E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cancer related fatigue in patients with haematological cancer disease |
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E.1.1.1 | Medical condition in easily understood language |
Fatigue in patients with cancer of the bone marrow or lymphomas |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066564 |
E.1.2 | Term | Chronic fatigue |
E.1.2 | System Organ Class | 100000004867 |
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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 |
To study if methylphenidate is superior compared to placebo in relieving fatigue and thereby improving • hours awake • time spend at work, being social, house work / gardening, being outside, participating in excercise • WHO performance status • muscle strength and endurance • quality-of-life • number of blood transfusions
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Malignant haematological disease such as o Myeloproliferative neoplasm o Myelodysplasia / Acute Myeloid Leukemia / Chronic Myelomonocytic leukemia o Acute lymphoblastic leukemia o Malignant lymphoma o Chronic lymphocytic leukemia o Multiple myeloma • Patient reported fatigue equals to a VAS score of 4 or more on a scale of 0 to 10 on (0 = no fatigue to 10 = worst possible fatigue). Score must be the patients retrospective estimate of usual fatigue during the past two weeks • Out-patient at inclusion • Hb ≥ 5 mmol/l on the past three hb measurements • Age ≥ 18 years • Ability to read and understand Danish language • Safe contraception for fertile women
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E.4 | Principal exclusion criteria |
• Chemotherapy within last 8 weeks. Patients on a stable dose previous 4 weeks of the following, may be included: o Kinase inhibitors (such as Imatinib, Dasatininb, Nilotinib, Ruxulitinib, Bosutinib and others) o Hydroxyurea o Chlorambucil o Busulfan o Melphalan o alfa-interferon o IMIDs (such Thalidomide, Lenalidomide, Pomalidomide and others) o monoclonal anti-bodies o 5-azacytidin o Combinations of above mentioned drugs and with corticosteroids (CS) are allowed as long as CS dose restrictions are followed. • Glucocorticoid treatment exceeding the equal of prednisolone 10mg / day or equivalent average dose / week and dosage must have remained stable during the past 4 weeks. • Current infection • Previous or current diagnosis made by a psychiatrist of psychosis, mania, or Tourette • Known previous suicidal attempts • Current psycho-pharmacological treatment • Known cardio-vascular disease. Patients with known stable angina pectoris may be included. • Prolonged QT interval corrected (QTc) >500msec at screening ECG • Known cerebro-vascular disease • Uncontrolled hypertension defined as SBP > 160 mmHg or DBP > 100mmHg • Cognitive impairment as judged by investigator • Change in opiod dose during the past two weeks • Life expectancy < 4 months • EPO started or dosage changed < 6 weeks prior to inclusion • Hypothyroidism with thyroid hormone supplementation treatment started or dosage changed < 6 weeks before inclusion • Known hyperthyroidism • Known pheochromocytoma • Known glaucoma • Previous or current substance abuse • Use of monoamine oxidase inhibitors within last two weeks • Known allergy to or side-effects from previous methylphenidate treatment • Pregnancy or breast feeding • Serious medical illness which in the judgement of the investigator would make the patient inappropriate for inclusion in the study
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary end-point • Patient reported fatigue reduction after six weeks of MTP treatment measured by FACIT-F scale.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
End of six weeks, end of 13th week and end of study 15th week |
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E.5.2 | Secondary end point(s) |
Secondary end-points • Changes in hours awake • Changes in time spend at work, being social, house work / gardening, being outside, participating in excercise • Changes in WHO performance status • Changes in muscle strength and endurance • Changes in quality-of-life • Changes in number of blood transfusions
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
End of six weeks, end of 13th week and end of study 15th week |
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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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3 | Other | No |
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
| 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 | 7 |
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 | 7 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 0 |