Date: 2014-03-05
Type of information: Initiation of the trial
phase: 2-3
Announcement: initiation of the trial
Company: Kamada (Israel)
Product: Glassia® (alpha-1 antitrypsin)
Action
mechanism: Glassia® (Alpha1-Proteinase Inhibitor -Human) 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. Glassia is administered once a week to augment the levels of AAT in the blood to normal values. 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. Glassia® is approved by the FDA for the treatment of AAT deficiency. It is marketed through a strategic partnership with Baxter International Inc. in the United States. The scientific rationale for Glassia® to treat type 1 diabetes is based on the fact that AAT has an adjunct anti-inflammatory activity that may modulate the immune system in a way that prevents it from attacking the pancreatic beta cells that would be destroyed by the autoimmune attack. Past studies have shown that despite having a normal serum level of AAT, the AAT of diabetic patients is inactive in this respect and, therefore, unable to cope with the developing inflammation in the beta cells. Additionally, a number of recent studies support the rationale for treating type 1 diabetes early in the disease diagnosis or the “honeymoon” period, a period during which there are still some existing functional beta cells. It is hypothesized that Glassia may halt pancreatic inflammation, thereby allowing the survival of active and operating beta cells that secrete insulin, a survival which may allow the patient to reduce dependence on external insulin and eventually decrease disease complications.
Disease: type 1 diabetes
Therapeutic area: Metabolic diseases
Country: Israel
Trial details:
Latest
news: * On March 5, 2014, Kamada, a plasma-derived protein therapeutics company focused on orphan indications, announced the initiation a Phase 2/3 clinical trial of Glassia®, the Company’s proprietary human Alpha-1 Antitrypsin (AAT), to treat newly diagnosed pediatric patients with type 1 diabetes (T1D). In T1D, autoimmune attacks occur on pancreatic beta cells that secrete insulin, thereby compromising insulin level and glycemic control. Over time there is progressive deterioration of self-insulin secretion, poor capability to control glucose levels and, eventually, full external insulin dependence. This double-blind, placebo-controlled, multicenter Phase 2/3 clinical trial of 190 pediatric patients with newly diagnosed type 1 diabetes will evaluate the safety and efficacy of intravenous Glassia® to halt disease progression and maintain the ability of the pancreas to produce insulin. By maintaining its ability to produce insulin, the body can independently control glucose level and avoid diabetes complications that result from poor glycemic control (e.g., cardiovascular disease, kidney disease, eye and vision problems, neurological damage and more). This two-year study follows U.S. Food and Drug Administration and European Medicines Agency guidelines for clinical trials evaluating beta cell preservation and will measure C-peptide parameters (which represent self-insulin secretion), HbA1C, hypoglycemic events and insulin daily dose, among others. Interim data are expected after approximately 90 patients complete one year of treatment, which will be in approximately two years. Initially, the trial will be conducted at four leading pediatric type 1 diabetes medical centers in Israel, with plans to expand the scope of the trial to include centers in other countries. Kamada previously reported positive preliminary data from the extension portion of its Phase 1/2 clinical trial of Glassia to treat pediatric patients newly diagnosed with type 1 diabetes. That preliminary data showed that at approximately 20 months from diagnosis and approximately 10 months following the last Glassia infusion, 60% of study subjects who participated in the extension portion of the trial had peak C-peptide levels greater than 0.2 pmol/ml, which indicates a functioning beta cell capacity and is considered to be a higher percentage than would be expected without intervention. In addition, patients continued to attain HbA1C targets according to International Society for Pediatric and Adolescent Diabetes, with an average HbA1C of 7.5%, and 75% of patients presented HbA1C levels even lower than 7.5%, which is the clinically desired level for glycemic control in pediatric diabetic patients, who usually demonstrate a more severe or volatile form of disease compared with adults.
