Safety and Efficacy of GL-2045 in Patients with Immune Thrombocytopenic Purpura
Research type
Research Study
Full title
Initial Safety and Efficacy, Sequential Group, Adaptive Dose Study of GL-2045 Subcutaneous Injection Repeat Doses in Patients with Immune Thrombocytopenic Purpura
IRAS ID
1013159
Contact name
David Block
Contact email
Sponsor organisation
Gliknik Inc.
Research summary
GL-2045 is a drug that mimics a blood product referred to as pooled human IVIg which is used to treat Immune Thrombocytopenic Purpura (ITP). ITP is a condition where the body attacks its own platelets, leading to low platelet counts and bleeding risk. GL-2045 has been demonstrated to be about 100 - 200-fold more potent compared to IVIg in a variety of laboratory and animal studies. GL-2045 was effective in a murine model of ITP. A previous study in healthy participants found GL-2045 to be generally well tolerated at the same dose to be used here. The goal of this study is to demonstrate GL-2045 safety in adult ITP patients who have been successfully treated with either IVIg or steroids and to show that the drug produces IVIg-like platelet responses in ITP patients. This study will have 2 cohorts. In Cohort 1, 6-18 ITP study participants will be dosed in an adaptive design. Within Cohort 1, the dose frequency may be increased or the dose or dose frequency decreased to assess the lowest effective dose or in the unexpected event of harmful effects. Cohort 2 is a fixed-dose design of 12-24 ITP patients. Within Cohort 2, 1 or 2 dose levels and frequencies will be selected based on the safety and platelet count changes seen in Cohort 1. Dosing will consist of 1 subcutaneous (SC) priming dose on Day 1; progressive dose on Day 2 and multiple SC doses on Days 4 and 6 (3 doses in the first week); and multiple SC doses on Days 8, 11, 15, 18, 22, and 25 (twice weekly over the next 3 weeks). In Cohort 1, participants will be enrolled roughly in groups of 3 with dose and frequency adjustments made between groups based on the ongoing review of the safety, tolerability, and data on how the drug is working. The dosing schedule in the initial group is anticipated to be 75 mg GL-2045 priming dose on Day 1 followed by 225 mg GL-2045 progressive dose on Day 2 and 300 mg GL-2045, subject to adjustment, with each subsequent dose.
REC name
North East - York Research Ethics Committee
REC reference
25/NE/0187
Date of REC Opinion
5 Jan 2026
REC opinion
Further Information Favourable Opinion