Argus® II Retinal Prosthesis System Post-Market Surveillance Study
This study is being conducted to monitor the use of Argus II in a larger population than
available within pre-market approval studies. Safety data will be monitored to ensure
continued acceptability of risks to study participants, and an attempt will be made to
include all eligible and willing participants implanted with Argus II. Measures of visual
function that may contribute to device improvements will also be gathered and evaluated.
A Phase 1b Dose Escalation Study of OMP-54F28 in Combination With Nab-Paclitaxel and Gemcitabine in Patients With Previously Untreated Stage IV Pancreatic Cancer
Depending on safety in this study, additional lower or intermediate dose levels may be
evaluated. Depending on emerging safety data from the Phase 1a study 54F28-001 with
continuing dose escalation, additional higher dose levels of OMP-54F28 may be evaluated in
this study. No dose escalation of OMP-54F28 will be allowed within a dose cohort.
Once the maximum tolerated dose (MTD) or maximum administered dose (MAD) has been determined,
up to 10 patients may be enrolled in the cohort-expansion phase to better characterize the
safety, tolerability and PK of OMP-54F28 combined with nab-paclitaxel and gemcitabine. Up to
approximately 34 patients may be enrolled into the study.
Phase II Study of Metformin in a Pre-prostatectomy Prostate Cancer Cohort
PRIMARY OBJECTIVES:
I. To determine the effect of 4-12 weeks of metformin (metformin hydrochloride) intervention
on cell proliferation in the prostatectomy tissue.
SECONDARY OBJECTIVES:
I. To determine the effect of metformin intervention on prostate tissue bioavailability of
metformin.
II. To determine the effect of metformin intervention on apoptosis and angiogenesis in the
prostatectomy tissue.
III. To determine the effect of metformin intervention on potential molecular targets of
metformin including activated protein kinase (AMPK) activation, mammalian target of rapamycin
(mTOR) regulation, and cell cycle regulation in the prostatectomy tissue.
IV. To determine the effect of metformin intervention on changes in systemic hormones and
growth factors that have been shown to be modulated by metformin in other patient populations
including fasting glucose, fasting insulin, insulin-like growth factor axis, testosterone,
and sex hormone binding globulin (SHBG).
V. To determine the effect of metformin intervention on changes in prostate-specific antigen
(PSA) levels.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM I: Patients receive extended-release metformin hydrochloride orally (PO) once daily (QD)
for 4-12 weeks.
ARM II: Patients receive placebo PO QD for 4-12 weeks.
Patients in both arms undergo surgery one day after completion of treatment.
After completion of study treatment, patients are followed up within 30 days of surgery.
Pulmonary Arterial Hypertension (PAH) Quality Enhancement Research Initiative (QuERI) Extension Program
This is a prospective, U.S.-based, multi-center, knowledge translation program tracking patient management until approximately early 2018. To improve the management of PAH patients through an evidence-based approach aimed at achieving optimal WHO functional class (FC). The goal of the optimal functional class is to improve WHO functional class III and IV patients to functional class I or II, and to improve all functional class II patients to functional class I, or at least to maintain functional class I/II in patients presenting in that functional class. Endpoints:1. Proportion of patients achieving guideline-recommended treatment at each documented visit.2. Proportion of patients achieving optimal functional class using an evidence-based reatment algorithm.3. Proportion of patients on various therapies in relation to their WHO functional class.4. PAH management (use of therapies) by physicians across patient risk strata based on FC.5. Association between treatment and physician and patient adherence with recommended treatment.6. Utilization of Knowledge Translation Program system reminders, and response by the physicians when asked for reasons evidence based therapy was not utilized.7. Survival by FC (baseline and change), etiology, prevalent vs incident, academic vs community, adherence and reminders.Descriptive analysis of demographic variables, physical exam, medical history and treatment profile will be performed as appropriate. Confidence Intervals (95%) will be calculated for descriptive and comparative purposes.
Not recruiting | High Blood Pressure / Hypertension | Site Unknown
A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload
This randomized, double blind trial to evaluate deferasirox vs placebo in patients with
myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload consisted of four
periods, a screening period, a treatment period, a post treatment follow-up period and a
survival period. The trial recruitment period lasted until December 2014 and the trial
continued for three years from the date the last patient enrolled until February 2018 (last
patient last visit date).
Screening period:
The screening period lasting up to 35 days with two screening visits, at least 14 days apart,
used to assess patient eligibility. Eligible patients with low or int-1 risk myelodysplastic
syndromes (MDS) with transfusional iron overload were randomized in a 2:1 ratio to
deferasirox or placebo respectively. Randomization was also stratified using the
International prognostic scoring system of low or int-1 MDS and by geographical region (Asian
vs non-Asian countries) since the Asian population has been reported to have a longer
survival.
The following concomitant medications could be permitted for use while the patient was on
study, and information outlining start date(s) and end date(s) of each medication taken were
to be recorded on the appropriate eCRF: Erythropoietin (growth factor), G-CSF (growth
factor), GM-CSF growth factor), Azacitidine, Thalidomide, Arsenic trioxide, Lenalidomide,
Decitabine, Cyclosporine A, Vitamin C supplements (≤ 200 mg/day)
Treatment period:
The dosing schedule was 10 mg/kg/day (once daily) for the first 2 weeks, followed by 20
mg/kg/day (once daily). After 3 months of treatment at the dose of 20mg/kg/day, the dose
could be adjusted by 5 or 10 mg/kg/day up to 40 mg/kg/day based on the serum ferritin
response. Placebo matching to each strength of the active deferasirox was utilized to
maintain the double-blind trial design.
During the treatment period patients returned to the investigational site every four weeks
for routine procedures and to monitor safety, efficacy and compliance to treatment.
An external Data Monitoring Committee (DMC) monitored patient safety and trial conduct and
received a blinded summary of serious adverse events.
All suspected endpoint events were reviewed and adjudicated by the Endpoint Adjudication
Committee (EAC) to ensure that all events that were reported were judged uniformly using the
same criteria. The first confirmed suspected endpoint event for a patient was counted for the
trial's composite primary endpoint, "event free survival". The composite primary endpoint,
"event free survival," was defined for a patient as the date randomized to trial treatment to
the date of the first documented non-fatal event, related to cardiac and liver function,
transformation to AML, or death due to any cause.
When a patient had a non-fatal event, related to cardiac and liver function, and
transformation to AML, the trial treatment (deferasirox or placebo) was discontinued. After
trial treatment was discontinued, a 28 days post treatment safety assessment for AEs and SAEs
was completed. Any patient who died during the treatment or 28 day post treatment safety
assessment is represented in the all-cause mortality table in the safety section of this
result. After trial treatment was discontinued for a patient, their treatment was un-blinded.
Subsequent iron chelation treatment was subject to the patient's and investigator's decision.
Patients continued to be followed during the post-treatment evaluation or survival follow up
period, depending on their choice.
For patients who did not meet a non-fatal event, study treatment was continued as long as the
patient and the treating physician felt it was in the best interest for the patient or until
the trial terminated/completed. There was no un-blinding of the trial treatment for patients
who terminated trial treatment without meeting a non-fatal event. Patients continued to be
followed during the post-treatment evaluation or survival follow up period, depending on
their choice.
A patient who discontinued study treatment without meeting a non-fatal component of the
composite primary endpoint continued to be evaluated every 3 months. Once a patient stopped
study evaluations they were followed for at least every 6 months for overall survival and any
iron chelation therapies they are receiving up to the end of study.
Post-treatment evaluation period:
For patients who had a non-fatal event: After treatment termination, all patients were
followed for safety (28 days) and then evaluated with visits every three months if they
agreed to move into the post treatment evaluation phase.
For patients who did not meet a non-fatal event: After termination of study treatment, if a
patient and investigator chose the post-treatment evaluation period, the patient was followed
for safety and endpoints at visits occurring every three months.
Survival Follow Up period:
Subsequent to the post treatment evaluation period, or at the end of treatment period, if a
patient and treating physician decided that the patient would not participate in the post
treatment evaluation period, the patient was followed every 6 months for overall survival and
iron chelation therapies.
The end of the study was defined as three years from the date the last patient was enrolled
(last patient first visit).
The sample size of 210 patients did not provide sufficient power for testing statistical
hypotheses. The statistical analysis was revised accordingly to concentrate on evaluating the
treatment effect of deferasirox relative to placebo, and the study phase designation was
changed from Phase lll to Phase II. Amendment 4 of the study adjusted the sample size,
statistical analysis, and duration of the study and added two secondary endpoints:
Hematologic improvement (HI) in terms of erythroid response and Frequency and rate of
infections requiring intravenous (IV) antimicrobials. Upon approval of the amendment,
patients signed a new consent form and continued the appropriate visit schedule.
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