122 results
Objective Endpoint• To assess the clinical safety and tolerability of leniolisib in pediatric patients (aged 4 to 11 years) with APDS• Incidence of treatment-emergent AEs (TEAEs), SAEs, and AEs leading to discontinuation of study drug• Change from…
The aim of this research is:1. To test the safety of MK-1308A, MK-4280A, MK-7684A and MK 4830 + pembrolizumab against pembrolizumab as the single agent.2. To test how well MK-1308A, MK-4280A, MK-7684A and MK-4830 + pembrolizumab are tolerated by…
-To assess the safety and tolerability of CLDN6 CAR-T +/- CLDN6 RNALPX and to assess the comparability of CLDN6 CAR-T from the manualand automated processes-To identify the maximum tolerated dose (MTD)/RP2D for each IMP (i.e. CLDN6 CAR-T +/- CLDN6…
The objective of the study is to establish an optimal dose of AV-101 based primarily upon the change in PVR but also other efficacy, safety, and tolerability findings from the Phase 2b Part of the study. The optimal dose will be taken into the Phase…
The objectives of this open-label extension safety monitoring (OLE-SM) study are as follows:Part 1 (Open-Label Extension; OLE)• To assess the long-term safety and efficacy of etrolizumab in patients eligible for Part 1 (OLE)Part 2 (Safety Monitoring…
OBJECTIVES - Phase 2 (not to be conducted in NL):Phase 2 Primary Objective:- Identify a setrusumab dosing strategy in subjects with OI Phase 2 Secondary Objectives:- Evaluate the PK of setrusumab doses in subjects with OI- Determine the PD effects…
The sponsor is developing a compound (PB016) similar to Entyvio® (vedolizumab, hereafter referred to as Entyvio). As part of medical-scientific studies to confirm the similarity of the biological products, the sponsor wants to compare PB016 with EU-…
To evaluate the safety and tolerability of crovalimab compared witheculizumab
Primary Objective• Evaluate the efficacy of loncastuximab tesirine combined with rituximab compared to standard immunochemotherapy Secondary Objectives• Further evaluate the efficacy of loncastuximab tesirine combined with rituximab compared to…
To evaluate the efficacy of danicopan as compared to placebo as add-on therapy to a C5 inhibitor at 12 weeks.
Primary objectiveThe primary objective of the study is to determine the long term safety and tolerability of lucerastat in subjects with Fabry disease (FD).Secondary objectives* To evaluate the effect of lucerastat on renal function and cardiac…
The primary objective of this study is to determine whether SAR439859 in combination with palbociclib improves progression free survival (PFS) compared to letrozole in combination with palbociclib in patients with ER +, HER2-advanced breast cancer…
Primary:• To evaluate the safety of ORGN001 (formerly ALXN1101) over the first 6 months of treatmentSecondary:• To characterize the pharmacokinetics (PK) of increasing doses of ORGN001 (formerly ALXN1101) • To evaluate the effect of ORGN001 (…
Primary:To determine the efficacy (as defined by progression-free survival [PFS]) of nirogacestat in adult participants with progressing DT/AF.Secondary:To evaluate the safety and tolerability of nirogacestat in adult participants with progressing…
The purpose of this study is to demonstrate improvement in Disease-Free Survival (DFS) with xevinapant compared to placebo when added to RT irrespective of subsequent anticancer therapy.
The primary objectives of the study are- To evaluate the long-term safety and tolerability of evinacumab 15 mg/kg intravenous (IV) administered every 4 weeks (Q4W) in patients with homozygous familial hypercholesterolemia (HoFH).- To evaluate the…
To compare ACM at Day 42 following treatment with olorofim versus treatment with AmBisome® followed by SOC in the intent-to-treat (ITT) population of patients with IFD caused by proven IA at any site or probable lower respiratory tract disease (LRTD…
• Evaluate antitumor activity based on response assessment criteria (RECIST v1.1)• Determine RP2D (unless determined in the Escalation part)
The primary objective of this study is to assess the efficacy of a single intratympanic injection of AC102 compared to oral steroids in patients with moderately-severe to profound ISSNHL. After treatment of patients, the efficacy will be evaluated…
Primary objectives:# Safety Lead-in Phase: To assess the safety and tolerability, and to establish an RP2D if applicable, of treatment combinations that have not been evaluated in a separate study.# Efficacy Phase: To assess the safety and…