ACT is currently enrolling patients in three clinical trials in the U.S...The trials are prospective, open-label studies, designed to determine the safety and tolerability of hESC-derived RPE cells following subretinal transplantation in patients with dry AMD or SMD at 12 months, the study's primary endpoint

Mirna Therapeutics Inc., of Austin, Texas, has initiated a Phase I study of MRX34, the first miRNA to advance to a clinical trial for cancer...The Phase IV study is currently open at 12 sites and will be conducted in approximately 25 sites in the U.S. The open-label, two-arm study is enrolling mM patients with BRAF V600 oncogene mutations

Isis has 28 candidates in preclinical and clinical development across a wide variety of indications, with nine scheduled to report data in the next 12 months...Dendreon also is continuing to evaluate partnering strategies for European expansion and to enroll patients in an open-label sipuleucel-T

The NH004-3 clinical trial is a double-blind, placebo-controlled, randomized, crossover, multicenter study comparing NH004 and placebo thin films in Parkinson's disease patient complaining of sialorrhea...The study is an open-label, multiple-dose, dose-escalation study to evaluate the safety and tolerability of SNS01-T when administered by intravenous infusion to approximately 15 relapsed or refractory multiple myeloma, mantle cell (MCL) or diffuse large B-cell lymphoma (DLBCL) patients

The open-label trial will randomize 20 postmenopausal women with estrogen receptor-positive disease who have responded previously to hormone therapy to receive 9 mg of enobosarm once daily until they show clinical progression or have completed 336 days of treatment

In an open-label Phase II study conducted in children with simplex, recessive dystrophic EB or junctional EB, SD-101 application resulted in complete closure of 88 percent of target chronic lesions within one month.

The open-label trial will evaluate efficacy, safety and tolerability at two sites in France, enrolling up to 32 HCV-infected patients of any genotype who previously failed or relapsed following standard-of-care treatment...The open-label study, which randomized 153 patients, evaluated two schedules of TG4040 in combination with PegIFNα2a (P) and ribavirin (R) vs. P/R alone (arm A

The open-label, pivotal trial will enroll 400 subjects at about 80 sites in Australia, Canada, China, Europe, Latin America and the U.S. The primary endpoint is progression-free survival, and secondary endpoints include overall survival and safety...Data from the open-label, dose-escalation pilot study will be used to define the optimal dose for a larger Phase II/III study in infants

The primary objectives include the evaluation of adverse events and a six-month clinical response...The study is an open-label, multiple-dose, dose-escalation study that will evaluate the safety and tolerability of SNS01-T when administered by intravenous infusion to approximately 15 relapsed or refractory multiple myeloma, mantle cell or diffuse large B-cell lymphoma patients

The open-label study under an investigational new drug application is designed to evaluate the safety and efficacy profile of repeated escalating doses of BL-8040 in adults with relapsed/refractory AML...The open-label Phase I/II study is designed to assess the safety and potential efficacy of gevokizumab, a monoclonal antibody designed to bind to interleukin-1 beta

A preliminary open-label study of a precursor of the missing metabolite yielded promising results in 10 patients, Sedel said...The company's "exceptional team" and its potential to obtain rapid clinical efficacy data were two attractive attributes, Rafaèle Tordjman, managing partner at Sofinnova Partners, told BioWorld Today

The primary endpoint of the study is the induction of clinical remission at week 12...The study is being conducted by more than 60 clinical investigators worldwide, including about 11 sites in Brazil and 24 sites in India...The TRIG-05 study is expected to begin enrollment this month, with a primary clinical endpoint of demonstrating efficacy, tolerability and safety of TI-001

Array BioPharma Inc., of Boulder, Colo., reported clinical data at the International Myeloma Workshop in Kyoto, Japan, showing that ARRY-520, a selective KSP inhibitor, demonstrated single-agent activity in heavily pretreated patients with relapsed or refractory multiple myeloma, with 19 months median overall survival and a 16 percent overall response rate...Vital Therapies Inc., of San Diego, enrolled the first patient in a Phase III trial designated VTI-208, a randomized, open-label, multicenter

The open-label study will test the safety and efficacy of intra-articular injections of the autologous treatment in order to reduce inflammation and regenerate damaged joint tissue...Novavax Inc., of Rockville, Md., said top-line data from its Phase II dose-ranging study testing its respiratory syncytial virus (RSV) vaccine candidate in women of childbearing age hit its protocol-specified objectives, supporting progression to the next stage of advanced clinical testing

The open-label study assessed the safety and efficacy of CCH in Stage II unilateral idiopathic FSS in comparison to an exercise-only control group...OncoSec expects to complete enrollment in the single-arm, open-label, multicenter trial, with approximately 25 patients, in the second quarter

Benitec Biopharma Ltd., of Sydney, Australia, selected the Duke Clinical Research Unit in Durham, N.C., as a site for its upcoming Phase I/II first-in-human trial of TT-034 in hepatitis C virus (HCV...The open-label, dose escalation study will evaluate the safety and activity of single doses of TT-034 in patients with chronic HCV genotype 1 infection who failed previous treatments

In the single-dose, open-label study, ISIS-SMNRx was well tolerated in children with SMA at all dose levels tested and improvements were observed in Hammersmith scores, a measure of muscle function, in a number of the children...During the entire conduct of the study, including the double-blind and open-label phases, early start patients were less likely to experience disease progression than those with a delayed start of laquinimod (11.8 percent risk of confirmed disability progression vs. 16.7

The company examined the triglycerides data from its open-label study in the course of planning its Phase III development of CaPre...The NX03-0040 prostate cancer clinical study of 150 men is testing low (2.5 mg) and high (15 mg) single doses of NX-1207 for the effect to eradicate or shrink low-grade localized prostate cancer tumors

Secondary objectives of the trial include clinical activity by serum TTR levels...Merrimack Pharmaceuticals Inc., of Cambridge, Mass., enrolled its last patient in a Phase II, open-label, randomized trial of MM-121, a human monoclonal antibody, in combination with paclitaxel, compared to paclitaxel alone in advanced ovarian cancers...For the Uceris 9-mg group, 17.4 percent of patients achieved clinical and endoscopic remission, compared to 4.5 percent of the placebo group (p = 0.0047