Trial ID: ISRCTN15367345
Other - Phase 2 - No longer available
Background and study aims People develop type 1 diabetes (T1D) because their immune system, the part of the body which helps fight infections, mistakenly attacks and destroys the insulin-producing cells in the pancreas (beta cells). When the immune system destroys these cells, the body’s ability to produce insulin decreases, blood glucose levels run high, and T1D develops. At the time of diagnosis of T1D, there are usually a small number of beta cells (10-20%) left in the pancreas, which still produce small amounts of insulin. We call this level of activity beta-cell function. Most people with T1D will eventually stop producing insulin themselves. This may occur rapidly in a few months, or more slowly over several years. However, the longer people with T1D can produce their own insulin, the better it is for the control of blood glucose levels and to avoid long-term complications. Previous research has shown that a drug called anti-thymocyte globulin (ATG) may help prevent the immune system from attacking and destroying the insulin-producing beta cells. The aim of this study is to find the minimum effective low dose of ATG in young people newly diagnosed with T1D that can slow the decline of beta-cell function and preserve the body’s own insulin production, and has manageable side effects. Who can participate? Patients aged 5 to 25 years diagnosed with T1D in the previous 3 weeks What does the study involve? Participation will involve 8-10 hospital/clinic visits over about 13 months, and some at-home collections. Most visits take 1-4 hours. Treatment is given over two consecutive days and includes one overnight stay for most participants. After signing the Consent Form, participants will be invited for a screening visit to see if they are eligible for the study. They will be asked about their diabetes diagnosis, medical history, and recent medications and vaccinations. They will have blood samples taken which include tests to check they are fit and well, and to screen for T1D related auto-antibodies and c-peptide levels. Eligible participants will have a baseline visit no more than 3 weeks after the screening visit and undergo a number of assessments including height/weight, a physical exam, blood pressure, heart rate, temperature/breathing rate, review of any recent changes in health and medications and a mixed meal tolerance test (MMTT). If they are fit and well, participants will have a treatment visit no more than 9 weeks from the T1D diagnosis. Most participants will need an overnight stay for this visit, as there is no gap between treatment days 1 and 2. They will be able to eat and drink and take insulin as normal. Treatment will be assigned in a random way (by chance), much like flipping a coin, by a central computer programme. This treatment will be either an active drug (a low dose of ATG) or a placebo (dummy drug). Neither participants nor the research team will know which treatment they are given but overall 3 out of 4 people will get the active treatment. On treatment day 1 a nurse or doctor will talk to participants about their health. A cannula (tube) will be inserted into a vein, through which the study treatment will be given. Pre-treatment medications (an antihistamine, an anti-inflammatory and paracetamol) will be given. Some are given orally and others through the cannula. These medications are a precaution because some people can react to the study treatment. Treatment infusion will take at least 12 hours, blood samples will be collected and vital signs (blood pressure, heart rate, oxygen saturation, breathing rate and temperature) will be checked throughout. On treatment day 2 participants will start their second trial treatment infusion at least 12 hours after completing the first. With the exception of the study drug infusion which will only last 8 hours, day 2 is the same as day 1, including pre-treatment medications, collecting blood samples, and measuring vital signs. Where possible, the cannula will be kept in place and used again on day 2. Participants will be able to go home after the infusion, once the research team agrees they are fit and well enough to do so. Participants will have six follow-up visits at the hospital/clinic during the 12 months after finishing the study treatment. Visits will include medical review, height/weight, vital signs, blood samples and for visits 3, 6 and 12 an MMTT. Participants will also be asked to collect the following samples at home: collection of dried blood spot samples (via a small finger prick) and blood glucose measurements before and after a liquid meal (like the milkshake drink for the mixed-meal tolerance test); collection of urine and stool samples at home within 1 week before or after the baseline, 3, 6- and 12-month follow up visits; a short trial diary to complete at home following trial treatment, covering things like taking any medications and describing any possible side effects; a continuous glucose monitoring (CGM) device (provided by the study) for 14 days after each of the 3-, 6- and 12-month follow up visits, to measure blood glucose levels 24 hours a day. What are the possible benefits and risks of participating? Where possible, study visits will be booked to coincide with normal hospital visits to minimise the number of trips. Participants may experience some brief and/or minor discomfort when blood is taken or the cannula is inserted, and mild bruising or swelling can occur at the site. Occasionally, some people may become lightheaded during the procedure. Participants will need to have fasted for 8 hours (drinking water is allowed) before four of the hospital/clinic visits and before the monthly at-home dried blood spot sample collection. Participants may experience side effects from the study treatment (details in the participant information sheet). Participants should discuss their participation in this study with any insurance providers they have (e.g. travel insurance, protection insurance, life insurance, income protection, critical illness cover and private medical insurance) and seek advice if necessary, as failure to notify them may affect or invalidate their cover. Where is the study run from? University of Cambridge (UK) on behalf of the sponsor University Hospital Leuven (UZ Leuven, Belgium). When is the study starting and how long is it expected to run for? August 2018 to November 2024 Who is funding the study? European Commission Who is the main contact? Dr Emile Hendriks MELD-ATG@medschl.cam.ac.uk
Universitair Ziekenhuis Leuven
Source: https://www.isrctn.com/