E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Type 1 diabetes |
Type 1 diabetes |
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E.1.1.1 | Medical condition in easily understood language |
Type 1 diabetes |
Type 1 diabetes |
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E.1.1.2 | Therapeutic area | Body processes [G] - Metabolic Phenomena [G03] |
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 main objective is to investigate whether autologous faecal microbiota transplantation (FMT) in patients with newly diagnosed type 1 diabetes (<100 days) preserves residual beta cell function and mass after 12 months |
Het primaire doel is om te bepalen of autologe fecale microbiota transplantatie (FMT) in patiënten met nieuw gediagnosticeerd type 1 diabetes (<100 dagen) behoud van residuele bèta cel functie en massa hebben na 12 maanden. |
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E.2.2 | Secondary objectives of the trial |
Beta cell mass will be related to parameters derived in the ENCAPSULATE-DM1, FMT preserve-DM1, or DIMID study such as beta cell function and immunity status |
Bètacel massa wordt gecorreleerd aan parameters uit de ENCAPSULATE-DM1, FMT preserve-DM1, of DIMID studies zoals bètacel functie en immuunstatus. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Previously participated in ENCAPSULATE-DM1, FMT preserve-DM1, or DIMID trial • Type 1 diabetes with the diagnosis being made in the last 4.5 years • Presence of at least one autoantibody associated with type 1 diabetes (anti-GAD-65, anti-IA2, islet cell antibodies, insulin autoantibodies) • Age ≥ 18 years • BMI 18-30 kg/m2 • Insulin use
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• Eerdere deelname aan ENCAPSULATE-DM1, FMT preserve-DM1, of DIMID studie • Type 1 diabetes gediagnosticeerd in de laatste 4.5 jaar • Aanwezig van ten minste één autoantistof geassocieerd met type 1 diabetes (anti-GAD-65, anti-IA2, islet cell antistof, insuline autoantistof) • Leeftijd ≥ 18 jaar • BMI 18-30 kg/m2 • Insuline gebruik
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E.4 | Principal exclusion criteria |
• Inability to provide written informed consent • Other medication use than insulin • Smoking • Evidence of compromised immunity • Presence of a second autoimmune disease (other than type 1 diabetes); e.g. celiac disease, hyper- or hypothyroidism, inflammatory bowel disease. Vitiligo is allowed. • Pregnancy or the wish to become pregnant within 6 months after the study • Breastfeeding • Liver disease defined as aspartate aminotransferase or alanine aminotransferase level of more than three times the upper limit of normal range • Renal disease defined as MDRD < 40 ml/min/1.73 m²
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• Niet in staat om schriftelijk toestemming te geven • Andere medicatie dan insuline • Roken • Bewijs van gecompromitteerde immuniteit • aanwezigheid van een tweede autoimmuun ziekte (iets anders dan type 1 diabetes); zoals coeliakie, hyper- of hypothyroïdisme, inflammatory bowel disease (IBD). Vitiligo is toegestaan • Zwangerschap of een kinderwens tot 6 maanden na de studie • Borstvoeding • Leveraandoening gedefinieerd als aspartaat aminotransferase of alanine aminotransferase waarden van meer dan 3 keer de bovengrens van de normale range. • Nieraandoening gedefinieerd als MDRD < 40 ml/min/1.73 m²
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is to determine residual beta cell mass using 68Ga-NODAGA-exendin-4 tracer in patients received faceal microbiota transplantation during the ENCAPSULATE-DM1, FMT preserve-DM1, or DIMID trial. |
Het primaire einddoel is het bepalen van residuele bètacel massa door het gebruik van 68Ga-NODAGA-exendin-4 tracer in patiënten die fecale microbiota transplantatie hebben gehad tijdens de ENCAPSULATE-DM1, FMT preserve-DM1, of DIMID studies. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
after the administration of the tracer and the PET/CT scan |
Na toediening van de tracer en de PET/CT scan |
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E.5.2 | Secondary end point(s) |
Beta cell mass will be related to parameters derived in the ENCAPSULATE-DM1, FMT preserve-DM1, or DIMID study such as beta cell function and immunity status |
Bètacel massa wordt gecorreleerd aan parameters uit de ENCAPSULATE-DM1, FMT preserve-DM1, of DIMID studies zoals bètacel functie en immuunstatus. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
After the PET/CT scan en analyses of all parameters. |
Na de PET/CT scan en analyses of alle parameters. |
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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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
In this study, we want to investigate whether autologous faecal microbial transplantation (FMT) in patients with newly diagnosed type 1 diabetes (<100 days) preserves residual beta cell function and mass after 12 months. The 68Ga-NODAGA-Exendin-4 tracer is used to image beta cell mass. This will help us to understand how to prolong or even consolidate the ‘honeymoon phase’ in T1D, which will decrease patient dependence on insulin injections or infusions. |
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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 | 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 |
Eenarmige studie: een groep van patiënten met een fecale microbiota transplantatie. |
Single arm study: one group of patients with faecal microbiota transplantation |
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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.8.2.4 | 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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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 | |
E.8.9.1 | In the Member State concerned days | |