NCT01122446

Brief Summary

A double-blind, randomized investigator-initiated study to determine the safety and the effect of Diamyd® on the progression to type 1 diabetes in children with multiple islet cell autoantibodies Eligible children are 4 years or older, have positive GAD-antibodies and at least one additional autoantibody and not yet diabetes. Objectives: DiAPREV-IT is the first prevention study with Diamyd®, where the drug is given before onset of type 1 diabetes. The primary objective is to demonstrate that Diamyd® is safe in children at risk for type 1 diabetes. The secondary objective is to evaluate if Diamyd® 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2009

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2009

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

May 12, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 13, 2010

Completed
6.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
2.4 years until next milestone

Results Posted

Study results publicly available

May 6, 2019

Completed
Last Updated

May 6, 2019

Status Verified

May 1, 2019

Enrollment Period

7.7 years

First QC Date

May 12, 2010

Results QC Date

August 8, 2018

Last Update Submit

May 2, 2019

Conditions

PrediabetesType 1 Diabetes

Keywords

Alum-GADType 1 diabetespreventionimmune toleranceglucose toleranceglutamate decarboxylase autoantibodies

Outcome Measures

Primary Outcomes (1)

  • Adverse Events

    Adverse events, serious adverse events, hematology, chemistry, autoantibody titles by treatment group

    During 5 years follow up from treatment

Secondary Outcomes (9)

  • Number of Participants With Type 1 Diabetes

    During 5 years follow up from treatment

  • Fasting Glucose Over Time

    During 5 year follow-up from treatment

  • 120 Minutes Glucose From OGTT Over Time

    During 5 year follow-up from treatment

  • AUC Glucose From OGTT Over Time

    During 5 year follow-up from treatment

  • Fasting C-peptide Over Time

    During 5 year follow-up from treatment

  • +4 more secondary outcomes

Study Arms (2)

Placebo comparator

PLACEBO COMPARATOR

Two doses of placebo day 1 and 30

Other: Placebo comparator

Alum-GAD (Diamyd)

ACTIVE COMPARATOR

20 microgram Diamyd day 1 and 30

Drug: Diamyd

Interventions

Placebo comparatorOTHER

Placebo comparator day 1 and 30 in non-diabetic children with multiple islet autoantibodies. Post diagnosis: Two doses of 20 microgram Diamyd day 1 and 30 in children originally receiving placebo.

Placebo comparator
DiamydDRUG

20 microgram day 1 and 30 in non-diabetic children with multiple islet autoantibodies. Post diagnosis: Two doses of Diamyd followed to children originally receiving Diamyd

Also known as: Alum-GAD
Alum-GAD (Diamyd)

Eligibility Criteria

Age4 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Children from four (4) years of age and participating in DiPiS, TEDDY or Trial Net.
  • Positive GAD65Ab and at least one additional type 1 diabetes-associated autoantibody (IA-2Ab, ZnT8R/W/QAb or IAA).
  • Written informed consent from the child and the child's parents or legal acceptable representative(s) according to local regulations.

You may not qualify if:

  • Ongoing treatment with immunosuppressant therapy (topical or inhaled steroids are accepted).
  • Diabetes.
  • Treatment with any oral or injected anti-diabetic medications.
  • Significantly abnormal hematology results at screening.
  • Clinically significant history of acute reaction to vaccines or other drugs.
  • 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.
  • A history of epilepsy, serious head trauma or cerebrovascular accident, or clinical features of continuous motor unit activity in proximal muscles.
  • Participation in other clinical trials with a new chemical entity within the previous 3 months.
  • Significant illness other than diabetes within 2 weeks prior to first dosing.
  • Known human deficiency virus (HIV) or hepatitis.
  • 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.
  • Diabetes-protective HLA-DQ6-genotype.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Research Center, Pediatric Endocrinology, Jan Waldenströms gata 35, 60:11

Malmo, 205 02, Sweden

Location

Related Publications (3)

  • Andersson C, Carlsson A, Cilio C, Cedervall E, Ivarsson SA, Jonsdottir B, Jonsson B, Larsson K, Neiderud J, Lernmark A, Elding Larsson H; DiAPREV-IT Study Group. Glucose tolerance and beta-cell function in islet autoantibody-positive children recruited to a secondary prevention study. Pediatr Diabetes. 2013 Aug;14(5):341-9. doi: 10.1111/pedi.12023. Epub 2013 Mar 8.

    PMID: 23469940RESULT

  • Elding Larsson H, Larsson C, Lernmark A; DiAPREV-IT study group. Baseline heterogeneity in glucose metabolism marks the risk for type 1 diabetes and complicates secondary prevention. Acta Diabetol. 2015 Jun;52(3):473-81. doi: 10.1007/s00592-014-0680-1. Epub 2014 Nov 8.

    PMID: 25381193RESULT

  • Elding Larsson H, Lundgren M, Jonsdottir B, Cuthbertson D, Krischer J; DiAPREV-IT Study Group. Safety and efficacy of autoantigen-specific therapy with 2 doses of alum-formulated glutamate decarboxylase in children with multiple islet autoantibodies and risk for type 1 diabetes: A randomized clinical trial. Pediatr Diabetes. 2018 May;19(3):410-419. doi: 10.1111/pedi.12611. Epub 2017 Nov 24.

    PMID: 29171140RESULT

MeSH Terms

Conditions

Prediabetic StateDiabetes Mellitus, Type 1

Condition Hierarchy (Ancestors)

Diabetes MellitusGlucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System Diseases

Limitations and Caveats

Limitations: This study was a first study of Alum-GAD in non-diabetic children with multiple islet autoantibodies. The study was small in sample size which may limit the power.

Results Point of Contact

Title
Helena Elding Larsson, PI
Organization
LundU
helena.elding_larsson@med.lu.se
+4640337676

Study Officials

  • Helena Elding Larsson, MD, PhD

    Region Skåne and Lund University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD PhD

Study Record Dates

First Submitted

May 12, 2010

First Posted

May 13, 2010

Study Start

April 1, 2009

Primary Completion

December 1, 2016

Study Completion

December 1, 2016

Last Updated

May 6, 2019

Results First Posted

May 6, 2019

Record last verified: 2019-05

Locations

SE(1)