E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
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 | 10028414 |
E.1.2 | Term | Myalgic encephalomyelitis |
E.1.2 | System Organ Class | 100000004862 |
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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 |
The main purpose is to evaluate feasibility and safety of daratumumab subcutaneous injections in patients with moderate to severe ME/CFS. |
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E.2.2 | Secondary objectives of the trial |
We will also collect efficacy data. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
ME/CFS according to Canadian consensus criteria (2003) (1);moderate (mainly housebound) to severe (mainly bedridden) disease. Age 18 to 65 years. Signed informed consent. Duration of ME/CFS disease at least two years. The ME/CFS disease should have a defined onset, e.g. after an initial infection or other immunological trigger.
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E.4 | Principal exclusion criteria |
Chronic fatigue conditions not fulfilling Canadian consensus criteria. Age under 18 or over 65 years. Mild or mild-to moderate ME/CFS. Very severe ME/CFS, where patient is unable to travel to the hospital for intervention and assessments. Participation in a clinical trial with intervention aimed at ME/CFS during the last two years. Endogenous depression. Known multi-allergy with clinically assessed risk for hypersensitivity to daratumumab. Known contraindication to daratumumab. Significant comorbidity with reduced organ function (kidney, liver, heart, pulmonary). Previous long-term systemic treatment with immunosuppressants the last two years, excluding short steroid courses in e.g. obstructive lung disease. Chronic infections, including chronic hepatitis B or C, HIV, or other relevant infection. Previous or concomitant malignant disease, except basal carcinoma of the skin, or carcinoma in situ in the uterine cervix. Pregnancy or lactation. Inability to comply with protocol including follow-up.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome measures are safety and tolerability as measured by treatment-emergent adverse events > CTCAE grade 1.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Adverse events will be recorded bi-weekly during treatment period (weeks 12 to 20) and monthly until the end of study (52 weeks), and a total evaluation performed at end of study. A safety review will be performed after two patients have completed treatment. |
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E.5.2 | Secondary end point(s) |
Descriptive endpoints: Changes from baseline/run-in through follow-up for: - SF36 domains including SF36 Physical Function (SF36-PF) and SF36 Bodily pain (SF36-BP) - DSQ-SF - Self-reported Function level - Steps per 24 hours. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline, end of run-in (12 weeks) and at 16, 18, 20, 22, 24, 28, 32, 40, 48 and 52 weeks. |
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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) | 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.8.3 |
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
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |