E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hyperphosphatemia |
Hyperphosphatämie |
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E.1.1.1 | Medical condition in easily understood language |
increased phosphate concentration |
erhöhte Phosphatkonzentrationen |
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E.1.1.2 | Therapeutic area | Body processes [G] - Physiological processes [G07] |
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 |
Determination of abundances and relative frequencies of distinct bacteria in the collected samples from patients and the control group. Based on these data, potential shifts in the microbiome during Velphoro® medication will be assessed. |
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E.2.2 | Secondary objectives of the trial |
Determination of diversity measures describing the distribution of bacterial species within the microbiome (i. e. alpha-diversity, beta-diversity, Simpson index, Shannon-Wiener index), as well as the presence or absence of other medically relevant bacterial species (e,g, Pseudomonas, Enterobacteriaceae, Campylobacter etc.). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients: • Suffering from hyperphosphatemia • Current treatment with a stable dose of a non-iron containing phosphate binder • No or only parenteral iron application • Age of ≥ 18 years • Written informed consent prior to study participation • The subject is willing and able to follow the procedures outlined in the protocol
Control group: • Normal renal function • No hyperphosphatemia • Age- and sex-matched and oral disease status-matched (dental caries and periodontal disease) in comparison to the hyperphosphatemia group • Written informed consent prior to study participation • The subject is willing and able to follow the procedures outlined in the protocol |
Patienten: • Die an Hyperphosphatämie leiden • Die derzeitig in Behandlung mit einem Eisen-freien Phosphatbinder sind • Bei denen keine oder nur parenterale (außerhalb des Verdauungstraktes) Eisenanwendung stattfindet • volljährig • Die die Bereitschaft haben, den Anweisungen des Studienpersonals Folge zu leisten • Die eine Einwilligungserklärung unterschrieben haben
Kontrollgruppe: • Keine Beeinträchtigung der Nierenfunktion • Keine Hyperphosphatämie • Ein mit der Patientengruppe vergleichbares Alter, Geschlecht und Zahn(-fleisch)status • Unterschriebene Einwilligungserklärung • Bereitschaft, den Anweisungen des Studienpersonals Folge zu leisten |
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E.4 | Principal exclusion criteria |
• Currently on oral iron application • Antibiotic treatment within the last two months • Severe medical events within the last three months • Planned surgery for the duration of the sampling • Acute/chronic gastrointestinal infections • Smokers • Oral candidiasis • Oral cancer • Pregnant and lactating females • Haemochromatosis history • Committed to an institution by legal or regulatory order • Participation in a parallel interventional clinical trial • Receipt of an investigational drug within 30 days prior to inclusion into this study • The subject is mentally or legally incapacitated
Only for the patient group: • Allergy to Velphoro® • Never got any phosphate binder • Celiac disease or any other chronic inflammatory bowel disease • Previous major surgery in the gastrointestinal tract
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• Orale Einnahme eines Eisen-haltigen Medikamentes • Einnahme eines Antibiotikums innerhalb der letzten zwei Monate • Schwere medizinische Ereignisse innerhalb der letzten drei Monate • Ein geplanter operativer Eingriff innerhalb der Studiendauer • Akute- oder chronische Darmerkrankungen • Raucher (außer gelegentlich) • Orale Kandidose • Mundhöhlenkarzinom • Hämochromatose • Schwangerschaft und Stillzeit • Personen, die auf gerichtliche oder behördliche Anordnung in einer Anstalt untergebracht sind • Gleichzeitige Teilnahme an einer anderen klinischen Studie • Einnahme eines anderen Prüfpräparates innerhalb der letzten 30 Tage • Einwilligungsunfähigkeit und / oder nicht in der Lage, sein Wesen, Bedeutung und Tragweite der Studie zu verstehen und ihre Einwilligung schriftlich abzugeben
Zusätzlich nur für die Patientengruppe: • Allergie gegen Velphoro® • Bisher keine Einnahme eines Phosphat-Binders (Eisen-frei) • Glutenunverträglichkeit oder chronisch entzündliche Darmkrankheit • Vorangegangene große Operationen am Magen-Darm-Trakt
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E.5 End points |
E.5.1 | Primary end point(s) |
A significant shift (non-parametric Mann-Whitney-U test) in the relative abundance of iron-depending bacterial species will reject the 0-hypothesis |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
screening, 1 week, 4 weeks |
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E.5.2 | Secondary end point(s) |
- A significant shift in total bacterial cell numbers - A significant shift in species diversity - A significant change in clinical parameters, including dental.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
screening, 1 week, 4 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 | 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 | Yes |
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
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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
| 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 | 2 |
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |