A clincial study evaluating the safety of the diabetes vaccine Diamyd® in combination with Vitamin D, and if it can delay or stop the process leading to type 1 diabetes in children at high risk of developing the disease

Phase 1

• Conditions: Children with HLA risk and persistent islet autoantibody positivity which is associated with a defined risk for type 1 diabetes; Therapeutic area: Body processes [G] - Immune system processes [G12]

• Registration Number: EUCTR2014-003755-64-SE
• Lead Sponsor: Helena Elding Larsson

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-09-15
• Last Update Posted: 4 September 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

1.Children from four (4) to 17,99 years of age.
2.Positive GAD65Ab and at least one additional type 1 diabetes-associated autoantibody (IA-2Ab, ZnT8R/W/Q/AAb or IAA).
3.Written informed consent from the child and the child’s parents or legal acceptable representative(s) according to local regulations.

Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.Ongoing treatment with immunosuppressant therapy (topical or inhaled steroids are accepted).
2.Diabetes.
3.Treatment with any oral or injected anti-diabetic medications.
4.Significantly abnormal hematology results at screening.
5.Clinically significant history of acute reaction to vaccines or other drugs.
6.Treatment with any vaccine, other than influenza, within one month prior to the first dose of the study drug or planned treatment with vaccine up to two months after the last injection with the study drug.
7.A history of epilepsy, serious head trauma or cerebrovascular accident, or clinical features of continuous motor unit activity in proximal muscles.
8.Participation in other clinical trials with a new chemical entity within the previous 3 months.
9.History of hypercalcemia.
10.Unwilling to abstain from other medication with Vitamin D during the study period.
11.Significant illness other than diabetes within 2 weeks prior to first dosing.
12.Known human deficiency virus (HIV) or hepatitis.
13.Presence of associated serious disease or condition, including active skin infections that preclude subcutaneous injection, which in the opinion of the investigators makes the patient non-eligible for the study.
14.Diabetes-protective HLA-DQ6-genotype.
15.Females who are lactating or pregnant (for females who have started menstruating the possibility of pregnancy must be excluded by urine ßHCG onsite within 24 hours prior to the study drug administration)
16.Males or females not willing to use adequate contraception, if sexually active, until 1 year after the last Diamyd administration. Adequate contraception is as follows:

For females of childbearing potential:

a.oral (except low-dose gestagen (lynestrenol and norestisteron)), injectable, or implanted hormonal contraceptives (females)
b.intrauterine device (females)
c.intrauterine system (for example, progestin-releasing coil) (females)
d.vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)

For males of childbearing potential:

a.condom (male)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to evaluate if Diamyd®, in children treated with relatively high dose vitamin D, may delay or stop the autoimmune process leading to clinical type 1 diabetes in children with ongoing persistent beta cell autoimmunity as indicated by multiple positive islet cell autoantibodies.<br>;Secondary Objective: The secondary objective is to demonstrate that Diamyd® is safe in children at risk for type 1 diabetes. ;Primary end point(s): The proportion of subjects diagnosed with clinical type 1 diabetes in the Diamyd® treated group, compared to the placebo treated group at five years after the first injection. ;Timepoint(s) of evaluation of this end point: At 5 years