14 results
To provide subjects who have completed participation in a Phase 2 or Phase 3 trial continued access to therapy and to evaluate the long-term safety and tolerability of leuco-methylthioninium bis(hydromethanesulfonate) given in flexible doses of up…
Primary objective:• Assess the pharmacokinetics (PK) of rVWF:rFVIII and rVWF, and to assess the safety and efficacy of rVWF:rFVIII and rVWF in the treatment of bleeding events in subjects with severe hereditary VWDSecundary objectives:• Compare the…
• 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…
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…
Primary objective: - To confirm the efficacy of twice-daily applications of delgocitinib cream 20 mg/g compared with cream vehicle in the treatment of adult subjects with moderate to severe CHE. Secondary objective: - To confirm the health-related…
The purpose of this study is to investigate how safe modafinil is and how well it is tolerated when it is administered in combination with aniracetam to generally healthy elderly volunteers with subjective cognitive decline. Modafinil and aniracetam…
Primary objective:To compare the efficacy with belantamab mafodotin vs pomalidomide plus low dose dexamethasone (pom/dex) in participants with relapsed/refractory multiple myeloma (RRMM)Secondary objectives:- To compare the overall survival with…
• Allow patients to continue or start AP01 therapy for the treatment of ILD and IPF prior to regulatory approval or until the study is terminated• To evaluate safety outcomes of patients on AP01 therapy
To determine the maximum tolerated dose (MTD) and Recommended Phase 2 Dose (RP2D) of RLY-4008 and to determine RLY-4008's safety and tolerability.
Primary Objective1. To evaluate the efficacy of IMU-838 versus placebo as measured by quantitative magnetic resonance imaging (MRI) analysis for whole brain atrophy in progressive multiple sclerosis (PMS) patients with the Structural Image…
Primary objective: To establish the PK profile after multiple SC administrations of tralokinumab in children with moderate-to-severe AD.Secondairy objectives: - To assess the safety and tolerability of multiple SC administrations of tralokinumab in…
• 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 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…
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…