Clinical Trials

Date: 2016-09-07

Type of information: update on patient enrollment

phase: 2

Announcement: update on patient enrollment

Company: Diamyd Medical (Sweden)

Product: Diamyd® diabetes vaccine

Action mechanism:

• protein. Diamyd® is an antigen-based diabetes therapy under development to prevent, delay, or stop the autoimmune attack on beta cells in type 1 diabetes and other forms of autoimmune diabetes, thereby preserving the body's capacity to regulate blood sugar. The active substance in the Diamyd® diabetes vaccine is glutamic acid decarboxylase isoform 65kDa (GAD). GAD is one of the most important targets when the immune system attacks the beta cells in autoimmune diabetes. Accordingly, GAD is an autoantigen. Treatment using Diamyd® is intended to stop the autoimmune attack against the beta cells by inducing tolerance to GAD.

Disease: type 1 diabetes

Therapeutic area: Metabolic diseases

Country: Sweden

Trial details:

• The new study, named DiAPREV-IT 2, is a researcher-initiated study comprising 80 children aged four and above with a 50-percent risk of developing clinical symptoms of type 1 diabetes within five years. The study is double-blind and placebo-controlled, which means that half of the children will receive two doses of Diamyd® and half will receive placebo (non-active substance). No-one will know who has received what until the end of the five year study. The intent is to see if the diabetes vaccine can prevent or delay the onset of clinical symptoms of type 1 diabetes in the children. Simply delaying the onset of the disease with the help of Diamyd® would be a major medical success. Those children that develop clinical symptoms of type 1 diabetes during the study will receive injections of active Diamyd® after diagnosis, irrespective of whether they have received active or placebo as preventative treatment. In this manner, it will also be possible to follow the efficacy of the diabetes vaccine in new-onset patients. The study will be much like the ongoing DiAPREV-IT study, except that supplementation of vitamin D is included for all participants and that the latest findings, namely that two early stages of type 1 diabetes exist prior to clinical onset, have been taken into account. The study will stratify the participants according to the stage they belong to at the start of the study. The first stage comprises children with two or more auto-antibodies directed at their own insulin-producing cells, but with normal glucose metabolism. The other stage is children with both auto-antibodies and an impaired glucose metabolism. Vitamin D supplement is given to lower the immune system’s inflammatory components to, thereby, increase the diabetes vaccine’s tolerance-inducing effect with the aim of maintaining the ability to produce insulin.

Latest news:

• • On September 7, 2016, Diamyd Medical provided update on clinical studies with Diamyd® for the treatment and prevention of type 1 diabetes.  The company announced that the placebo-controlled study DiAPREV-IT 2 is not yet fully enrolled.
• • On March 10, 2015, Diamyd Medical announced that DiAPREV-IT 2 has started. In this new study, the second of its kind, the diabetes vaccine Diamyd® is tested to prevent or delay the onset of type 1 diabetes in children at very high risk of presenting with the disease. The first participant out of 80 has now been included in DiAPREV-IT 2, a placebo controlled Phase II study where the diabetes vaccine Diamyd® is administered with the aim of preventing or delaying type 1 diabetes. The children participating in the study are seemingly healthy, but have been identified to have a very high risk of presenting with type 1 diabetes. The participants will also be supplemented with Vitamin D with the aim of strengthening the efficacy of the diabetes vaccine. The autoimmune process causing type 1 diabetes starts before the appearance of any clinical symptoms of the disease. Years or months prior to symptoms of insulin deficiency the immune system has already started to attack the insulin producing cells in the body. During this period there are not yet any noticeable symptoms of the disease but the autoimmune process can be detected by screening for certain markers in the blood. In large screening studies at Lund University, Sweden, children with these markers have been identified and those children will thus most probably present with symptomatic type 1 diabetes. The new study, DiAPREV-IT 2, is a complement to the ongoing study DiAPREV-IT. When the children are included in the study they will be stratified according to which of the early stages of the autoimmune process leading to type 1 diabetes they are in. The first stage comprises children with two or more auto-antibodies directed at their own insulin-producing cells, but with normal glucose metabolism. The second stage comprises children with both auto-antibodies and impaired glucose metabolism. Vitamin D supplement is given to down-regulate the immune system’s inflammatory components in order to increase the diabetes vaccine’s tolerance-inducing effect regarding the preservation of the body’s insulin-producing capacity.
• •  On October 24, 2014, Diamyd Medical informs that a new study is planned for the Diamyd® diabetes vaccine that will comprise 80 children with high risk of presenting with type 1 diabetes. The aim is to test whether the diabetes vaccine can prevent or delay the onset in the children. To increase the possibilities of demonstrating efficacy for Diamyd®, a decision has also been taken to keep the ongoing prevention study DiAPREV-IT intact and blinded until all 50 children involved have been monitored for five years, toward the end of 2016. Thus far, 14 children have been diagnosed with type 1 diabetes in this study, which is fewer than expected. “The planned strategy means that more children who have entered the autoimmune process, but who have not yet been diagnosed with type 1 diabetes, can obtain access to the diabetes vaccine both prior to and following any diagnosis,” says Anders Essen-Möller, Chairman of Diamyd Medical. “This could shorten the path to market acceptance.” In order to increase the scientific value of the research on preventative treatment with the Diamyd® diabetes vaccine, the research team at Lund University, who have been driving the DiAPREV-IT study since 2009, decided to expand the dataset with additional children at high risk of presenting with type 1 diabetes. Therefore, after discussions with the Swedish Medical Products Agency and Diamyd Medical, a new, larger study of a similar design to DiAPREV-IT is being planned, in which new research findings are taken into consideration. “We have discovered that children with a high risk of presenting with type 1 diabetes can be divided into two distinct groups already at screening: those with a normal and those with an impaired glucose metabolism. Furthermore, almost all 14 children that have been diagnosed with type 1 diabetes in the ongoing DiAPREV-IT study belong to the latter group,” says Dr. Helena Elding Larsson, pediatrician in Malmö and researcher at Lund University but also the lead investigator and sponsor for both studies. “This discovery, combined with the fact that 14 out of 50 children was fewer than we had expected to have presented with type 1 diabetes by this stage, means that the scientific value of the ongoing study increases substantially through us keeping the study blind for a further two years. This implies that the statistical base of children who have presented with the disease in the study will increase. By planning a new study with, in principle, the same design, but where the two groups we recently identified are taken into account, we further increase the chance of being able to ascertain any efficacy for the Diamyd® diabetes vaccine.” The new study, named DiAPREV-IT 2, will be much like the ongoing DiAPREV-IT study, except that supplementation of vitamin D is included for all participants and that the latest findings, namely that two early stages of type 1 diabetes exist prior to clinical onset, have been taken into account.

Is general: Yes