24 results
• To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects with LCA or RP caused by RPE65 or LRAT gene mutations who have been treated previously with a single 7-day course of QLT091001 in…
We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…
The objectives of the study are to assess the safety and tolerability of ascending doses of SAR422459 in patients with Stargardt macular disease and to evaluate the possible biological activity of SAR422459.
Primary objectives: To assess the feasibility of ZW800-1 in intraoperative detection of the urinary tract using the NIR fluorescence imaging system. Secondary objectives: To define the optimal dose of ZW800-1 for intraoperative imaging of the…
Primary Objective:To assess the ability of BR55 to identify area(s) of VEGFR2 expression in human prostate by ultrasound molecular imaging on the basis of a visual score in comparison with histopathology analysis (location based on expression of…
Primary:to examine the effect of co-administration with omeprazole on the pharmacokinetics of SSP-002358.Secondary:to provide additional safety information for SSP-002358 when administered alone or in combination with omeprazole.
Primary: To assess the effect of multiple doses of tasqinimod on the single-dose pharmacodynamics (PD) of warfarin. Secondary: To assess the effect of multiple doses of tasqinimod on the single-dose pharmacokinetics (PK) of warfarin. To assess the…
Primary objective:* To determine the safety, tolerability, dose-limiting toxicities (DLT), maximum acceptable dose (MAD) and maximum tolerated dose (MTD) of CHR-2845 when administered orally to patients with advanced or treatment refractory…
Primary goal is the evaluation of the safety and tolerability of MMMAE-anti-CD79b-conjugate administered every 3 weeks. The maximal tolerated dose and the dose-limiting toxicity are the important parameters. Based on these data, the dose of MMAE-…
The primary objective is to assess the effect a resuscitation strategy using balanced solutions versus unbalanced solutions on regional and microvascular perfusion relative to global hemodynamics in intensive care patients following elective cardiac…
Primary Objectives:* Identify the Maximum Tolerated Dose (MTD) and the Recommended Phase 2 Dose (RP2D, 200mg BID) of TAS-119.
The purpose of the study is to investigate to what extent blood concentrations of Fluticasone Furoate increase proportionately to rising doses of Fluticasone Furoate in three steps administered via a powder inhalator. In addition, the absolute…
To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
Primary- To evaluate the effects of the 15 mg E4/3 mg DRSP combination and the 20 mcg EE/3 mg DRSP used as reference combination on ovarian function inhibition at Treatment Cycle 1 and Treatment Cycle 3.Secondary- To evaluate levels of luteinizing…
Primary:To evaluate the effects of 15 mg E4/3 mg DRSP, of 30 mcg EE/150 mcg LNG, and of 20 mcg EE/3 mg DRSP on hemostasis, endocrine function and lipid and carbohydrate metabolism parameters during 6 treatment cycles.Secondary:To assess the safety…
Primary objective:To assess the clinical activity of AL101 using radiographic assessments and RECIST v1.1 in ACC patients with activating Notchmutations.Secundary objectives:• To assess quality of life in ACC patients with activating mutations.• To…
To evaluate the effect of BIVV020 on the durabilityof platelet response in participants withpersistent/chronic immune thrombocytopenia (ITP)Secondary• To assess the safety and tolerability of BIVV020• To assess the pharmacokinetics (PK) of BIVV020•…
Primary Objective:Assess the antitumor activity of BDTX-189 as a single agent by evaluation of objective response as determined by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 in patients with allosteric human epidermal growth…
Main objective:1. To estimate the Overall Response Rate (ORR) and the clinical benefit rate in selected cohorts of patients with hematologic malignancies,separately for each cohort2. To evaluate the safety of the selected dose regimes in an expanded…
DOSE ESCALATION PHASE: PART A: Primary Objectives: • To assess the safety and tolerability of KBA1412 when given as monotherapy. • To determine the maximum tolerated dose (MTD) and/or Recommended Phase II dose (RP2D) of KBA1412 when given as…