Pre-POINT (Primary Oral/intranasal INsulin Trial) study A dose finding safety and immune efficacy study for primary mucosal insulin therapy in islet autoantibody negative children at high genetic risk for type 1 diabetes - Pre-POINT

• Conditions: pre-type 1 diabetes (islet autoimmunity in non-diabetic relatives of subjects with type 1 diabetes)

• Registration Number: EUCTR2005-001621-29-AT
• Lead Sponsor: Faculty of Medicine Carl Gustav Carus, University of Technology, Dresden

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-05-20
• Last Update Posted: 9 December 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Children aged 2 years to 7 years who:
A. have a multiplex first degree family history of T1DM
(both parents, parent and sib, or two sibs);
and have one of the following HLA genotypes:
DR4- DQA1*0301-DQB1*0302 / DR3- DQA1*0501-DQB1*0201
DR4- DQA1*0301-DQB1*0302 / DR4- DQA1*0301-DQB1*0302
DR4- DQA1*0301-DQB1*0302 / DR4- DQA1*0301-DQB1*0201
DR4- DQA1*0301-DQB1*0302 / DR4- DQA1*0301-DQB1*0304
or
B. have a sibling with T1DM;
and are identical by descent for the HLA DR3/DR4-DQ8 genotype with their
diabetic sibling;
2. Islet autoantibody negative at time of recruitment.

Are the trial subjects under 18? yes
Number of subjects for this age range:
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. Children with any kind of congenital or acquired chronic disease that
potentially interfere with the study objectives.
2. Prior or current participation in another intervention trial.
3. Chronic oral steroid use and/or other chronic oral immunosuppressant

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: The objective of this study is to determine the feasibility, safety and bioavailability of mucosal insulin administration via two alternative routes in children with high genetic risk for type 1 diabetes (T1DM) in a dose escalation primary intervention pilot study.<br><br>To find a dose and route with proven drug bioavailability to the immune system for use in a phase II/III primary T1DM vaccination trial (POINT study) in genetically at risk subjects.;Secondary Objective: ;Primary end point(s): The development of immunity to insulin (IgG or IgA antibodies, or T cell response to insulin and/or insulin peptides).