An Adaptive Dose Escalation and Expansion Basket Trial to Explore the Safety, Pharmacology, and Clinical Activity of TGD001 in Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) and Other Thrombotic Microangiopathies

Condition studied

Immune-Mediated Thrombotic Thrombocytopenic Purpura, Thrombotic microangiopathy

Eligibility

Primary endpoint

Treatment emergent adverse events

Endpoint detail

Time to clinical response, Change in platelet count from baseline, Time to recovery of platelets to ≥150×10E9/L, Change of organ damage markers from baseline, Time to recovery of LDH to ≤2 × ULN, Assessment or improvement (improvement of ≥1 grade in CTCAE v5.0 scale) of disease-related signs and symptoms within 90 days post intervention (by number of unique participants and by total number of AEs), Number of days in the intensive care unit and the number of days hospitalized, All-cause and disease-specific mortality within 90 days post-intervention, Plasma concentrations of TGD001, PK parameters such as Cmax, Tmax, AUC, t1/2, Development of ADA