FDA lifts clinical hold on trial for novel stem cell-derived therapy for type 1 diabetes
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The FDA has lifted a clinical hold placed on a trial for an investigational stem cell-derived therapy for people with type 1 diabetes, according to an industry press release.
As Healio previously reported, in May the FDA halted the phase 1/2 clinical trial for VX-880 (Vertex Pharmaceuticals), an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy for people with type 1 diabetes with impaired hypoglycemia awareness and severe hypoglycemia. The clinical hold was placed on the trial due to insufficient data to support dose escalation. On July 5, the FDA lifted the clinical hold, allowing the trial to be reopened for screening, enrollment and dosing at multiple sites in the U.S.
The VX-880 phase 1/2 trial is a multicenter, single-arm, open-label study enrolling patients with type 1 diabetes, impaired hypoglycemic awareness and severe hypoglycemia. The study is designed as a three-part trial, with two patients receiving half of the target dose in part A, five patients receiving the target dose in part B, and concurrent dosing at the full dose taking place in part C. Approximately 17 patients will be enrolled in the study.
At the time the trial was halted, three patients have been dosed with VX-880 in the phase 1/2 study. Two patients received half the target dose of cells in part A of the study, whereas a third patient received the full target dose in part B of the study. Part B of the trial will evaluate safety and efficacy in five patients at the target dose before expanding to additional patients in part C.
Researchers presented findings from the two patients in part A of the study at the American Diabetes Association Scientific Sessions. As Healio previously reported, a man aged 64 years with a 40-year duration of type 1 diabetes experiencing impaired hypoglycemia awareness had no detectable fasting and stimulated C-peptide at baseline. After a single VX-880 infusion, fasting C-peptide was detected at day 29 and increased rapidly, with HbA1c and daily insulin decreasing. By day 241 to 270, the patient had a time in range of 99.9% and an HbA1c of 5.2%, down from a baseline HbA1c of 8.6%. The second patient, a woman aged 35 years with type 1 diabetes for 11 years, had an increase in time in range from 35.9% at baseline to 51.9% at day 121 to 150, with a decrease in HbA1c from 7.5% to 7.1%. The treatment was generally safe and well tolerated, with most adverse events being mild or moderate and consistent with immunosuppression.
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