Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Type 1 Diabetes Mellitus

In brief

This is a multicenter, randomized, partial-blinded, five-arm, placebo-controlled study of human plasma-derived alpha1-proteinase inhibitor (alpha1-PI) in children (ages 6-11 years old) and teens/adults (ages 12-35 years old) with new onset Type 1 Diabetes Mellitus (T1DM). Currently enrolling ages 12-35 only. Once 25 patients are randomized and data is reviewed enrollment will be opened to the child cohort. The purpose of this study is to evaluate the safety and efficacy of four dosing regimens of human plasma-derived alpha1-PI in T1DM.

Key facts

Study ID

NCT02093221

Run by

Grifols Therapeutics LLC

People needed

76

Starts

2014-03-01

Expected to finish

2017-06-01

Last updated by the study team

2018-09-05

Who can join

Age: 6 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Diagnosis of T1DM according to the ADA criteria.
• Current use of injected insulin therapy and one positive result on testing for any of the following antibodies (If not currently on insulin therapy, must have positive result for at least two of the below antibodies):
• Anti-islet-cell antibodies (islet cell antigen 512, insulinoma associated protein 2),
• Anti-glutamic acid decarboxylase antibodies, or
• Anti-insulin antibodies (unless received insulin therapy for > 7 days).
• Body Mass Index (BMI) ≤ 28 kg/m2 for adults (≥ 20 years of age) OR ≤ 90th percentile in accordance with the Centers for Disease Control BMI assessment for children and teens (2 through 19 years old).

You may not qualify if…

• History of or current diabetic retinopathy, neuropathy, or nephropathy.
• Known thrombophilia or history of thrombosis.
• Malignant disease (including malignant melanoma; however, other forms of skin cancer are allowed) within five years of randomization.
• Active Hepatitis A virus, Hepatitis B virus, Hepatitis C virus, or Human Immunodeficiency Virus infection.
• History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
• Known selective or severe Immunoglobulin A deficiency.
• Elevated liver enzymes (aspartate transaminase, alanine aminotransferase, and alkaline phosphatase) equal to or greater than 2.5 times the upper limit of normal.
• Therapy with exenatide or any other agents that stimulate pancreatic β cell regeneration or insulin secretion, or any antidiabetic agents (oral or parenteral) other than insulin within one month prior to screening.
• Use of omega-3 fatty acid supplements, including fish oil, within seven days prior to screening.
• Current or planned therapy with inhaled insulin, if it becomes available.
• Chronic use of systemic steroids, with the exception of inhaled steroids, above a stable dose equivalent to 5 mg/day prednisone (e.g., 10 mg every 2 days) within 4 weeks prior to randomization. It is recommended to maintain the same dose throughout the study. (Note: Subjects with autoimmune conditions (i.e., asthma) necessitating treatment with systemic short-term corticosteroids and administered a rapid taper are eligible per protocol with the caveat that the tapering is complete or decreased to the minimum requirement (i.e., 5 mg/day) at least 1 week prior to the Baseline visit (when randomization occurs) to ensure the subject is stable. For longer term steroid usage, please consult the Grifols Medical Monitor before considering the subject for study participation.)
• Treatment with immunosuppressants or cytostatic agents within 6 months of randomization.

Where it is running

• University of Arizona — Tucson, Arizona, United States
• Clinica Medica San Miguel — Los Angeles, California, United States
• Diabetes Associates Medical Group — Orange, California, United States
• Rady Children's Hospital San Diego — San Diego, California, United States
• Metabolic Institute of America — Tarzana, California, United States
• Ronald H Chochinov MD — Ventura, California, United States
• Yale New Haven Hospital — New Haven, Connecticut, United States
• Christiana Care Health Services — Newark, Delaware, United States
• Solutions Through Advanced Research Inc. — Jacksonville, Florida, United States
• CCM Clinical Research — Miami, Florida, United States
• Advanced Pharma CR LLC — Miami, Florida, United States
• Atlanta Diabetes Associates — Atlanta, Georgia, United States
• Rocky Mountain Diabetes and Osteoporosis Center — Idaho Falls, Idaho, United States
• Cook County Hospital — Chicago, Illinois, United States
• Methodist Research Institute — Indianapolis, Indiana, United States
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States
• University of Louisville — Louisville, Kentucky, United States
• Barry J. Reiner MD, LLC. — Baltimore, Maryland, United States
• UMass Memorial Medical Center — Worcester, Massachusetts, United States
• Wayne State University — Detroit, Michigan, United States
• Children's Hospitals and Clinics of Minnesota — Saint Paul, Minnesota, United States
• Morristown Medical Center — Morristown, New Jersey, United States
• University of New Mexico, Health Sciences Center — Albuquerque, New Mexico, United States
• Women and Children's Hospital — Buffalo, New York, United States
• WakeMed Children's Hospital — Raleigh, North Carolina, United States

Full record on ClinicalTrials.gov

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