E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
primary lactase deficiency |
deficit primario lattasi |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10017947 |
E.1.2 | Term | Gastrointestinal disorders |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
|
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 |
To test the efficacy of two different doses (low, standard) of tilattase enzymes on gastrointestinal symptoms and hydrogen excretion in subjects with primary lactase deficiency. |
Testare l'efficacia di due diverse dosi (bassa, standard) di tilattasi orali sui sintomi gastrointestinali e sulla escrezione di idrogeno in pazienti con deficit primario di lattasi. |
|
E.2.2 | Secondary objectives of the trial |
Due to the chronic features of the primary lactase deficiency, to assess the outcome of prolonged treatment with tilactase enzymes on: gastrointestinal symptoms, quality of life, markers of osteopoenia/osteoporosis |
In conseguenza della cronicita' del deficit primario di lattasi, dimostrare lefficacia di una terapia prolungata con tilattasi su: sintomi gastrointestinali, qualita' di vita, markers di osteopenia/osteoporosi |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects with persisting symptoms (i.e. longer than 6 months) located either in the upper and/or lower abdomen suggestive of lactose intolerance, fructose intolerance, small intestinal bacterial overgrowth (SIBO), functional gastrointestinal disorders (i.e. irritable bowel, functional dyspepsia, functional diarrhea) |
Soggetti con persistenza di sintomi (piu' di 6 mesi) localizzati addominali suggestivi di intolleranza al lattosio, intolleranza al fruttosio, contaminazione batterica dell'intestino tenue (SIBO), disordini funzionali gastrointestinali (intestino irritabile, dispepsia funzionale, diarrea funzionale). |
|
E.4 | Principal exclusion criteria |
Subjects with alarm symptoms (e.g. anaemia, unexplained weight loss, chronic pain, ineffective therapies) or older than 50 years will be further investigated with upper a/o lower endoscopy and blood tests in order to rule out inflammatory bowel diseases, celiac disease, intestinal neoplasms, colonic diverticula, and thyroid diseases. If needed, patients will undergo Rx barium enema for characterization of colonic diverticula and severe dolicocolon, as a potential cause of lower intestinal symptoms. Excluded from the study will be also non-compliant subjects or those with an history of liver, renal, lung, heart, metabolic, or neurological disease and those taking laxatives, antibiotics, prokinetics, or any other drug known to influence colonic flora or gastrointestinal motility in the month preceding the study. All patients with known causes of secondary osteoporosis other than lactose intolerance will be not included in the soubgroup evaluating bone markers. |
Soggetti con sintomi d'allarme (anemia,calo ponderale non altrimenti spiegabile, dolore cronico, terapie inefficaci) o di eta' superiore a 50 saranno sottoposti ad altre indagini con colonscopia, gastroscopia ed esami ematochimici per escludere malattie infiammatorie, celiachia, neoplasie intestinali, diverticoli del colon e malattie della tiroide. Se necessario, i soggetti saranno sottoposti ad un clisma opaco per valutare la presenza di diverticoli del colon e dolicocolon, poiche' queste situazioni potrebbero essere la causa dei sintomi intestinali. Inoltre saranno esclusi dallo studio i soggetti non collaboranti, quelli con storia di patologie epatiche, renali, polmonari, cardiache, metaboliche o neurologiche e coloro che nei mesi precedenti lo studio avranno assunto lassativi, antibiotici, procinetici o qualunque farmaco che possa aver influenzato la flora o la motilita' gastrointestinale. Tutti i soggetti nei quali si evidenziera' una causa secondaria di osteoporosi, oltre all'intolleranza al lattosio, non saranno inclusi nel sottogruppo per valutare i markers ossei. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
breath H2 excretion and gastrointestinal symptoms |
livelli di idrogeno nell'espirato con il breath test, sintomatologia gastrointestinale (valutazione con scale analogico-visive sul grado di intensita' dei sintomi) |
|
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 | Yes |
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 | No |
E.8.1.3 | Single blind | Yes |
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 | Yes |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
- Stesso farmaco ad altro dosaggio |
- same IMP used at different dosage |
|
E.8.2.4 | Number of treatment arms in the trial | 3 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 24 |
E.8.9.1 | In the Member State concerned days | 0 |