Rapamycin

Number of Participants With Adverse Drug Reactions

Long term safety of mTOR inhibitor treatment in LAM

Volumetric changes in complicated vascular anomalies to sirolimus

Percentage of Participants With Main Reason for the Use of Sirolimus (Rapamune) Therapy

This study has a primary endpoint of allograft function as determined by Schwartz GFR at 18 months after conversion to CNI free protocol (2 years post transplantation).

Association Between the Magnitude of mTOR Target Inhibition Post-treatment in Leukemic Blasts and Clinical Response in Patients With High Risk AML Treated With Sirolimus MEC

GO overall response

Change in muscle mass from baseline

The rate of change in response of primary tumor and metastases (if applicable)in participants.

Insulin stimulated muscle glucose uptake

Slope in estimated glomerular filtration rate (eGFR; 4 variables MDRD equation) and proteinuria within six months of exposure to sirolimus.

Incidence of Cytomegalovirus Infection or Disease

Glomerular Filtration Rate (GFR) estimated by iohexol clearance at Week 52 post kidney transplantation

Angiographic restenosis

Cytomegalovirus infection/disease recurrence

Incidence of Efficacy Failure

Differences in Costs Between Two Revascularization Strategies for de Novo Coronary Lesions.

Renal function

Calculated creatinine clearance at month 6 post transplantation

Change From Baseline in Calculated Creatinine Clearance (Cockcroft-Gault Equation) at 52 Weeks Post-randomization

Safety evaluation

response to treatment

Best Overall Response Rate (BORR)

Plasma creatinine and creatinine clearance (Cockcroft-Gault) after 6 months of treatment

Number of Participants With Graft Survival at 12 Months

Progression of actinic keratosis or invasive squamous cell carcinoma (SCC) or incidence/reoccurrence of neoplastic skin tumors

Angiomyolipoma volume

Composite endpoint of either acute rejection, biopsy proven calcineurin inhibitor toxicity, or failure of the estimated BSA indexed GFR by MDRD method to improve by 20% from time randomization to 12 months.

Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps

Progression Free survival

Progression-Free Survival (PFS): Independent-Assessment

First biopsy-confirmed acute rejection, patient death or graft loss in the first 12 months following transplantation. First occurence of kidney graft loss or patient death in the first 12 months following transplantation.

Event-free Survival (EFS)

Progression Free Survival (PFS)

Progressive-free Survival (PFS)

Fistula Suitability for Dialysis at 6 Months (FSD6)

Freedom from Cinical Relevant Target Lesion Failure

Creatinine Clearance Calculated and Corrected According to Cockcroft Gault at Month 60

Progression-Free Survival (PFS)

To evaluate the safety and efficacy of an immunosuppressive regimen using synergic action of SRL and CsA, but which will limit (ab initio), the nephrotic effect of such combination. To compare Rapamune/low dose Cyclosporine vs. Rapamune/Steroids.

The Proportion of Patients Who Achieve an SLE Responder Index-4 (SRI-4) Composite Response at Week 24

KC development

Change in IBM Functional Rating Scale (IBM-FRS) from Baseline to Week 84

Number of Patients Experiencing Biopsy Confirmed Acute Rejection Through Month 6 After Transplantation.

facial angiofibroma severity index (FASI)

HCC recurrence free survival

Percentage of Participants Surviving at One and Four Years After Liver Transplant

Cumulative One-year Acute Rejection Rates

Total Kidney Volume (mL)

Change in Cognitive Function as Assessed by the National Institute of Health (NIH) Toolbox Cognitive Battery

Early and Late Pharmacokinetics of Sirolimus (SRL)

Objective Response Rate defined as % of participants in a cohort with complete or partial response or with stable disease according to standard response criteria

Number of Participants Who Progressed or Died by 25 Months From Enrollment

Progression Free Survival Rate

Therapeutic Benefit

Incidence of de novo Donor Specific Antibody (dnDSA) (central lab) OR decline in estimated glomerular filtration rate (eGFR) >7.5 mL/min/1.73m^2 (central lab)

Progression-free survival

Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score

Event-Specific Survival Comparisons

Objective Response

Overall response rate (ORR)

Overall survival at 1 year

Event Free Survival Probability

Progression-free Survival (PFS)

To determine if the rate of relapse at 24 months after CSA discontinuation can be improved by conversion to sirolimus in severe aplastic anemia patients who have responded to IST.

Percentage of Participants With Clinical Benefit

Overall Survival

The primary outcome is to explore the safety of sirolimus in the management of BSCMs.

Percentage of Participants With Evidence of Acute Graft Versus Host Disease (aGVHD), Post Transplant

Response rate of 20%

Progression-free Survival at 2 Years

Overall Response Rate (Complete and Partial Responses) as Defined by the International Workshop Criteria

Incidence of Treatment-Emergent Adverse Events (safety) of loratadine in combination with sirolimus after 52 weeks of treatment

Objective Response Rate (ORR)

Best-corrected visual acuity by ETDRS

Complete Response Rate

Response (CR or PR)

Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year

Time to Disease Progression Based on Volumetric MRI

Number of Participants With Response

The absolute number of T-reg after renal transplant in patients in treatment with rapamycin compared to patients treated with cyclosporine

Two-year progression-free survival in patients with high-risk solid tumors

Efficacy of a 12-week application period of 0.1% topical sirolimus in cutaneous microcystic lymphatic malformation versus topical vehicle

Proportion of confirmed tumor responses, defined to be either a CR or PR noted as the objective status on 2 consecutive evaluations at least 4 weeks apart

Rate of change in area of geographic atrophy

Evaluation of the FLT-PET and FDG-PET

Treatment efficacy at M12

Confirmed tumor response according to the Response Evaluation Criteria in Solid Tumors (RECIST)

Number of Patients Alive Without Disease Progression

Percentage of Patients With Overall Survival at 6 Months

To evaluate recurrence-free survival

Transplant-related Mortality

Progression free rate

4-month Progression-Free Survival Rate

Time to clinical recovery

Number of adverse events

Overall survival (OS)

6-month Progression Free Survival (PFS)

Objective Response Rate

Progression Free Survival

Overall response rate

Clinical Activity

Progression free survival (PFS) rate

Percentage of patients being progression free

Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants

Percentage of Patients Who Are Alive and Free From Advanced Respiratory Support Measures at Day 28.

Clinic Objective improvement rating at week 12

Proportion of patients who achieve a confirmed CR or PR during the first 24 weeks of treatment defined by the International Workshop criteria

Volume of Angiomyolipomata

Evaluation of the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis.

Progression-free Survival

Disease Progression

Clinical Regression of Metastatic Renal Cell Carcinoma (Partial Response (PR)) or Complete Remission of Tumor (Complete Response (CR))

The Percentage of Participants Achieved at Least 2-grade Improvement

Response to Window Therapy (2 Courses) for Group B (High-risk) - ESFT Participants

To evaluate the effect of the administration of Rapamune on a clinical composite variable (change in proteinuria, blood pressure and hematuria) in patients with IgA nephropathy with poor prognosis criteria

Change from baseline in morphologic, chromatographic and spectrometric scores at week 6

Craving Levels in Response to Cue

Overall response rate (ORR)

Number of lesions with no response

Overall hematological response rate using modified IWG criteria (combination of CR, PR, marrow-CR and SD with HI).

To assess the safety of a combination of rapa administration, donor Treg infusion and low-dose IL-2 in patients with steroid-refractory chronic GVHD.

Objective Tumor Response (Complete Response and Partial Response) and Progression in Participants With Stage III or IV Melanoma Following Treatment With Temsirolimus and Bevacizumab

Rate of Adverse Events

Probability of Acute Graft Versus Host Disease (GVHD)

Graft Rejection

longest diameter of renal angiomyolipomas assessed by MRI scan, toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events v3.0

To estimate the overall response rate (complete or partial response and stable disease by the IWG criteria) to temsirolimus

Progression-free survival in months

Change in Circulating Tumor Cell (CTC) Counts in Men With Metastatic Treatment-refractory Castration-resistant Prostate Cancer.

changes of pulmonary lymphatic perfusion distribution

Clinical progression free survival.

Tumor Response Rate, in Terms of the Proportion of Confirmed Tumor Responses (CR or PR) Assessed Using RECIST

plasma C-peptide AUC (mixed meal tolerance test [MMTT])

Change in colon polyp burden by number

Acute Rejection at 6-Months

The primary endpoint is progression-free survival (PFS)

Number of Participants Who Progressed

Incidence and Severity of Acute Graft-vs-host Disease (GVHD)

Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)

Insulin independence

Incidence and Severity of Acute GVHD Using Fludarabine Phosphate / Busulfan / Bortezomib Preparative Regimen and Triple Immune Suppression With Tacrolimus, Sirolimus and Anti-thymocyte Globulin

Measure of Squamous Cell Skin Carcinoma in Patients

PFS-6 rate

response rate

Initially, an ascending dose design will be used in order to evaluate the tolerability and safety of the combination and to determine the MTD of this combination (dose escalation phase)

Incidence of Dose-limiting Toxicity (DLT), Phase I Patients Only

Maximum Tolerated Dose (MTD) of the Each Combination Agent Combined With a Fixed Dose of BAY 43-9006 Determined by Dose-limiting Toxicities (DLT) (Phase I)

Cmax for Sirolimus

Percentage of Participants Who Have Not Rejected Their Stem Cell Graft and Who Are Without Severe Graft-versus-host Disease Following Stem Cell Transplant

Recommended Dose Level for Phase II Testing (RPTD) (Phase I)

Tumor Response

Phase I: Maximum Tolerated Dose (MTD) of Pemetrexed

Phase I: MTD / Phase II: ORR

Maximum Tolerated Dose (MTD) - Phase 1

Percent (%) Change in Levels of pS6, pAKt473, and Ki-67

Pharmocodynamically Optimal Dose (POD) of Rapamycin as Determined by Number of Participants With Greater Than or Equal to 60% Tumor S6 Kinase Inhibition by Immunohistochemistry (IHC).

Proportion of Treg cells in peripheral blood

Maximum Tolerated Dose (MTD) of Temsirolimus (Phase I)

Patients With Dose Limiting Toxicity

Number of Dose Limiting Toxicities of Ganetespib When Administered in Combination With Sirolimus.

recommended dose

The Maximum-tolerated Dose (MTD) of Nab-rapamycin in Combination With Pazopanib (Phase I) - Nab-Rapamycin Dose

Change in cerebral glucose metabolism

Safety of sirolimus, including number of lesions detected by brain MRI

Phase I Only: Maximum Tolerated Dose (MTD) of Pemetrexed That Could be Administered Weekly in Combination With Temsirolimus

Rate of Change in Area of Geographic Atrophy (GA) in the Study Eye at 24 Months as Compared to Baseline

Safety of Simvastatin in the Treatment of LAM-S and LAM-TS Patients

Overall Participant Survival at One Year Post Kidney Transplant

Safety, tolerability and efficacy of rapamycin in combination with HiVAC in relapsed and refractory patients with aggressive lymphoid malignancies

Improvement of the Rate of Graft Versus Host Disease (GVHD) From the Accepted Rate of 74%.

Change in Cerebral Blood Flow as measured by MRI

Maximum tolerated doses (MTDs) and Dose-limiting toxicities (DLTs)

Maximum Tolerated Dose (MTD) of Lenalidomide in Combination With Bevacizumab, Sorafenib, Temsirolimus, or 5-Fluorouracil, Leucovorin, Oxaliplatin (FOLFOX)

Maximum tolerated dose (MTD) of bevacizumab, defined as the dose level below the dose at which 2 of 6 patients experience drug-related dose limiting toxicity (DLT)

Number of Subjects With Dose Limiting Toxicities (DLT)

Muscle protein synthesis

Maximum Tolerated Dose (MTD) of Sirolimus or Vorinostat in Combination with Hydroxychloroquine in Participants with Advanced Cancers

Safety and tolerability of twice daily oral doses of AZD6244 when administered in combination with standard doses of selected chemotherapies.

RD, defined as the highest dose level at which less than 33% of at least six patients experience DLT

Number of dose limiting toxicity incidents as per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 3.0 (Phase I)

Number of participants with adverse events as assessed by CTCAE v5.0

maximum tolerated dose

Maximally tolerated dose (MTD), determined according to dose-limiting toxicities (DLTs) graded using Common Terminology Criteria for Adverse Events version 3.0 (CTCAE v3.0)

Pharmacokinetic interaction

Maximum tolerable dose (MTD) and recommended dose for phase II determined by dose-limiting toxicities (DLT) graded according to NCI Common Terminology Criteria for Adverse Events (CTCAE) version 3.0

Incidence of adverse events

Pharmacokinetic interactions

Number of Subjects With Dose Limiting Toxicities (DLTs)

Lenalidomide PK-AUC (0-24)

MTD, pharmacokinetic parameters

MTD for patients with moderate hepatic impairment

To determine the safety and tolerability of tivozanib (AV-951) when given in combination with temsirolimus

Toxicity

Incidence of dose-limiting toxicities (DLTs)

Maximum tolerated dose (MTD) of temsirolimus when given together with lenalidomide

Number of patients achieving successful donor engraftment

number of participants with adverse events based on the CTCAE v.4

Graft failure rate

MTD of CCI-779 and Bryostatin-1 administered in combination, graded according to NCI Common Toxicity Criteria, Version 3.0

Dose Escalation Part: Number of Dose-Limiting Toxicities (DLTs) at Each Dose Level on Cycle 1

To determine the safety, dose-limiting toxicities, and maximum tolerated dose of weekly oral Rapamycin in combination with weekly intravenous Abraxane for three out of four consecutive weeks in patients with advanced solid cancers.

Feasibility of haploidentical HSCT

safety and feasibility

Pharmacokinetics (PK) of sirolimus in whole blood: AUCinf

improve port wine stain (PWS) therapeutic outcome

Maximum tolerated dose (MTD)

Maximum tolerated dose of ruxolitinib phosphate, defined as less than or equal to 1 of 6 dose limiting toxicities, graded according to the Bearman criteria and the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.03

Dose-limiting toxicity and maximum tolerated dose

Maximum tolerated dose of bortezomib as defined as the highest dose tested in which none or only one patient experiences dose limiting toxicity attributable to the study regimen

Incidence of adverse events (CTCAE)

Incidence of treatment-emergent adverse events.

Maximum tolerated dose of temsirolimus

Dose-limiting Toxicities

Maximum tolerated dose of vinblastine in combination with sirolimus

Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients

Dose-limiting toxicity of temsirolimus in combination with intensive re-induction chemotherapy graded according to the NCI CTCAE v4.0

AUC0-48

MTD and RP2D of tivantinib in combination with temsirolimus defined as the highest safely tolerated dose where 0/6 or 1/6 patients experience a dose-limiting toxicity (DLT) and two or more patients have experienced a DLT at the next higher dose level

To determine the maximum tolerated dose of cediranib with temsirolimus in patients with recurrent/refractory gynecological malignancies.

Determine the maximum tolerate dose of temsirolimus given with radiation

Proportion of Participants who Achieve Operational Tolerance

Safety of ALVAC(2)-NY-ESO-1 (M)/TRICOM vaccine in combination with varying dose levels and schedules of sirolimus, assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Safety and toxicity

Phase 1: Safety and tolerability in the study population.

Incidence of grade 3+ treatment-related adverse events (TRAEs)

Severe Infusional Toxicity

To determine the dose-limiting toxicities and maximal tolerated doses of gemcitabine combined with temsirolimus

Number of Participants achieving engraftment without severe Graft-versus-host disease (GVHD)

Percentage of patients who are able to complete therapy

Safety and tolerability of treatment regimen as measured by grade 3, 4, and 5 toxicities

Change in Biological Age from Baseline to End of Study based on Tru Diagnostic Tru Age OMICm Age test results.

Stem Cell Transplant Engraftment

Safety/Efficacy of a novel regimen of sirolimus, tacrolimus and methotrexate

Pharmacokinetic interactions

5 year Event Free Survival

Blood Brain Barrier Penetration of RAPA

Percent of Participants Successfully Withdrawn From Immunosuppression and Remained Off Immunosuppression for at Least 52 Weeks

Number of Participants With Count Recovery That Allows for Starting a Phase II Study to Evaluate Response Rates and Survival

Number of Participants with Adverse Events

Improvement of ABSIS Score While Reducing Steroid Dosage

Proportion of confirmed responses, evaluated using the RECIST v1.1