Atorvastatin in New Onset Type 1 Diabetes Mellitus (T1DM)

NCT ID: NCT00529191

Last Updated: 2017-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-07-31
• Study Completion Date: 2013-07-31

Brief Summary

The goal of this application is to evaluate the safety and efficacy of atorvastatin as a potential treatment to preserve beta cell function in children and young adults with newly diagnosed type 1 diabetes (T1DM).

Detailed Description

Type 1 diabetes is an autoimmune disease that is characterized by destruction of the insulin-producing beta cells of the pancreas. T1DM therapy requires insulin administration, either by multiple daily injections or by insulin pump. However, in many patients, blood sugar control remains suboptimal and complications develop that shorten life expectancy and severely impact quality of life. At the time of diagnosis, most patients still have significant residual beta cell function. Previous research has shown that weakening the immune system's attack on the pancreatic beta cells may help to preserve or potentially increase insulin production.

Preliminary studies have shown that members of the statin family of medications, including atorvastatin (Lipitor®), preserve beta cell function in a mouse model of type 1 diabetes. These finding suggest that use of atorvastatin in combination with insulin therapy may delay and potentially reverse the destruction of beta cells in patients who have recently developed type 1 diabetes. Atorvastatin (Lipitor®) is approved for use in adults and children (>10 years of age) who have elevated blood cholesterol levels. This study will examine whether atorvastatin (Lipitor®) may also help the body preserve insulin production in patients with newly diagnosed (within 8 weeks) type 1 diabetes.

Patients will be randomly assigned to take either atorvastatin (Lipitor®) or placebo. Two out of every 3 patients will receive atorvastatin and 1 out of 3 will get placebo. As this is a double-blinded study, neither the care team nor the patient will know if they are actually taking atorvastatin (Lipitor®). Patients who have given consent to participate in the study and pass the required screening tests will take the assigned treatment every day for 12 months. All patients will begin taking 10 mg once daily, the recommended starting dose. After 4 weeks, the dose will be increased to 20 mg. In addition to a high standard of diabetes care and the medication, patients will have blood tests during 7 visits over an 18 month period.

Conditions

Type 1 Diabetes

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Atorvastatin

Two out of every three patients will receive atorvastatin.

Group Type: EXPERIMENTAL

Atorvastatin

Intervention Type: DRUG

Pill, initially at 10 mg, then after 4 weeks, 20 mg Once daily for a total of 12 months

Placebo

One out of three subjects will receive a placebo.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

One out of three subjects will receive a placebo.

Interventions

Atorvastatin

Pill, initially at 10 mg, then after 4 weeks, 20 mg Once daily for a total of 12 months

Intervention Type: DRUG

Placebo

One out of three subjects will receive a placebo.

Intervention Type: OTHER

Other Intervention Names

Lipitor

Eligibility Criteria

Inclusion Criteria

• Individuals 10-19 years of age (Tanner Stage II or greater),
• The presence of one or more serum antibodies to islet cell proteins (anti- glutamic acid decarboxylase [GAD], islet antigen 2 or insulin autoantibodies) as assessed in standard practice,
• Diagnosis of T1DM within the 8 weeks prior to study entry
• Peak stimulated C-peptide level >0.2pmol/mL following mixed meal tolerance test (MMTT) performed at least 3 weeks after diagnosis,
• Females of reproductive potential must not plan on conceiving a child during the treatment program, and agree to use a medically accepted form of contraception

Exclusion Criteria

• Subjects currently receiving cyclosporine, fibric acid derivatives, niacin (nicotinic acid), erythromycin, clarythromycin, nefazodone, itraconazole, ketoconazole or protease inhibitors,
• Pregnancy or breast-feeding,
• Clinical AIDS, AIDS related syndrome (ARS) or known positive HIV serology,
• Subjects treated with immunosuppressive therapy in the past 12 months,
• Subjects receiving glucocorticoid therapy or therapy other than insulin that is likely to affect glucose homeostasis (such as sulfonylureas, thiazolidinediones, metformin or amylin),
• Subjects with other autoimmune diseases, except autoimmune thyroid disease,
• Subjects with any illness that might complicate diabetes management or preclude treatment with atorvastatin,
• Transplant recipients,
• Evidence of liver dysfunction or myopathy

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

FDA Office of Orphan Products Development

FED

Sponsor Role: collaborator

Medical University of South Carolina

OTHER

Sponsor Role: collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Steven M Willi, M.D

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

Diabetes Center for Children & Clinical Translational Research Center

Philadelphia, Pennsylvania, United States

Countries

United States

Other Identifiers

R01FD003340

Identifier Type: FDA

Identifier Source: secondary_id

View Link

2006-5-4824

Identifier Type: -

Identifier Source: org_study_id