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Primary Objectives:• To assess the safety and tolerability of DCC 3014.• To characterize the pharmacokinetic (PK) profile of DCC 3014.• To determine the maximum tolerated dose (MTD) of DCC 3014.• To determine the recommended Phase 2 dose (RP2D) of…
• Randomized Placebo-Controlled Period:Primary:- to demonstrate the efficacy of mavorixafor in patients with Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) syndrome as assessed by increasing levels of circulating neutrophils…
The purpose of this study is to find out how safe and effective the new drug PHA-022121 is for the treatment of Hereditary Angioedema. The main purpose of this research study is to test the effectiveness of 3 different doses of PHA-022121 in…
Primary Objective:* To evaluate the long-term safety of efgartigimod in adult patients with primary immune thrombocytopenia (ITP).Secondary Objectives:First 52-week treatment period only:* To evaluate the long-term efficacy of efgartigimod on…
Primary Objective: • To evaluate anti-tumor activity of vimseltinib using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 by blinded independent radiological review (IRR)Secondary Objectives:• To assess anti-tumor activity of vimseltinib…
Primary Objective:- To evaluate the efficacy of efgartigimod compared to placebo in achieving a sustained platelet count response in patients with chronic primary immune thrombocytopenia (ITP), with a sustained platelet count response defined as…
The primary objective is to evaluate the efficacy of IFX-1 treatment as a replacement for glucocorticoids [GC] therapy in subjects with GPA and MPA.Secondary objectives:*To assess safety and tolerability of IFX-1*To compare GC-induced toxicity of…
• To evaluate the efficacy of VX-147 to reduce proteinuria• To evaluate the efficacy of VX 147 on renal function as measured by eGFR slope
Primary objective: To evaluate the clinical improvement of efgartigimod PH20 SC treatment compared with placebo, in addition to standard of care immunomodulatory therapy.Key secondary objectives:• To evaluate additional measures of the efficacy of…
Primary ObjectivesPhase 2The primary objectives of Phase 2 are:• To evaluate short- and long-term safety and tolerability of SNDX 5613.• To assess the CR+CRh rate. Secondary ObjectivesPhase 2Secondary objectives of Phase 2 are:• • To assess…
The primary objective of the study is to evaluate the efficacy of dupilumab on the lung function in patients with ABPA.The secondary objectives of the study are:• To evaluate the effects of dupilumab on exacerbations in patients with ABPA • To…