E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Surgical Procedures, Operative [E04] |
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 |
Primary objective: - To assess the feasibility of a single intravenous injection of OTL38 in detecting endometriosis during surgery by determining the concordance between fluorescent signal and histopathological confirmed endometriotic tissue. - To assess the safety and tolerability of single intravenous dose of OTL38.
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E.2.2 | Secondary objectives of the trial |
Secondary objective: - Localization of occult endometriotic lesions with NIR imaging and OTL38, that would otherwise remain invisible with white light only.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Female (premenopausal) patients 18 years of age and older 2. Patients scheduled for elective surgery (laparoscopic or laparotomy) for the diagnosis or treatment of endometriosis 3. WHO performance score of 0-2 4. A negative serum pregnancy test at screening followed by a negative serum pregnancy test on the day of surgery or day of admission for female patients of childbearing potential 5. Female patients of childbearing potential agree to use an acceptable form of contraception from the time of signing informed consent until 90 days after study completion 6. Ability to understand the requirements of the study, provide written informed consent and authorization of use and disclosure of protected health information, and agree to abide by the study restrictions and to return for the required assessments |
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E.4 | Principal exclusion criteria |
1. Previous exposure to OTL38 2. Any medical condition that in the opinion of the investigators could potentially jeopardize the safety of the patient 3. History of anaphylactic reactions 4. History of allergy to any of the components of OTL38, including folic acid 5. Pregnancy (or positive pregnancy test) or breast-feeding 6. Presence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule 7. Impaired renal function defined as eGFR< 50 mL/min/1.73m2 8. Impaired liver function defined as values > 3x the upper limit of normal (ULN) for alanine aminotransferase (ALT) or aspartate aminotransferase (AST), alkaline phosphatase (ALP), or total bilirubin 9. Received an investigational agent in another investigational drug or vaccine trial within 30 days prior to surgery 10. Known sensitivity to fluorescent light |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint: - To determine the sensitivity or true positive rate (TPR) for OTL38 in combination with NIR fluorescence imaging, defined as the proportion of fluorescent positive tissue samples (of resected tissue or biopsies) that are histologically confirmed endometriotic tissue and FR+ relative to the total number of tissue samples confirmed to be FR+ and endometriotic tissue. Sensitivity = (True Positive)/(True Positive +False Negative). - False positive rate (FPR) for OTL38 in combination with fluorescent light, for the purpose of this protocol, will be calculated as 1 – the Positive Predictive Value (PPV) and is defined as the proportion of fluorescent positive tissue samples removed (of resected tissue or biopsies) that are histologically confirmed to be non-endometriotic tissue by the pathologist relative to the total number of tissue samples removed with fluorescent light imaging. False Positive Rate = (False Positives) / (True Positives + False Positives). - Incidence rates of all treatment-emergent AEs (TEAEs), adverse device effects (ADEs), and SAEs, from the time of OTL38 administration until follow-up visit 3.
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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) |
Secondary endpoint: - The proportion of lesions identification with OTL-38 and NIR that could not otherwise be identified by white light and finger palpation.
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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 | 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 | Yes |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | Yes |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 2 |
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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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 | |