E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Phaeochromocytoma and Neuroblastoma |
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MedDRA Classification |
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 demonstrate that 123I-mIBG planar scintigraphy is sensitive and specific in confirming or excluding the diagnoses of neuroblastoma and phaeochromocytoma |
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E.2.2 | Secondary objectives of the trial |
To determine the incremental value of single-photon emission computed tomography (SPECT) for improving the sensitivity and specificity of 123I-mIBG planar scintigraphy for the diagnoses of neuroblastoma and phaeochromocytoma. To collect safety data on 123I-mIBG.
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1)a)The subject has known or suspected neuroblastoma and is undergoing evaluation of disease status (for which a mIBG scintigraphic examination is clinically appropriate) OR b) The subject is at least 18 years of age with either: i) Known phaeochromocytoma. ii) Suspected phaeochromocytoma based on abnormal levels of catecholamines or metabolites in the urine or blood with difficult to control chronic or paroxysmal hypertension and/or abnormalities in the adrenal region on ultrasound, CT, or MRI. iii)A diagnosis of a familial or hereditary condition known to be associated with phaeochromocytoma (multiple endocrine neoplasia, von Hippel-Landau disease, neurofibromatosis, etc).
(2) The subject is able and willing to comply with study procedures and a signed and dated informed consent is obtained.
(3) The subject is male; or a female who is either pre-menarchal, surgically sterile (has had a documented bilateral oophorectomy and/or documented hysterectomy), postmenopausal (cessation of menses for more than 1 year), non-lactating, or of childbearing potential for whom a urine pregnancy test (with the results known prior to investigational medicinal product administration) is negative.
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E.4 | Principal exclusion criteria |
(1) The subject was previously entered into this study or has participated in any other investigational medicinal product or medical device study within 30 days of enrolment. (2) The subject has a history or suspicion of significant allergic reaction or anaphylaxis to iodide or iodinated imaging agents. (3) The subject presents with any clinically active, serious, life-threatening disease other than neuroblastoma or phaeochromocytoma, with a life expectancy of less than 30 days or where participation in the study might compromise the management of the subject or other reason that in the judgement of the investigator(s) makes the subject unsuitable for participation in the study. (4) The subject has a history of renal insufficiency (serum creatinine more than 3.0 mg/dL [265 micromoles/L]). (5) The subject uses medications that are known to interfere with 123I-mIBG uptake and these medications cannot be safely withheld 24 hours before study procedures.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint is focal increased uptake (presence or absence) on planar scintigraphy which is consistent with active tumour (either phaeochromocytoma or neuroblastoma). The primary efficacy endpoint will be used for the primary analysis of imaging procedure’s sensitivity and specificity |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
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 | Information not present in EudraCT |
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 | Yes |
E.7.1.3.1 | Other trial type description |
Diagnostic confirmatory Phase III |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
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 | 6 |