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
Overview
- Phase
- Phase 1
- Intervention
- NBI-6024
- Conditions
- Type 1 Diabetes Mellitus
- Sponsor
- Neurocrine Biosciences
- Enrollment
- 188
- Locations
- 21
- Primary Endpoint
- 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
- Status
- Completed
- Last Updated
- 17 years ago
Overview
Brief Summary
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.
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.
Investigators
Eligibility Criteria
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
Exclusion Criteria
- •Use of an excluded medication/therapy including any of the following:
- •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
Arms & Interventions
1 Experimental
NBI-6024 0.1 mg
Intervention: NBI-6024
2 Experimental
NBI-6024 0.5 mg
Intervention: NBI-6024
3 Experimental
NBI-6024 1 mg
Intervention: NBI-6024
Outcomes
Primary Outcomes
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)
Secondary Outcomes
- 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(monthly assessments, up to 24 months (end of study))