E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cobalamin (vitamin B12) deficiency |
Cobalamine (vitamine B12) deficientie |
|
E.1.1.1 | Medical condition in easily understood language |
Vitamin B12 deficiency |
Vitamine B12 tekort |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
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 |
In this study we investigate the effects of two intranasal cobalamin dosing regimens i.e. 1000 microgram administered once every three days and 1000 microgram administered once every day for two weeks, followed by once every week in elderly cobalamin deficient subjects on total transcobalamin and holotranscobalamin concentration.
The objective of this study is to establish the optimal intranasal cobalamin dosing regimen in elderly.
|
In deze studie wordt het effect van twee intranasale cobalamine doseerregimes; 1000 microgram elke 3 dagen toegediend en 1000 microgram elke dag toegediend gedurende twee weken, gevolgd door 1000 microgram elke week bij cobalamine deficiente ouderen op de transcobalamine en holotranscobalamine serum concentraties met elkaar vergeleken.
Het doel van dit onderzoek is om het optimale intranasale doseerregime bij ouderen vast te stellen. |
|
E.2.2 | Secondary objectives of the trial |
Not applicable |
Niet van toepassing |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- 65 years of age or over
- Total cobalamin serum level less than 250 pmol/l
- Suspect for cobalamin deficiency; hyperhomocysteinemia (> 15 micromoles/l) and/or 2 or more symptoms of cobalamin deficiency e.g. fatigue, memory impairment, irritability, personality changes, muscle weakness, depression, poor appetite, weight loss
- Capable of understanding the study information
- Informed consent
|
- 65 jaar of ouder
- Cobalamineconcentratie in serum kleiner dan 250 pmol/l
- Aanwijzigingen voor cobalamine deficientie; hyperhomocysteinemie (> 15 micromol/l) en/of twee of meer symptomen van cobalamine deficientie zoals moeheid, geheugenstoornissen, geirriteerdheid, gedragsveranderingen, spierzwakheid, depressie, verminderde eetlust, gewichtsverlies
- In staat om de studieinformatie te begrijpen
- Informed consent |
|
E.4 | Principal exclusion criteria |
- Concomitant use intranasally administered medication
- Chronic rhinitis
- Use of cobalamin containing dietary supplements
- Severe Renal impairment i.e. MDRD less than 20 ml/min |
- Gelijktijdig gebruik van andere nasaal toegediende medicatie
- Chronische rhinitis
- Gebruik van cobalamine bevattende voedingssupplementen
- Nierinsufficientie MDRD klaring kleiner dan 20 ml/min |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in total transcobalamin and holotranscobalamin (holoTC) serum concentrations over time. |
Verandering in totale transcobalamine en holotranscobalamine (holoTC) serumconcentraties in de tijd. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Total transcobalamin and holotranscobalamin serum concentrations will be established at t = 11, 7, 14, 30, 60 and 90 days.
|
Totale transcobalamine en holotranscobalamine serum concentraties worden bepaald op t = 1, 7, 14, 30, 60 en 90 dagen.
|
|
E.5.2 | Secondary end point(s) |
Changes in methylmalonic acid (MMA) and homocysteïne (tHcy) serum concentrations and changes in Geriatric Depression Scale (GDS) 5 and Mini Mental State Examination (MMSE) scores over time. |
Veranderingen in methylmalonzuur en homocysteine serumconcentraties en veranderingen in de GDS (5) en MMSE scores in de tijd. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Methylmalonicacid and homocysteine serumconctrations will be evaluted at t = 1, 14 and 90 days
GDS and MMSE scores will be evaluated at t = 1 and 90 days |
methylmalonzuur en homocysteine serumconcentraties worden bepaald op dag 1, 14 en 90.
De GDS en MMSE scores zullen op dag 1 en 90 worden bepaald. |
|
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 | 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) | 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 | Yes |
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 |
zelfde product maar met een ander doseerregime |
same medicinal product but different dosing regimen |
|
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
| 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
|
The last visit of the last subject undergoing the trial. |
Het laatste bezoek van de laatste patient in het onderzoek. |
|
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 | |