Clinical trial NCT00435981: Efficacy and Safety of Diamyd® in Children and Adolescents With Type 1 Diabetes
Official title: A Phase II, Randomised, Double-Blind, Placebo-Controlled, Multi-Centre Study to Investigate the Impact of Diamyd® on the Progression of Diabetes in Patients Newly Diagnosed With Type 1 Diabetes Mellitus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Type 1 Diabetes
Keywords: Type 1 diabetes
GAD65
Autoimmunity
C-peptide
Children
Lead sponsor: Diamyd Therapeutics AB
Sponsor class: INDUSTRY
First posted: 2007-02-16
Enrollment: 70
Sex: ALL
Minimum age (years): 10
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a randomized, placebo-controlled phase II study to investigate if a prime and boost of 20ug Diamyd® (rhGAD65 formulated in Alhydrogel®), administered subcutaneously four weeks apart, is safe and can preserve beta cell function in children and adolescents with type 1 diabetes with a diabetes duration less than 18 months at intervention.


Eligibility criteria: Key Inclusion Criteria:

* Male and female patients between 10 and 18 years of age, diagnosed with type 1 diabetes within 18 months
* Fasting C-peptide level above 0.1 nmol/l
* Presence of GAD65 antibodies
* Written informed consent (patient and parent/guardian)

Key Exclusion Criteria:

* Secondary diabetes mellitus or MODY
* Treatment with immunosuppressants
* Treatment with any vaccine within 1 month prior to first Diamyd® dose or planned treatment with vaccine up to 2 months after the second Diamyd® dose
* Participation in other clinical trials with a new chemical entity within the previous 3 months
* History of certain diseases or conditions (e.g. epilepsy, anaemia, alcohole or drug abuse)
* HIV or hepatitis
* Presence of associated serious disease or condition which in the opinion of the investigator makes the patient non-eligible for the study
* Pregnancy (or planned pregnancy within one year after 2nd administration)

Primary outcomes:
measure: To evaluate the efficacy of Diamyd® 20ug versus placebo with respect to preserving residual insulin secretion as measured by C-peptide levels. The effect of intervention will be evaluated at month 15 (main study period) and at month 30 (Extension phase).
Secondary outcomes:
measure: To evaluate the safety of Diamyd® 20ug.
Other outcomes:
Locations:
city: Borås
country: Sweden
facility: Borås Hospital
city: Göteborg
country: Sweden
facility: The Queen Silvia Children´s Hospital
city: Halmstad
country: Sweden
facility: Halmstad Hospital
city: Jönköping
country: Sweden
facility: Ryhov Hospital
city: Linköping
country: Sweden
facility: Linköping University
city: Malmö
country: Sweden
facility: University Hospital, MAS
city: Stockholm
country: Sweden
facility: Astrid Lindgren Children´s Hospital
city: Örebro
country: Sweden
facility: Örebro University Hospital
Study officials:
affiliation: Div of Pediatrics, Dept of Clinical and Molecular Medicine, Faculty of Health Sciences, Linkoping University, Sweden
name: Johnny Ludvigsson, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ludvigsson J, Cheramy M, Axelsson S, Pihl M, Akerman L, Casas R; Clinical GAD-Study Group in Sweden. GAD-treatment of children and adolescents with recent-onset type 1 diabetes preserves residual insulin secretion after 30 months. Diabetes Metab Res Rev. 2014 Jul;30(5):405-14. doi: 10.1002/dmrr.2503.
citation: Ludvigsson J, Hjorth M, Cheramy M, Axelsson S, Pihl M, Forsander G, Nilsson NO, Samuelsson BO, Wood T, Aman J, Ortqvist E, Casas R. Extended evaluation of the safety and efficacy of GAD treatment of children and adolescents with recent-onset type 1 diabetes: a randomised controlled trial. Diabetologia. 2011 Mar;54(3):634-40. doi: 10.1007/s00125-010-1988-1. Epub 2010 Nov 30.
citation: Ludvigsson J, Faresjo M, Hjorth M, Axelsson S, Cheramy M, Pihl M, Vaarala O, Forsander G, Ivarsson S, Johansson C, Lindh A, Nilsson NO, Aman J, Ortqvist E, Zerhouni P, Casas R. GAD treatment and insulin secretion in recent-onset type 1 diabetes. N Engl J Med. 2008 Oct 30;359(18):1909-20. doi: 10.1056/NEJMoa0804328. Epub 2008 Oct 8.