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A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with the study drug. The information gained from this study is expected to further characterize the efficacy and safety of BMN 044 over a…

A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Biodistribution, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.

A phase I/II *minor histocompatibility antigen UTA2-1 loaded, PD-L silenced Dendritic cell vaccination trial after allogeneic Stem Cell Transplantation to improve the safety and efficacy of Donor Lymphocyte Infusions.

Primary objectives:- To evaluate the toxicity and feasibility of a DLI-combined minor H ag UTA2-1 peptide-loaded, PD-L silenced donor DC vaccination in B cell hematological malignancies- To evaluate the effect of a DLI-combined minor H ag UTA2-1…

7-day study of the safety, efficacy and the pharmacokinetic and pharmacodynamic properties
of oral rivaroxaban in children from birth to less than 6 months with arterial or venous
thrombosis

The primary objective is:- to characterize the pharmacokinetic/pharmacodynamic profile of a 7-day treatment with oral rivaroxabanThe secondary objectives are:- to assess the incidence of major bleeding and clinically relevant non-major bleeding- to…

LungTech Stereotactic Body Radiotherapy (SBRT) of inoperable centrally located NSCLC: A phase II study in preparation for a randomized phase III trial

The primary objective of this trial is to assess the effectiveness of IG-SBRT in patients with medically inoperable early stage, centrally located NSCLC and in those who are not willing to undergo surgical treatment.The primary measure of…

A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) (study CB8025-21427)

Primary: To evaluate the effect of MBX-8025 on LDL-C.Secondary: To evaluate the effects of MBX-8025 on other lipid parametersTo evaluate the safety and tolerability of MBX-8025.To evaluate steady-state trough plasma levels of MBX-8025 and its…

Randomized phase II trial of cabazitaxel or prolonged infusional ifosfamide in metastatic or inoperable locally advanced dedifferentiated liposarcoma

The main objective is to determine whether cabazitaxel or prolonged infusional ifosfamide demonstrate sufficient antitumor activity (as measured by progression free survival at 12 weeks) in pre-treated patients with metastatic or inoperable locally…

Adult mesenchymal stromal cells to regenerate the neonatal brain: the PASSIoN trial (Perinatal Arterial Stroke treated with Stromal cells IntraNasally)

This study will investigate whether bone marrow-derived allogeneic MSCs, as administered by the nasal route, can induce the formation of neuronal tissue and restore brain function in neonates who suffered from perinatal arterial ischemic stroke (…

EMDR in the treatment of Chronic Fatigue Syndrome (CFS): can EMDR make the perception of fatigue less negative?
A sequential randomized and replicated single-case experimental phase design with multiple measurements.

Primary Objective: The main aim of this study is to investigate if EMDR diminishes negative associations with fatigue in CFS-patients who follow CBT.Secondary Objective(s): Second, we will investigate if EMDR strengthens the belief of the patient…

*Perception of Emotional Expressions in a different Social Contexts
Allows People to Infer Each Other Preferences*

Primary objective We investigate whether observers* inferring accuracy differs when the consumer is in the presence of a friend compared to being completely alone. We test whether observers* brain activations during inferring observation differ…

weak magnetic fields against pain in fibromyalgia

We aim to study the effects of PEMF on pain in fibromyalgia.

A Phase 1b/Adaptive Phase 2 Study of Docetaxel With or Without MLN1117 in Patients
With Locally Advanced or Metastatic Non-small Cell Lung Cancer

Phase 2: To evaluate progression-free survival (PFS) as the primary efficacy measure of MLN1117 plus docetaxel versus docetaxel alone in patients with advanced NSCLC

The physiology of glucagon-like peptide-1 receptor expression in patients with endogenous hyperinsulinism: correlation with histopathology

The main study aim is the in vivo and ex vivo investigation of the expression and distribution of the GLP-1R in the pancreas of children (< 16 years) with proven endogenous congenital hyperinsulinism who qualify for surgery based on response…

Therapy of Refractory Inhibitors with immUne Modulation in Patients with Hemophilia. The TRIUMPH trial

To evaluate the efficacy, safety and feasibility of combined immune modulation with rituximab, ITI and MSCs in terms of eradication of FVIII inhibitory activity in hemophilia A patients.

A Multicenter, Open-Label, Single- and Multiple-Dose, Dose-Finding Study, with an Optional Open-Label Extension to Assess the Safety, Tolerability, and Pharmacokinetics of Obeticholic Acid in Pediatric Subjects with Biliary Atresia.;Trial Acronym: ObetiCholic Acid in Pediatric Subjects with BiliaRy AtrEsia (CARE)

Primary Objectives• Safety and tolerability• PK of OCA and its conjugates o SD PK Phase: To assess the PK of OCA and its conjugates after a single low dose of OCA on Day 1 and determine subject eligibility for the MD Phase o MD Phase: To assess the…