1926 results
To characterize the natural history of exocrine pancreatic function as measured by FE-1 in infants with CF <12 months of age
Identification of type 2 diabetes susceptibility genes in the Duch population.
The primary objectiveTo evaluate the efficacy of eculizumab in the treatment of pediatric refractory generalized myasthenia gravis (gMG) based on change from Baseline in the Quantitative Myasthenia Gravis score for disease severity (QMG).The…
Primary objective: To evaluate the safety, tolerability, and maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of investigational therapies in subjects with metastatic castration-resistant prostate cancer (mCRPC).Secondary objective:*…
Primary-To evaluate the safety and tolerability of a single dose of EDIT-101 when administered to participants with LCA10-IVS26 mutationSecondary-To evaluate different doses of EDIT-101 for subsequent clinical evaluation-To evaluate the efficacy of…
Primary Objective:To determine if the time in the desired glycemic range is equivalent by following the insulin infusion rate advice from the Glycostat algorithm compared to historical data in the ICU. The historical data for glycemic control…
Primary Objectives:• To determine the safety of ELA026 administered IV and SC to participants with sHLH.• To identify the RP3D and schedule for ELA026.Secondary Objectives:• To characterize the pharmacokinetic (PK) profile of ELA026 administered IV…
This study will evaluate the efficacy and safety of multiple therapies that are selected using somatic alterations and potential predictive biomarkers identified via NGS assays in patients with solid tumors:
Safety Run-in (SRI) part:To confirm the recommended phase 2 dose (RP2D) of NIS793 or any other investigational drug(s), in combination with SOC anti-cancer therapy. In protocol amendment 1, an arm with tislelizumab has been added. In this arm, the…
The primary objective of the study is to evaluate the progression free survival of patients when these are treated with ramucirumab and cyclophosphamide and vinorelbine, in comparison to treatment without ramucirumab.The secondary objectives of the…
Primary - To evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.Secondary - To further evaluate the efficacy of parsaclisib in the treatment of participants with wAIHA.
Primary ObjectiveThe primary objective of this study is to examine the feasibility of using the clubfoot brace. This will be done by recording the effect of the brace on the clubfoot deformity by determining the improvement of the Dimeglio and…
The primary objective of this study is to assess the efficacy of IgPro20 0.5 g/kg weekly subcutaneous (SC) doses in comparison to placebo in adult subjects with DM, as measured by responder status based on the Total Improvement Score (TIS)…
The aim of this study is to assess the safety and the potential efficacy of personalized Local Field Potential (LFP)-based adaptive Deep Brain Stimulation (aDBS), using the implantable pulse generator (IPG), «AlphaDBS» System, in Parkinson*s Disease…
Primary objective:To evaluate the efficacy of ION-682884, based on the change from Baseline in serum Transthyretin (TTR) concentration, modified Neuropathy Impairment Score +7 (mNIS+7), and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (…
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 Objectiveo evaluate the safety and tolerability of a one-time, intrathalamic administration of AVB-101 in subjects with FTD-GRN.Secondary Objectiveo To evaluate the preliminary clinical and biomarker measures of efficacy of a one-time,…
validation Phase objective: Primary objective is to validate the high sensitive Troponin Quidel Triage True hsTnI. Secondary objective is to determine the applicability and repeatability. The focus will be on the ease of use, time spent performing…
The current prospective observational study proposes to use intra-patient comparisons of periods without and with contrast to elucidate the relationship between renal function and contrast administration in this population. The central question is:…
The main objective of study 20190530 is to estimate the efficacy of Apremilast compared to placebo in the treatment of oral ulcers in pediatric subjects from 2 to < 18 years of age with oral ulcers associated with BD through week 12. See…