Israel-based Kamada has started a Phase II/III clinical study of its proprietary human Alpha-1 Antitrypsin (AAT) ‘Glassia’ for the treatment of newly diagnosed paediatric patients with type 1 diabetes (T1D).
In type 1 diabetes, autoimmune attacks occur on pancreatic beta cells that secrete insulin, compromising insulin level and glycemic control.
The company said that over time there is progressive deterioration of self-insulin secretion, poor capability to control glucose levels and eventually, full external insulin dependence.
Around 190 paediatric patients with newly diagnosed T1D will be enrolled in the trial, which is designed to evaluate the safety and efficacy of intravenous Glassia to halt disease progression and maintain the ability of the pancreas to produce insulin.
The two-year trial follows US Food and Drug Administration (FDA) and European Medicines Agency (EMA) guidelines for clinical trials assessing beta cell preservation and will measure C-peptide parameters, HbA1C, hypoglycemic events and insulin daily dose, among others.
Interim data from the trial is expected after 90 patients complete one year of treatment.
The double-blind, placebo-controlled, multicentre trial will initially be conducted at four paediatric T1D medical centres in Israel, with plans to expand the scope of the trial to include centres in other countries.
The company previously reported positive preliminary data from the extension portion of its Phase I/II clinical study of Glassia to treat paediatric patients newly diagnosed with T1D.
Preliminary data from that trial showed that at about 20 months from diagnosis and around ten months following the last Glassia infusion, 60% of subjects who participated in the extension portion of the trial had peak C-peptide levels greater than 0.2pmol/ml.
Kamada co-founder and chief executive officer David Tsur said: "The complications of type 1 diabetes remain an unmet need that is inadequately addressed with existing therapies, which include insulin, diet and behavioral changes.
"Moreover, these complications have direct correlation with the extent of glycemic control the patient reaches during early disease phases.
"We believe Glassia can be a groundbreaking treatment for newly diagnosed type 1 diabetes in pediatric patients as it should demonstrate the ability to halt disease progression and allow the pancreas to produce its own insulin."
According to the company, Glassia is the first available ready-to-use liquid alpha1-proteinase inhibitor (Alpha1-PI) and is indicated as a chronic augmentation and maintenance therapy in adults with alpha-1 antitrypsin (AAT) deficiency.
The company said that Glassia is administered once a week and is augmenting the levels of AAT in the blood.
AAT is a protein derived from human plasma with known and newly discovered therapeutic roles given its immunomodulatory, anti-inflammatory, tissue protective and antimicrobial properties.