Efficacy Endpoints. [*] to be quantified in [*]. In the case, that no [*] are available, [*] are to be quantified in the peripheral blood. Any one of the following three endpoints: - [*] fold enrichment of gene modified T cells [*] weeks after treatment [*], or - proportion of [*] > [*], [*] weeks after treatment, or - [*] of [*] over the course of the clinical trial. [*] Certain information on this page has been omitted and filed separately with the Securities and Exchange Commission. Confidential treatment has been requested with respect to the omitted portions. In addition, the following endpoint must also be met: -[*] from [*] following treatment. Efficacy endpoints shall apply for at least [*] out of [*] patients treated in Phase I/II Clinical Trial. [*] Certain information on this page has been omitted and filed separately with the Securities and Exchange Commission. Confidential treatment has been requested with respect to the omitted portions.
(1) License and Supply Agreement dated October 15, 1999 by and between XCYTE and Diaclone S.A., as amended
(2) Non-Exclusive License Agreement dated October 20, 1999 by and between XCYTE and Xxxx Xxxxxxxxxx Cancer Research Center, as amended
(3) License Agreement dated July 8, 1998 by and between XCYTE and Genetics Institute, L.L.C. (“GI”), as amended, including the exhibits:
(A) License Agreement between GI and the Secretary of the Navy dated December 10, 1996, as amended,
(B) License Agreement dated May 28, 1992 between GI and the University of Michigan, as amended,
(C) License Agreement dated July 20, 1993 between GI (as successor-in-interest to Repligen Corporation) and Xxxx Xxxxxx Cancer Institute, as amended. Xcyte™ Dynabeads® Volume: Storage: Storage buffer: 10 ml Store at 2-8°C [*]
Efficacy Endpoints. The primary efficacy endpoint is: Percent change from baseline in liver fat at Week 12, as determined by MRI-PDFF. The key secondary efficacy endpoint is: The percentage of subjects with at least a 30% reduction in liver fat at Week 12, as determined by MRI-PDFF. Additional efficacy endpoints are: Percent change from baseline in liver fat at Week 16, as determined by MRI-PDFF. Percentage of subjects with at least a 30% reduction in liver fat at Week 16, as determined by MRI-PDFF. Change from baseline over the 12-week treatment period in: – Liver aminotransferases. – Lipid and lipoprotein parameters. – XXXX and fibrosis markers. – Eicosanoids. Exploratory efficacy endpoints are: Change from baseline over the 12-week treatment period in: – Liver fibrosis, as determined by VCTE. – Metabolic parameters. – Anthropometric parameters. – Lipidomic analyses for DNL. Proportion of subjects with <5% liver fat (normalized) at Week 12.
Efficacy Endpoints. Primary efficacy endpoint Secondary efficacy endpoints:
Efficacy Endpoints. The primary efficacy endpoint of the Phase 2a is the proportion of evaluable participants (i.e. in the PP set) with a response of ≥ 5 letter gain in BCVA from baseline to week 12 according to ETDRS criteria in the combination aflibercept + OPT-302 group. Based on the results of 72 evaluable participants in the aflibercept + OPT-302 arm the combination therapy will be considered to have clinical activity if ≥ 27 of 72 participants have a ≥ 5 letter gain in BCVA from baseline to week 12 according to ETDRS criteria in the aflibercept + OPT-302 group. Likewise the combination therapy will be considered to have insufficient clinical activity if ≤ 25 of 72 participants have a ≥ 5 letter gain in BCVA from baseline to week 12 according to ETDRS criteria in the aflibercept + OPT-302 group. The difference in the mean change from baseline to week 12 between treatments will be tested for three secondary efficacy endpoints: mean BCVA, mean CST and mean macular volume. A model for repeated measures, fitted by restricted maximum likelihood method, will be used for the analysis. This model takes into account the presence of missing data and yields valid estimates under the assumption of data missing at random. In addition, the following secondary outcomes will be estimated in the Phase 2a by treatment arm, and their 95% confidence intervals will be obtained: • Percent of eyes with ≥ 50% reduction in excess foveal thickness from baseline to week 12 on SD-OCT • Percent of eyes with CST < 300 µm on SD-OCT through week 12 • Percent of participants with a ≥ 2 step improvement from baseline to week 12 in ETDRS Diabetic Retinopathy Severity Score • The mean time to, and number of, retreatment injections of aflibercept anti-VEGF-A therapy based on protocol specified criteria during week 12 to week 24 follow-up Further details of the analysis methods will be provided in the statistical analysis plan.
Efficacy Endpoints. The primary endpoints are the differences between *** and *** groups in mean percent loss of baseline body weight, determined by weight at randomization (baseline) and weight at end of treatment (week 56), and percent of subjects with weight loss of 5% or more at end of treatment (week 56). Percent weight loss will be calculated as ***. Secondary efficacy endpoints are: · The difference in absolute weight loss between randomization (baseline) and end of treatment (week 56) for *** and *** groups; · The difference in percent of subjects who achieve a reduction in total body weight of at least 10% between randomization (baseline) and end of treatment (week 56) for *** and *** groups; and · The difference in change in waist circumference (randomization to week 56) for *** and *** groups. Additional efficacy endpoints include: · Effect on ***; · Effect on body composition as indicated by ***. *** assessments will be performed at only at a ***. · Effect on *** of *** and ***; · Change from baseline to week 28 and week 56 in BMI; · The change in obesity-associated risk factors (HgbA1c, total cholesterol, triglycerides, LDL-C, HDL-C, fasting glucose, fasting insulin, a measure of insulin sensitivity, systolic blood pressure, diastolic blood pressure, C-reactive protein); · Change in urinary microalbumin and albumin/creatinine ratio (ACR) from screening to week 28 and week 56; · Change from baseline in medication number and dosages for medication to treat cardiovascular or metabolic risk factors; · Difference between *** and *** groups in the rate of progression to type 2 diabetes (subjects non-diabetic at screening); and · Baseline adjusted change in Framingham 10-year risk score at weeks 28 and 56. The change in weight loss (absolute, percent, percent of subjects achieving weight loss of > 5% and > 10% of starting weight), waist circumference and obesity associated risk factors will also be assessed over time. Subgroup analyses, including but not limited to analysis by gender, age and race, may be performed. *** will also be evaluated. Data will be obtained using a multiple trough sampling scheme with samples collected at *** and ***. Effects of various cofactors including (but not limited to) ***, gender, race, *** and age will be evaluated.
