A Study to Evaluate NBI-6024 in Adult and Adolescent Patients With New Onset of Type 1 Diabetes Mellitus

March 30, 2009 updated by: Neurocrine Biosciences

A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Parallel, Dose-Ranging Study to Evaluate The Efficacy, Safety, Tolerability, and Pharmacodynamics of NBI-6024 In Adult and Adolescent Patients With New Onset Type 1 Diabetes Mellitus

This was a study designed to evaluate the efficacy of multiple doses of an investigational drug, NBI-6024, in adult (18 to 35 years of age) and adolescent (10 to 17 years of age) patients with new onset type 1 diabetes mellitus, on endogenous insulin production.

A total of 188 patients were enrolled in the study. The study was divided into three periods: screening, treatment (comprising an induction phase and maintenance phase), and follow-up.

NBI-6024 was generally well tolerated and exhibits a benign safety profile, as there were no significant safety issues with NBI-6024 treatment. In summary, NBI-6024 did not demonstrate statistically significant efficacy compared with placebo.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This was a Phase II, multicenter (international), randomized, double-blind, placebo-controlled, parallel, dose-ranging study to evaluate the efficacy of multiple doses of an altered peptide ligand, NBI-6024, in adult (18 to 35 years of age) and adolescent (10 to 17 years of age) patients with new onset type 1 diabetes mellitus.

Study drug was administered subcutaneously a total of 26 times over a 24-month period. The first three doses were administered every 2 weeks (induction phase); all subsequent dosing occurred monthly (maintenance phase). Patients returned to the study center to receive study drug and have efficacy and safety assessments collected. The primary efficacy endpoint was the 2-hour peak C-peptide at Month 24. Other secondary analyses included AUC C-peptide, prescribed insulin usage, AUC blood glucose, HbA1c, hypoglycemic events, and hyperglycemic events.

Study Type

Interventional

Enrollment (Actual)

188

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
      • Laval, Canada
        • Centre de Recherche Clinique de Laval
    • Ontario
      • Ottawa, Ontario, Canada
        • Children'S Hospital Of Eastern Ontario
    • Quebec
      • Sherbrooke, Quebec, Canada
        • Center Hospitalier Universitaire de Sherbrooke
  • Czech Republic
      • Olomouc, Czech Republic
        • University Hospital and School of Medicine
      • Prague, Czech Republic
        • Faculty Hospital Motol
  • Finland
      • Helsinki, Finland
        • Helsinki University Hospital
  • France
      • Lyons, France
        • Hopital Debrousse
      • Paris, France
        • Hôpital St Vincent de Paul
  • Germany
      • Hannover, Germany
        • Diabetes Center for Children and Adolescents
      • Munchen, Germany
        • Institut für Diabetesforschung
  • South Africa
      • Cape Town, South Africa
        • New Groote Schuur Hospital
      • Durban, South Africa
        • Parklands Medical Center
      • Johannesburg, South Africa
        • Center for Diabetes and Endocrinology
      • Johannesburg, South Africa
        • Donald Gordon Medical Center
      • KwaZulu Natal, South Africa
        • Medigate Medical Center
      • Somerset West, South Africa
        • Helderberg Diabetic Clinic and Practice
  • Spain
      • Barcelona, Spain
        • Hospital Clínic de Barcelona
      • Cruces-Baracado, Spain
        • Hospital de Cruces
      • Malaga, Spain
        • Hospital Materno-Infantil
      • Sevilla, Spain
        • University Hospital Virgen del Rocio
  • United Kingdom
      • Dundee, United Kingdom
        • Maternal and Child Health Services 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years to 35 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Male or female between the age of 12 and 35 years, inclusive (changed to between the age of 10 and 35 years, inclusive, under Amendment 2)
  • If female of childbearing potential, patient must use an acceptable method of birth control prior to and for 30 days post study
  • Adult (greater than or equal to 18 years) female patients who were not of childbearing potential must be 2 years postmenopausal, or have had a hysterectomy or tubal ligation
  • Were newly diagnosed with type 1 diabetes mellitus

  • Presence of one or more of the following:

    • Anti-ICA512 antibodies
    • Anti-GAD antibodies
    • Anti-insulin antibodies, provided that the patient was not on insulin therapy for greater than 1 week
  • Body mass index (BMI) < 28 kg/m2
  • Stimulated serum C-peptide peak level between 0.4 pmol/mL and 3.0 pmol/mL, inclusive, at the time of screening
  • Laboratory and 12-lead electrocardiogram (ECG) results within normal ranges or, if abnormal, considered by the investigator as non clinically significant for the safety and well being of the patient or for the purposes of the study

Exclusion Criteria:

  • Use of an excluded medication/therapy including any of the following:

    • Steroids
    • Oral hypoglycemic agents
    • Chemotherapy and radiation
    • Immunosupressants
    • Nicotinamide >100 mg per day
    • Any drugs containing sibutramine
  • Female patients with a positive pregnancy test or who are lactating
  • Adult patients with body weight <45 kg; adolescent patients with body weight <30 kg; 10- and 11-year-old patients with body weight <25 kg
  • History of cancer or have existing or actively managed cancer
  • History of severe or anaphylactic allergic reactions
  • Patients suffering from active skin infections that would prevent subcutaneous injection
  • Positive test for HIV antigens, hepatitis B surface antigen, or hepatitis C antibodies
  • History of alcohol or substance abuse

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: 1 Experimental
NBI-6024 0.1 mg
Drug: NBI-6024
0.1, 0.5 or 1 mg NBI-6024 First 3 doses every 2 weeks. Remaining doses given monthly. Total duration of dosing 24 months. Placebo controlled.
Active Comparator: 2 Experimental
NBI-6024 0.5 mg
Drug: NBI-6024
0.1, 0.5 or 1 mg NBI-6024 First 3 doses every 2 weeks. Remaining doses given monthly. Total duration of dosing 24 months. Placebo controlled.
Active Comparator: 3 Experimental
NBI-6024 1 mg
Drug: NBI-6024
0.1, 0.5 or 1 mg NBI-6024 First 3 doses every 2 weeks. Remaining doses given monthly. Total duration of dosing 24 months. Placebo controlled.
No Intervention: 4 placebo
Placebo injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To assess the effect of repeated administrations of NBI-6024 on endogenous insulin production as measured by C-peptide levels in adult and adolescent patients with new onset type 1 diabetes mellitus
Time Frame: monthly assessments, up to 24 months (end of study)
monthly assessments, up to 24 months (end of study)

Secondary Outcome Measures

Outcome Measure
Time Frame
To examine the effects of repeated administrations of NBI-6024 on insulin usage, glycemic control, and immune function (immunodynamics and pharmacodynamics) To examine the safety and tolerability of repeated administrations of NBI-6024
Time Frame: monthly assessments, up to 24 months (end of study)
monthly assessments, up to 24 months (end of study)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Investigators

  • Principal Investigator: Areti Philotheou, MD, New Groote Schuur Hospital, Capetown, South Africa

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2001

Primary Completion (Actual)

April 1, 2006

Study Completion (Actual)

July 1, 2006

Study Registration Dates

First Submitted

March 27, 2009

First Submitted That Met QC Criteria

March 30, 2009

First Posted (Estimate)

April 1, 2009

Study Record Updates

Last Update Posted (Estimate)

April 1, 2009

Last Update Submitted That Met QC Criteria

March 30, 2009

Last Verified

March 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NBI 6024-0101

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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