E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
sperimentation only in patients |
|
MedDRA Classification |
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 |
Primary endpoint of the study is to estabilish the efficacy of retinoic acid treatment in patients with Cushing`s disease, a disorder as yet lacking causal medical therapy. Efficacy of retinoic acid will be evaluated in terms of decrease in ACTH and cortisol concentrations and improvement in features of hypercortisolism. Retinoic acid treatment is expected to lower hormone concentrations and ameliorate clinical features compared with the same prior to treatment. An additional primary objective is the evaluation of the safety profile of long-term retinoic acid administration to patients with Cushing`s disease, thus defining its benefit-risk ratio. |
|
E.2.2 | Secondary objectives of the trial |
Secondary endpoint of the study is to establish whether the cortisol-lowering effects of retinoic acid in Cushing`s disease result in a reduction in symptomatic drug requirements, i.e., drugs used to treat complications of chronic hypercortisolism such as antihypertensive medication, oral hypoglycemic agents and/or insulin, antidepressant and antidislipidemic drugs. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria: adult males or postmenopausal women; patients awaiting surgery, surgical failures or relapses; |
|
E.4 | Principal exclusion criteria |
Exclusion: women in child-bearing age; children, patients submitted to pituitary radiosurgery/radiotherapy. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint of the study is to estabilish the efficacy of retinoic acid treatment in patients with Cushing`s disease, a disorder as yet lacking causal medical therapy. Efficacy of retinoic acid will be evaluated in terms of decrease in ACTH and cortisol concentrations and improvement in features of hypercortisolism, e.g., glucose tolerance, insulin resistance, dyslipidemia, hypertension, weight excess, proximal muscle hypotrophy, osteopenia/osteoporosis, intraocular pressure and psychiatric disturbances. Retinoic acid treatment is expected to lower hormone concentrations and ameliorate clinical features compared with the same prior to treatment. An additional primary objective is the evaluation of the safety profile of long-term retinoic acid administration to patients with Cushing`s disease, thus defining its benefit-risk ratio.Aim of the present study is to establish the efficacy of retinoic acid in the treatment of Cushing`s disease. Drug efficacy will be evaluated in terms of reduction in ACTH and cortisol concentrations and improvement in clinical features of hypercortisolism. An additional primary objective is the evaluation of the safety profile of long-term retinoic acid administration to patients with Cushing`s disease, |
|
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 | 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) | 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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
|
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
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 | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |