15 results
Primary objectives:*To determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of EPZ-5676 when administered as a 21-day or 28 day CIV infusion to patients with refractory hematologic malignancies.*To assess the safety and…
The aim of this study is to improve 6MP treatment in pediatric leukemia patients, by developing and licensing a pediatric liquid formulation of 6MP, assessing its stability and bioequivalence, and ensuring a nationwide introduction of the new…
Primary objective: • To assess the efficacy of 2 different dosing regimens of subcutaneous GSK2402968 administered over 24 weeks in ambulant subjects with DMD.Secondary objectives:• To assess the safety and tolerability of 2 different dosing…
To compare the effects of various upper extremity positions on the sagittal spinal alignment, to determine which position corresponds the most with the neutral position and to determine which position is the most reliable across repeated…
The objectives of this study are to:- Evaluate the safety and tolerability of up to 2 dosing cycles of GemRIS for up to 21 days per dosing cycle.- Evaluate the pharmacokinetics of gemcitabine and 2', 2'-difluorodeoxyuridine (dFdU, a…
The objectives of the study are to: -To evaluate the safety and tolerability of MOTREM in patients with septic shock. -To evaluate the effects of MOTREM exposure over up to 5 days in patients with septic shock -To evaluate the PK/PD and dose/PD…
The primary objective of this clinical trial is to obtain pilot safety and efficacy data on treatment of PAH patients by 6-MP. The secondary objective of this clfnical trial is to determine whether LEPs transcriptome analysis will identify a subset…
- Evaluate the safety and tolerability of up to 2 dosing cycles of GemRIS for up to 7 days per dosing cycle - Evaluate the pharmacokinetics of gemcitabine and 2',2'-difluorodeoxyuridine (dFdU, a gemcitabine-related metabolite) exposure in…
Part I (dose-escalation): to evaluate the safety of SYD985 and to determine the Maximum Tolerated Dose and Recommended Phase 2 DosePart II (expanded cohorts): to evaluate the Objective tumour Response Rate
Primary objective:Evaluate the safety, tolerability, pharmacokinetics (PK), and immunogenicity of multiple doses of intravenous (IV) ATYR1940 in adults 18 to 65 years of age, inclusive, with FSHDSecondary objective:Explore pharmacodynamic (PD)…
The primary objective is to assess the effects on amyloid plaque burden of administration of VX-745 for 12-weeks, as assessed by Dynamic 11C-PiB (Carbon-11 labeled Pittsburgh Compound B) PET Amyloid Scanning in patients with Mild Cogntitive…
The objectives of this study are to:• Evaluate the safety, tolerability, and immunogenicity of long-term treatment with intravenous (IV) ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD) previously enrolled in clinical…
Diabetic Macular Ischemia (DMI) is a complication of diabetic retinopathy (DR) and can lead to vision loss. Currently, there are no approved or effective treatments to prevent either onset or progression of DMI in DR patients.As a transition from…
The primary objective is to evaluate the safety, tolerability, and doselimiting toxicities, and determine the recommended dose for expansionof INCA00186 as monotherapy and of combination treatments of INCA00186 with retifanlimab and/or INCB106385 in…
The primary objective of this study is to demonstrate that SYD985 is superior to physician*s choice in prolonging progression-free survival (PFS) on the basis of the blinded independent central review of tumour assessment.The secondary objectives of…