E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Specific polysaccharide antibody deficiency. |
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E.1.1.1 | Medical condition in easily understood language |
Specific polysaccharide antibody deficiency is an immunodeficiency characterized by a deficient production of antibodies to encapsulated bacteria, leading to recurrent ENT and lung infections. |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Diagnosis [E01] |
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 |
The purpose of this study is to assess the diagnostic value of the Typhim antibody response and allohemagglutinin titers as an alternative to the Pneumovax 23 response to detect polysaccharide specific antibody deficiency.
Primary objective:
• To establish whether antibody response to Typhim in patients with suspected PID (1) and in healthy adults (2) is non-inferior relative to the antibody response to Pneumovax 23.
• To assess the correlation between Typhim antibody response and the pneumococcal antibody response.
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E.2.2 | Secondary objectives of the trial |
• To establish whether allohemagglutinin titers in patients with suspected PID (1) and in healthy subjects (2) are non-inferior relative to the antibody response to Pneumovax 23.
• To establish normal values for Typhim response and allohemagglutinins in healthy adults.
• To correlate the measured antibody response to the clinical phenotype.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Cohort 1:
Subjects, in whom humoral immunity is evaluated, will be included in cohort 1 when the below-mentioned criteria are fulfilled.
• Assessment of polysaccharide antibody response is indicated for the clinical care of the patient
• Age between 18 months and 55 years
• Informed consent given
Cohort 2:
Healthy volunteers, recruited at the travel clinic and by advertising, will be included in cohort 2 when the below-mentioned criteria are fulfilled.
• Age between 18 months and 55 years
• Informed consent given
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E.4 | Principal exclusion criteria |
• Age >55 years
• Age <18 months
• History of serious adverse reaction to a vaccine
• Vaccination with Typhim or Pneumovax 23 in 5 years prior to the study
• Pregnancy
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E.5 End points |
E.5.1 | Primary end point(s) |
• Sensitivity and specificity, predictive values and likelihood ratios of low Salmonella typhi capsular Vi response, with Pneumovax 23 as a reference standard.
• Correlation between Pneumovax 23 antibody response and Typhim antibody response.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At inclusion of 100 subjects and at inclusion end. |
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E.5.2 | Secondary end point(s) |
• Sensitivity and specificity, predictive values and likelihood ratios of low allohemagglutinin titers, with Pneumovax 23 as a reference standard.
• Receiver Operating Characteristic (ROC) curves for Typhim response and allohemagglutinins will be calculated using Pneumovax 23 as the reference standard to identify polysaccharide antibody deficient subjects.
• Association of low allohemagglutinins and low Typhim response to clinical signs of polysaccharide specific antibody deficiency (recurrent respiratory tract infections, bronchiectasis).
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At inclusion of 100 subjects and at inclusion end. |
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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 | No |
E.6.5 | Efficacy | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Diagnostic cohort study with within-subject comparison |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |