E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028483 |
E.1.2 | Term | Mycosis fungoides |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 |
Proof of concept study for application of hyperpolarized [1-13C]Pyruvate in magnetic resonance spectroscopy for early detection of therapeutic effects in treatment of mycisis fugoides. |
|
E.2.2 | Secondary objectives of the trial |
to test whether the sensitivity of MR-hyperpolarized [1-13C]Pyruvate based MR-spectroscopy is high enough for future studies for of treatment effects in skin cancers. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria • Patients diagnosed with MF, untreated or in stable dose systemic treatment for at least 2 months and no topical treatment (topical steroids, nitrogen mustard or UV treatment) for at least 14 days. • At least 18 years of age. • Adequate hematologic and organ function, i.e. • ANC at least 1.500/uL • Platlet count >100/uL • Se bilirubin <1.5 UNL • ASAT, ALAT and AP up to 2.5 x UNL, up to 5 x UNL with liver metastases • Se creatinine up to 1.5 x UNL or creatinine clearance at least 30 ml/min • INR and aPTT at most 1.5 x UNL, except for patients on AK treatment • Women who are not postmenopausal or surgically sterile must have a negative serum or urine pregnancy test performed at time of inclusion in the study. Safe and highly effective contraception must be used throughout the study meaning either hormonal anti-conception or an anti-fertility intrauterine device. If the partner is non fertile or the patient has no sexual activities, this is also accepted. • Danish speaking • Able and willing to comply after informed consent
|
|
E.4 | Principal exclusion criteria |
• Contraindications to contrast enhanced MRI • Not able or willing to receive the treatment • Patients not willing to participate • Uncontrolled serious medical condition, such as uncontrolled heart disease, uncontrolled diabetes, intestinal obstruction, uncontrolled hypertension, or recent cerebral ischemia or cardiac events • Intolerant to Pyruvate • Pregnancy • Predicted remaining survival <3 months
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The study will include 20 patients with MF treated at Department of Dermatology, Aarhus University Hospital. The first up to 10 patients in the study will be used for optimization of the technique (scanning parameter settings an coil selection: either 13C/21Na coil with 10x10 cm FOW or 13C/1H coil with 5x5 cm FOW). If sufficient signals can be obtained, the study will move on and include further 10 patients, who will be scanned 3 times, before treatment (day 0), day 8 and day 21 after initiation of treatment. Scanning’s of anatomical sites for each patient include the selected target MF skin lesion and non-lesional skin. Moreover, 5 patients with psoriasis and 5 healthy volunteers will be scanned once.
The study will end after completing the above given exams. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Milestones: a) if metabolic data form the first patients examined for adjusting the system do show too low signal to noise level the program will be canceled
b) if the first metabolic data (before treatment) from MF patients are not different from the findings in patients with psoriasis the aim of the program shifts. |
|
E.5.2 | Secondary end point(s) |
if metabolic data form the first patients examined for adjusting the system do show too low signal to noise level the program will be canceled
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
after the first 10 MF patients |
|
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 | No |
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) | Yes |
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 |
|
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) | 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
| 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
|
a patients involvement ends at the control visit 14 days after the last MR examn. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |