E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immunoglobulin deficiency and recurrent infections with chronic fatigue syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Immunoglobulin deficiency and recurrent infections with chronic fatigue syndrome |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10008874 |
E.1.2 | Term | Chronic fatigue syndrome |
E.1.2 | System Organ Class | 10018065 - General disorders and administration site conditions |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10021485 |
E.1.2 | Term | Immunoglobulin G decreased |
E.1.2 | System Organ Class | 100000004848 |
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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 aim of this study is to improve chronic fatigue syndrome in patients with chronic immunodeficiency and recurrent infections |
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E.2.2 | Secondary objectives of the trial |
• to ameliorate the frequency and severity of infections • to assess the tolerability of HyQvia in patients with CFS and patients contentment with handling of self IgG application and the mechanical pump • to identify biomarkers for responsenter information in English and add any other language that is applicable |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Age 18 -65 years, both genders • immunoglobulin deficiency, with indication for immunoglobulin substitution therapy • recurrent bacterial or viral infections: > 4 infections per year, other than common cold • CFS (performance status < 50 points on the Bell CFS scale) • ALAT, ASAT, AP, gamma -glutamyl transpeptidase (g-GT) each < 3 x upper limit of normal (ULN) • Normal or slightly reduced hemoglobin (>12 g/dl male, >11g/dl female), normal or slightly reduced leucocytes (>3/nl), normal thrombocytes (150 - 370/nl)
• Normal PTT and INR/Quick
• normal renal function: serum creatinine < 1.2 mg/dl • Signed informed consent.
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E.4 | Principal exclusion criteria |
• Other serious underlying medical conditions which could impair the ability of the patient to participate in the study • Women of childbearing potential meeting any one of the following criteria o subject presents with a positive pregnancy test o subject is breast feeding o subject intends to begin nursing during the course of the study • Subject has participated in another clinical study and has been exposed to an investigational product (IP) or device within 30 days prior to study enrollment (exception: treatment with immunoglobulin pre-study) • Subject is scheduled to participate in another interventional clinical study involving an IP or device during the course of the study • Subject has known hypersensitivity to any of the components of the medicinal product • Severe dermatitis that would preclude adequate sites for safe product administration • Subject is a family member or employee of the investigator
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy to improve fatigue, assessed by: • CFS Symptom Scale • SF-36 physical functioning
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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) |
• Efficacy to control infections documented by patients diary (type of infection, symptoms, duration, severity, days missing work, antibiotics, hospitalization)
• Functional assessment of fatigue by measuring muscle strength, composition of body tissues by bioimpedance, vessel function by flow mediated dilatation and activity tracking. • Additional questionaires to assess symptoms and physical functioning: CFS symptom scoring and COMPASS-31 • Immune marker of response.
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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 | 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) | 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
| 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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |