Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes.

Promising findings of α-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for β-cell preservation in recent-onset T1D patients. Our prospective, open-label Phase I/II extension study demonstrated that the administration of multiple repeated AAT infusions (up to 36) to AAT-sufficient pediatric T1D patients is safe and well-tolerated. Long-term surveillance of participants (up to 5 years) from diabetes onset revealed normal growth and pubertal progression through adolescence to attainment of full puberty and near adult height.

Alpha-1 antitrypsin therapy is safe and well tolerated in children and adolescents with recent onset type 1 diabetes mellitus.

Background And Objectives: Alpha-1 antitrypsin (AAT) has been shown to reduce pro-inflammatory markers and protect pancreatic islets from autoimmune responses in recent studies. Our aim was to evaluate its safety and tolerability in three different doses, in a pediatric population with recent onset type 1 diabetes mellitus (T1DM).

Methods: A 37-wk prospective, open-label, phase I/II interventional trial, comprised of 24 recently diagnosed subjects (12 males; age 12.