using contraceptives
•Impaired kidney function, GFR < 40 ml/min
•Intolerance to Mimpara
•Previously treated with Mimpara
•Participating in other ongoing clinical study
•Epilepsy
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E.5 End points |
E.5.1 | Primary end point(s) |
•If four weeks of treatment with Mimpara will normalize hypercalcemia in patients with PHPT |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
•Changes in quality of life in view of the physical symptoms and well-being, evaluated with two validated forms (QLQ-C30 and the Positive States of Mind (PSOM)) during Mimpara treatment and postoperatively
•Depression; evaluated with the Hospital Anxiety and Depression (HAD) scale during Mimpara treatment and postoperatively
•Muscle strength; repeated ascent from a chair, repetitive stand up on tiptoes (measures the number which can be carried out) and thigh and hand strength evaluated by simple dynamometric apparatus during Mimpara treatment and postoperatively
•AE/SAE recording and safety blood samples; serum levels of (p)-PTH, serum(s)-ionized serum calcium, p-total calcium, p-albumin, p-creatinine, p-phosphate, s-25-OH-D and thyroid-stimulating hormone (TSH) during Mimpara treatment and postoperatively
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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 | 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) | 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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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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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6 month follow-up, last visit of last subject |
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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 | |
E.8.9.1 | In the Member State concerned days | |