E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Ulcerative colitis |
Colitis ulcerosa |
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E.1.1.1 | Medical condition in easily understood language |
Ulcerative colitis |
Blødende tyktarmsbetændelse |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
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 |
Investigate the effects of adipose derived mesenchymal stem cells as a local treatment of ulcerative colitis |
At undersøge om behandling med injektion af allogene fedtderiverede mesenkymale stamceller lokalt i slimhinden hos patienter med colitis ulcerosa (CU) reducerer graden af slimhindeinflammation og tarmsymptomer. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inklusionskriterier a) Alder 18-70 år b) Kendt colitis ulcerosa med udbredning begrænset til venstre side af tyktarmen c) Mild-moderat aktivitet i CU vurderet ved komplet Mayoscore > 2 og < 10 d) Taler og forstår dansk e) Fuldt informeret samtykke
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E.4 | Principal exclusion criteria |
Eksklusionskriterier a) Svær sygdomsaktivitet vurderet på Mayoscore (score > 10) b) Akut svær colitis ifølge Truelove-Witts kriterier c) Sygdomsudbredning proksimalt for venstre fleksur (ekstensiv colitis) d) Aktiv malign sygdom e) Graviditet (kontrolleres med graviditetstest (urin-HCG), hvis fertil kvinde) f) Ryger (defineret som rygestop< 12uger) g) Penicillinallergiker (slutproduktet indeholder spor af penicillin)
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E.5 End points |
E.5.1 | Primary end point(s) |
Reduceret inflammatorisk aktivitet vurderet med endoskopisk Mayoscore (bilag 1) 14 dage efter behandling med injektion af allogene fedtderiverede mesenkymale stamceller. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Dag 14 og 30 efter behandling (dag 4 og 7 er valgfrie) |
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E.5.2 | Secondary end point(s) |
1) Reduceret inflammatorisk aktivitet vurderet med endoskopisk Mayoscore 4, 7, 14 og 30 dage efter behandlingen. 2) Bedring i histologisk graduering af inflammation i vævsprøver dag 4, 7, 14 og 30 efter behandling. 3) Bedring i symptomer 4, 7, 14 og 30 dage efter behandling vurderet ved komplet Mayoscore (bilag 1) 4) Komplikationer/adverse events ved behandlingen i relation til dosisøgning 5) Påvise dannelsen eller fraværet af HLA-antistoffer, svarende til donors HLA-type, som følge af behandlingen. 6) Påvise forskelle imellem forskellige donorers mesenkymale stamcellers evne til immunmodulering vha. potency assays.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Dag 14 og 30 efter behandling (dag 4 og 7 er valgfrie)
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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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | No |
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 | 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 | Yes |
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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The total duration of the project is 2.5 years. Trial participants are expected to be included and followed from 03.04.23 (first patient, first visit) to 01.10.24 (last patient, last visit). After that, there will be a period of 9 months with data processing. The clinical effects of the trial are expected to be published immediately after LPLV
In E.8.9.1/2, the time period for patient inclusion and treatment is specified. |
Projektets samlede varighed er 2,5 år. Forsøgsdeltagere forventes inkluderet og fulgt fra 03.04.23 (first patient, first visit) til 01.10.24 (last patient, last visit). Herefter vil være periode på 9 måneder med databehandling. De kliniske effekter af forsøget forventes publiceret umiddelbart efter LPLV
I E.8.9.1/2 er angivet tidsperioden for patientinklusion og behandling. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |