17 results
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
The purpose of this study is to investigate how quickly and to what extent different doses of EUapproved Tysabri® are absorbed and eliminated from the body (this is called pharmacokinetics) and how the body responds to different doses of Tysabri (…
Primary Objective: Compare the uptake of 68Ga-NODAGA-exendin-4 in the liver between T1D patients with functional islet grafts (C-peptide > 0.8 nmol/L after mixed meal stimulation test and relevant laboratory parameters as HbA1C and exogenous…
Primary ObjectiveThe primary objective of this study is to evaluate glycogen clearance in muscle tissue samples collected pre and post alglucosidase alfa treatment in patients with late-onset Pompe disease.Secondary ObjectivesThe secondary…
The primary objective is to correlate ex vivo 111In-exendin tracer accumulation in the pancreas of patients undergoing pancreatectomy for pancreatic cancer or chronic pancreatitis to establish the relation between tracer uptake and beta cell mass in…
The primary objectives of this study are:• To determine whether ART-123, when administered to subjects with infection complicated by at least one organ dysfunction and coagulopathy, can reduce mortality.• To determine the safety of ART-123 in this…
1. To evaluate the safety and tolerability of ascending multiple oral doses of HTL18318 in healthy adult male and female subjects.2. To evaluate the pharmacokinetics of ascending multiple oral doses of HTL18318 in healthy adult male and female…
Main objectives: safety and efficacy of BAY94-9027 for prophylaxis and treatment of bleeding in PTP with hemophilia ASecondary objective: PK
To investigate how quickly and to what extent diclofenac is absorbed and eliminated from the body (this is called pharmacokinetics) when it is administrated by using a HP 5000 transdermal patch. Furthermore, the pharmacokinetics of HP-5000…
To assess safety, tolerability and pharmacokinetics of a single subcutaneous administration of HPV-NIRD1.
Primary Objective: The primary objective is to determine whether the administration of Gelofusine will reduce the kidney uptake of 111In-labeled exendin in humans by enhancing the excretion of 111In-labeled exendin. These highly relevant data can…
The primary objectives of this study is:* To determine the safety and tolerability of rhHNS via ascending doses administered via a surgically implanted intrathecal drug device (IDDD) once monthly for 6 months, in patientswith MPS IIIA.The secondary…
Primary Objective: To determine whether topical application of Calcitriol (Silkis) 3 µg/g, Diclofenac 10% or a combination of both can lead to a 40% histological reduction (*)/increase (*) of expression of the following antibodies: Ki67 (*), BCL2…
The objective of this study is to assess the efficacy and safety of adalimumab in combination with a topical treatment for the treatment of patients with moderate to severe chronic plaque psoriasis.
To assess the safety and tolerability of escalating single IV doses of NI-0801.To determine the pharmacokinetics and pharmacodynamics parameters of escalating single IV doses of NI-0801.
PRIMARY OBJECTIVES Part I - To evaluate the efficacy (as measured by PFS) of GDC-0941 340 mg +carboplatin * paclitaxel (Arm A) versus carboplatin * paclitaxel (Arm B) in all patients with squamous NSCLC -To evaluate the efficacy (as measured by PFS…
The co-primary objectives of this study are: * To evaluate the efficacy of as measured by investigator-assessed PFS according to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). * To evaluate the efficacy of atezolizumab as…