scholarly journals Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study

2015 ◽  
Vol 46 (3) ◽  
pp. 783-794 ◽  
Author(s):  
Hilary J. Goldberg ◽  
Sergio Harari ◽  
Vincent Cottin ◽  
Ivan O. Rosas ◽  
Elizabeth Peters ◽  
...  
Keyword(s):  
Lung Function ◽  
Adverse Events ◽  
Mammalian Target Of Rapamycin ◽  
Mtor Inhibitors ◽  
Collagen Iv ◽  
Blood Levels ◽  
Forced Expiration ◽  
Open Label ◽  
Serious Adverse Events ◽  
Cystic Lung

Lymphangioleiomyomatosis is a rare, progressive cystic lung disorder characterised by dysregulated activation of mammalian target of rapamycin (mTOR) signalling.This was a phase IIa, multicentre, open-label study of the mTOR inhibitor everolimus (2.5 mg·day−1 escalated to 10 mg·day−1) in 24 women with lymphangioleiomyomatosis. Primary endpoints were safety, pharmacokinetics and serum vascular endothelial growth factor-D (VEGF-D) levels; secondary endpoints were measures of lung function.Following 26 weeks of everolimus treatment, forced vital capacity exhibited stability, while forced expiration volume in 1 s improved from baseline, with mean changes (95% confidence interval) of 10 mL (−111–132) and 114 mL (11–217), respectively; 6-min walk distance improved by 47 m. Median VEGF-D and collagen IV levels decreased from baseline, from 1730 pg·mL−1 to 934.5 pg·mL−1, and 103 ng·mL−1 to 80.5 ng·mL−1, respectively. Adverse events were mostly grade 1−2; mouth ulceration, headache, nausea, stomatitis and fatigue were common. Serious adverse events suspected to be treatment related included peripheral oedema, pneumonia, cardiac failure and Pneumocystis jirovecii infection. Everolimus blood levels increased dose proportionally.In this study, everolimus improved some measures of lung function and exercise capacity and reduced serum VEGF-D and collagen IV. Side effects were generally consistent with known toxicities of mTOR inhibitors, although some were severe.

scholarly journals Safety of Denervation Following Targeted Lung Denervation Therapy for COPD: AIRFLOW-1 Three-year Outcomes

2020 ◽  
Author(s):  
Christophe Pison ◽  
Pallav Shah ◽  
Dirk-Jan Slebos ◽  
Vincent Ninane ◽  
Wim Janssens ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Lung Function ◽  
Adverse Events ◽  
Late Onset ◽  
Open Label ◽  
Serious Adverse Events ◽  
Copd Exacerbations ◽  
Clinical Consequences

Abstract Background: Targeted lung denervation (TLD) is a novel bronchoscopic therapy that disrupts parasympathetic pulmonary nerve input to the lung reducing clinical consequences of cholinergic hyperactivity. The AIRFLOW-1 study assessed safety and TLD dose in patients with moderate-to-severe, symptomatic COPD. This analysis evaluated the long-term impact of TLD on COPD exacerbations, pulmonary function, and quality of life over three years of follow up.Methods: TLD was performed in a prospective, energy-level randomized (29 W vs 32 W power), multicenter study (NCT02058459). Additional patients were enrolled in an open label confirmation phase to confirm improved gastrointestinal safety after procedural modifications. Durability of TLD was evaluated at one, two, and three years post-treatment and assessed through analysis of COPD exacerbations, pulmonary lung function, and quality of life. Results: Three-year follow-up data were available for 73.9% of patients (n=34). The annualized rate of moderate to severe COPD exacerbations remained stable over the duration of the study. Lung function (FEV1, FVC, RV, and TLC) and quality of life (SGRQ-C and CAT) remained stable over three years of follow-up. No new gastrointestinal adverse events and no unexpected serious adverse events were observed. Conclusion: TLD in COPD patients demonstrated a positive safety profile out to three years, with no late-onset serious adverse events related to denervation therapy. Clinical stability in lung function, quality of life, and exacerbations were observed in TLD treated patients over three years of follow up.

scholarly journals Safety of denervation following targeted lung denervation therapy for COPD: AIRFLOW-1 3-year outcomes

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Pison Christophe ◽  
L. Shah Pallav ◽  
Slebos Dirk-Jan ◽  
Ninane Vincent ◽  
Janssens Wim ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Lung Function ◽  
Adverse Events ◽  
Late Onset ◽  
Open Label ◽  
Serious Adverse Events ◽  
Copd Exacerbations ◽  
Clinical Consequences

Abstract Background Targeted lung denervation (TLD) is a novel bronchoscopic therapy that disrupts parasympathetic pulmonary nerve input to the lung reducing clinical consequences of cholinergic hyperactivity. The AIRFLOW-1 study assessed safety and TLD dose in patients with moderate-to-severe, symptomatic COPD. This analysis evaluated the long-term impact of TLD on COPD exacerbations, pulmonary function, and quality of life over 3 years of follow up. Methods TLD was performed in a prospective, energy-level randomized (29 W vs 32 W power), multicenter study (NCT02058459). Additional patients were enrolled in an open label confirmation phase to confirm improved gastrointestinal safety after procedural modifications. Durability of TLD was evaluated at 1, 2, and 3 years post-treatment and assessed through analysis of COPD exacerbations, pulmonary lung function, and quality of life. Results Three-year follow-up data were available for 73.9% of patients (n = 34). The annualized rate of moderate to severe COPD exacerbations remained stable over the duration of the study. Lung function (FEV1, FVC, RV, and TLC) and quality of life (SGRQ-C and CAT) remained stable over 3 years of follow-up. No new gastrointestinal adverse events and no unexpected serious adverse events were observed. Conclusion TLD in COPD patients demonstrated a positive safety profile out to 3 years, with no late-onset serious adverse events related to denervation therapy. Clinical stability in lung function, quality of life, and exacerbations were observed in TLD treated patients over 3 years of follow up.

scholarly journals Comparison of Safety and Tolerability of Deutetrabenazine During Titration and Maintenance in Patients with Tardive Dyskinesia

CNS Spectrums ◽  
2021 ◽  
Vol 26 (2) ◽  
pp. 164-180
Author(s):  
Amanda Wilhelm ◽  
Karen E. Anderson ◽  
Hubert H. Fernandez ◽  
Hadas Barkay ◽  
Nayla Chaijale ◽  
...  
Keyword(s):  
Suicidal Ideation ◽  
Adverse Events ◽  
Tardive Dyskinesia ◽  
Dry Mouth ◽  
Fixed Dose ◽  
Open Label ◽  
Serious Adverse Events ◽  
Open Label Extension ◽  
Pharmaceutical Industries ◽  
Flexible Dose

AbstractBackgroundDeutetrabenazine is approved to treat tardive dyskinesia (TD) in adults and is titrated weekly by 6 mg/day, from 12 to 48 mg/day, based on dyskinesia control and tolerability. This analysis compared the safety of deutetrabenazine during titration versus maintenance.MethodsSafety was assessed during titration versus maintenance using integrated data from two 12-week placebo-controlled studies (ARM-TD and AIM-TD) and the open-label extension study. Rates were compared for overall and serious adverse events (AEs), AEs leading to discontinuation, treatment-related AEs, common AEs (≥4%), and specific AEs (parkinsonism, suicidal ideation, akathisia, restlessness).ResultsIn titration versus maintenance, AE rates with placebo (n=130) were: overall, 43.1% vs 25.4%; serious, 4.6% vs 2.3%; leading to discontinuation, 3.1% vs 0; treatment-related, 26.9% vs 10.0%. For placebo, common AEs during titration were somnolence, headache, nausea, fatigue, and dry mouth; none occurred during maintenance. In titration versus maintenance, AE rates in fixed-dose deutetrabenazine 12–36 mg (n=216) were: overall, 33.3–38.9% vs 22.2–29.2%; serious, 2.8–6.9% vs 0–1.4%; leading to discontinuation, 2.8–5.6% vs 0; treatment-related, 8.3–16.7% vs 8.3–13.9%. For fixed-dose deutetrabenazine, common AEs during titration were headache, diarrhea, nasopharyngitis, depression, hypertension, and dry mouth; headache was the only common AE during maintenance. In titration versus maintenance, AE rates with flexible-dose deutetrabenazine (n=168) were: overall, 49.4% vs 32.7%; serious, 3.6% vs 2.4%; leading to discontinuation, 2.4% vs 0.6%. For flexible-dose deutetrabenazine, the only common AE during titration was somnolence; none occurred during maintenance. Rates of parkinsonism, suicidal ideation, akathisia, and restlessness were low and comparable in titration and maintenance.ConclusionsDeutetrabenazine was well-tolerated, with AE rates similar to placebo during both phases; AE rates were higher during titration and decreased during maintenance.FundingTeva Pharmaceutical Industries Ltd., Petach Tikva, Israel

Allopurinol dose escalation to achieve serum urate below 6 mg/dL: an open-label extension study

2017 ◽  
Vol 76 (12) ◽  
pp. 2065-2070 ◽  
Author(s):  
Lisa K Stamp ◽  
Peter T Chapman ◽  
Murray Barclay ◽  
Anne Horne ◽  
Christopher Frampton ◽  
...  
Keyword(s):  
Adverse Events ◽  
Dose Escalation ◽  
Controlled Trial ◽  
Serum Urate ◽  
Extension Study ◽  
Open Label ◽  
Serious Adverse Events ◽  
Open Label Extension ◽  
Randomised Controlled ◽  
Kidney Impairment

ObjectivesTo determine the long-term safety and efficacy of allopurinol dose escalation (DE) to achieve target serum urate (SU) in gout.MethodsPeople, including those with chronic kidney disease, who completed the first 12 months of a randomised controlled trial continued into a 12-month extension study. Participants randomised to continue current dose for the first 12 months began allopurinol DE at month 12 if SU was ≥6 mg/dL (control/DE). Immediate DE participants who achieved target SU maintained allopurinol dose (DE/DE). The primary endpoints were reduction in SU and adverse events (AEs) at month 24.ResultsThe mean (SE) change in SU from month 12 to 24 was −1.1 (0.2) mg/dL in control/DE and 0.1 (0.2) mg/dL in DE/DE group (p<0.001). There was a significant reduction in the percentage of individuals having a gout flare in the month prior to months 12 and 24 compared with baseline in both groups and in mean tophus size over 24 months, but no difference between randomised groups. There were similar numbers of AEs and serious adverse events between groups.ConclusionsThe majority of people with gout tolerate higher than creatinine clearance-based allopurinol dose and achieve and maintain target SU. Slow allopurinol DE may be appropriate in clinical practice even in those with kidney impairment.Trial registration numberACTRN12611000845932

scholarly journals Achieving blood pressure control targets in hypertensive patients of rural China – A pilot randomized trial

2020 ◽  
Author(s):  
Xiao Huang ◽  
Lishun Liu ◽  
Yun Song ◽  
Lan Gao ◽  
Min Zhao ◽  
...  
Keyword(s):  
Adverse Events ◽  
Randomized Trial ◽  
Real World ◽  
Large Scale ◽  
Rural China ◽  
Standard Group ◽  
Open Label ◽  
Target Groups ◽  
Serious Adverse Events ◽  
Hypertensive Patients

Abstract Background This study aimed to test the feasibility and titration methods to achieve specific BP control targets in hypertensive patients of rural China. Methods A randomized, controlled, open-label trial was conducted in Rongcheng, China. We enrolled 105 hypertensive participants aged over 60 years, and who had no history of stroke and cardiovascular disease. The patients were randomly assigned to one of three systolic BP target groups: standard: 140 - < 150mmHg; moderately intensive: 130 - < 140mmHg; and intensive: <130mmHg. Patients were followed for 6 months. Discussion The optimal target for SBP lowering is still uncertain worldwide and such information is critically needed, especially in China. However, in China the rates of awareness, treatment and control are only 46.9%, 40.7% and 15.3%, respectively. It is challenging to achieve BP control in the real world and it is very important to develop population-specific BP control protocols that fully consider the population’s characteristics, such as age, sex, socio-economic status, compliance, education level and lifestyle. This randomized trial showed feasibility and safety of the titration protocol to achieve desirable SBP targets (<150, <140, and <130mmHg) in a sample of rural Chinese hypertensive patients. The three BP target groups had similar baseline characteristics. After 6 months of treatment, the mean SBP measured at an office visit was 137.2mmHg, 131.1mmHg, and 124.2mmHg in the three groups. Home BP and central aortic BP measurements were also obtained. At 6 months, home BP measurements (2 hours after drug administration) showed a mean SBP of 130.9 mmHg in the standard group, 124.9 mmHg in the moderately intense group, and 119.7 mmHg in the intensive group. No serious adverse events were recorded over the 6-month study period. Rates of adverse events including dry cough, palpitations, and arthralgia were low and showed no significant differences between the three groups. This trial gained real world experience and laid the foundation for a future large-scale BP target study.

scholarly journals Effect of Patiromer in Hyperkalemic Patients Taking and Not Taking RAAS Inhibitors

2018 ◽  
Vol 23 (6) ◽  
pp. 524-531 ◽  
Author(s):  
Robert A. Kloner ◽  
Coleman Gross ◽  
Jinwei Yuan ◽  
Ansgar Conrad ◽  
Pablo E. Pergola
Keyword(s):  
Adverse Events ◽  
Serum Potassium ◽  
Common Adverse Event ◽  
Open Label ◽  
Serious Adverse Events ◽  
End Point ◽  
The Mean ◽  
Baseline Characteristics ◽  
Raas Inhibitors ◽  
Post Hoc

Introduction: Hyperkalemia (potassium >5.0 mEq/L) affects heart failure patients with renal disease regardless of the use of renin–angiotensin–aldosterone system inhibitors (RAASi). The open-label TOURMALINE study showed that patiromer, a sodium-free, nonabsorbed potassium binder, lowers serum potassium of hyperkalemic patients similarly when given with or without food; unlike prior studies, patients were not required to be taking RAASi. We conducted post hoc analyses to provide the first report of patiromer in patients not taking RAASi. Methods: Hyperkalemic patients received patiromer, 8.4 g/d to start, adjusted to achieve and maintain serum potassium of 3.8 to 5.0 mEq/L. If taking RAASi, stable doses were required. The primary end point was the proportion of patients with serum potassium 3.8 to 5.0 mEq/L at week 3 or 4. This analysis presents data by patients taking or not taking RAASi. Results: Demographics and baseline characteristics were similar in patients taking (n = 67) and not taking RAASi (n = 45). Baseline mean (SD) serum potassium was 5.37 (0.37) mEq/L and 5.42 (0.43) mEq/L in patients taking and not taking RAASi, respectively. Mean (SD) daily patiromer doses were similar (10.7 [3.2] and 11.5 [4.0] g, respectively). The primary end point was achieved in 85% (95% confidence interval [CI]: 74-93) of patients taking RAASi and in 84% (95% CI: 71-94) of patients not taking RAASi. From baseline to week 4, the mean (SE) change in serum potassium was −0.67 (0.08) mEq/L in patients taking RAASi and −0.56 (0.10) mEq/L in patients not taking RAASi (both P < .0001 vs baseline, P = nonsignificant between groups). Adverse events were reported in 26 (39%) patients taking RAASi and 25 (54%) not taking RAASi; the most common adverse event was diarrhea (2% and 11%, respectively; no cases were severe). Five patients (2 taking RAASi) reported 6 serious adverse events; none considered related to patiromer. Conclusions: Patiromer was effective and generally well-tolerated for hyperkalemia treatment, whether or not patients were taking RAAS inhibitors.

Abstract 19503: Durability of Antiplatelet Effect of a Novel Extended-release Formulation of Acetylsalicylic acid, Durlaza in Patients With Diabetes

Circulation ◽  
2015 ◽  
Vol 132 (suppl_3) ◽  
Author(s):  
Paul A Gurbel ◽  
Kevin Blide P. P Bliden ◽  
Jeff Patrick Patrick ◽  
Katayoon Saadin Saadin ◽  
Udaya Tantry
Keyword(s):  
Adverse Events ◽  
Extended Release ◽  
Platelet Reactivity ◽  
Cvd Risk ◽  
Open Label ◽  
Serious Adverse Events ◽  
Release Formulation ◽  
Antiplatelet Effect ◽  
Immature Platelet Fraction ◽  
Induced Aggregation

Background: High platelet turnover (HPT) is implicated in incomplete platelet inhibition and high platelet reactivity (HPR) during immediate release aspirin therapy in type II diabetes patients (T2DM). Durlaza is a new, extended-release orally administered aspirin formulation developed to provide 24-hour antithrombotic effects with once-daily dosing. Methods: In this open-label, single-center study, T2DM patients (n=40) and a history of cardiovascular disease (CVD) or multiple CVD risk factors were treated with daily 162.5 mg Durlaza for 14±4 days and adverse events were collected. Antiplatelet effects were determined by conventional aggregation (LTA), Multiplate analyzer, thrombelastography with PlateletMapping, PlateletWorks ,VerifyNow Assay, and serum thromboxane B2 (TxB2) at 1, 12, 16, and 24 hrs after the last dose. HPT was defined as immature platelet fraction of ≥3.0% or MPV≥11.0 fl. Patients exhibiting HPT and/or HPR (based on previously published cutpoints in ≥2 assays) were treated with Durlaza at 325mg for 14± 4 days and platelet function testing was repeated. Results: Prevalence of HPT and HPR was 47% and 27%, respectively. There was no loss of antiplatelet effect at 12, 16 and 24 h versus 1 h by all assays (Table 1). All patients responded to 162.5mg Durlaza as measured by arachidonic acid-induced aggregation with LTA and platelet reactivity levels were low at all timepoints (Table). Serum TxB2 was lower at 12 h (p<0.03) as compared to 1 h after 162.5mg and was lower at all times with the 325 mg vs. the 162.5 mg Durlaza (p < 0.05). HPT did not affect the PD profile of Durlaza (Pts with HPT vs. no HPT, p=NS for all). Patients had no serious adverse events and low treatment related AE rate (<5%). Conclusion: In this first comprehensive assessment, a new, extended-release 162.5 Durlaza provided sustained antiplatelet effects over 24 h in T2DM patients with a favorable safety profile. Doubling the dose further lowered serum TxB2 in pts with HPT and/or HPR.

scholarly journals A phase I study of a dual PI3-kinase/mTOR inhibitor BEZ235 in adult patients with relapsed or refractory acute leukemia

2020 ◽  
Vol 21 (1) ◽  
Author(s):  
Fabian Lang ◽  
Lydia Wunderle ◽  
Susanne Badura ◽  
Eberhard Schleyer ◽  
Monika Brüggemann ◽  
...  
Keyword(s):  
Adverse Events ◽  
Acute Leukemia ◽  
Phase I ◽  
Mtor Inhibitor ◽  
Prolonged Treatment ◽  
Small Subset ◽  
Open Label ◽  
Serious Adverse Events ◽  
Efficient Treatment ◽  
Link Type

Abstract Background Combined inhibition of phosphatidylinositol 3-kinase (PI3K) and the mammalian target of rapamycin (mTOR) complexes may be an efficient treatment for acute leukemia. The primary objective of this phase I single center open label study was to determine the maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of the dual pan-class I PI3K and mTOR inhibitor BEZ235 in patients with advanced leukemia. Methods Herein patients > 18 years of age who had relapsed or showed refractory leukemia were treated with BEZ235 (orally at 300–400 mg BID (cohort − 1/1)) to assess safety, tolerability, preliminary efficacy and pharmacokinetic (PK). Adverse events data and serious adverse events were analyzed and haematological and clinical biochemistry toxicities were assessed from laboratory test parameters. Response was assessed for the first time at the end of cycle 1 (day 29) and after every subsequent cycle. Pharmacokinetic and pharmacodynamic analyses of BEZ235 were also included (BEZ235 plasma levels, phosphorylation of AKT, S6 and 4EBP1). On statistics this trial is a multiple ascending dose study in which a following variant of the 3 + 3 rule (“Rolling Six”), a minimum of 6 and a maximum of 12 patients was recruited for the dose escalation and another 5 were planned for the expansion phase. Results Twenty-four patients with ALL (n = 11) or AML (n = 12) or CML-BP (n = 1) were enrolled. All patients had failed one (n = 5) or more lines of therapy (n = 5) and 14 patients were in refractory / refractory relapse. No formal MTD was defined, stomatitis and gastrointestinal toxicity at 400 mg BID dose was considered incompatible with prolonged treatment. The RP2D of BEZ235 was defined as 300 mg BID. Four of 24 patients showed clinical benefit. Twenty-two of 24 patients discontinued because of progression, (median time to progression 27 days (4d-112d). There was no association between PK parameters and efficacy or tolerability. Conclusions Combined inhibition of PI3K and mTOR inhibits a clinically meaningful driver pathway in a small subset of patients with ALL, with no benefit in patients with AML. Trial registration ClinicalTrials.gov, identifier NCT01756118. retrospectively registered 19th December 2012, https://clinicaltrials.gov/ct2/show/NCT01756118.

scholarly journals Open-Label Clinical Trials of Oral Pulse Dexamethasone for Adults with Idiopathic Nephrotic Syndrome

2019 ◽  
Vol 49 (5) ◽  
pp. 377-385 ◽  
Author(s):  
Monique E. Cho ◽  
Mary H. Branton ◽  
David A. Smith ◽  
Linda Bartlett ◽  
Lilian Howard ◽  
...  
Keyword(s):  
Nephrotic Syndrome ◽  
Adverse Events ◽  
Idiopathic Nephrotic Syndrome ◽  
High Dose ◽  
Open Label ◽  
Serious Adverse Events ◽  
Oral Dexamethasone ◽  
Dose Oral ◽  
Pulse Dexamethasone ◽  
Daily Prednisone

Background: In adults with primary focal segmental glomerulosclerosis (FSGS), daily prednisone may induce complete remissions (CR) and partial remissions (PR), but relapses are frequent and adverse events are common. Methods: We carried out 2 open-label, uncontrolled trials to explore the efficacy and tolerability of pulse oral dexamethasone as an alternative to daily prednisone. We enrolled adult patients with proteinuria > 3.5 g/day despite the use of renin-angiotensin-aldosterone blockade. In the first trial, we enrolled 14 subjects with FSGS and administered 4 dexamethasone doses (25 mg/m2) daily for 4 days, repeated every 28 days over 32 weeks. The second trial involved a more intensive regimen. Eight subjects received 4 dexamethasone doses of 50 mg/m2 every 4 weeks for 12 weeks, followed by 4 doses of 25 mg/m2 every 4 weeks for 36 weeks; subjects were randomized to 2 doses every 2 weeks or 4 doses every 4 weeks. Results: In the first trial, we enrolled 13 subjects with FSGS and 1 with minimal change disease and found a combined CR and PR rate of 36%. In the second trial, we enrolled 8 subjects. The combined CR and PR rate was 29%. Analysis combining both trials showed a combined CR and PR rate of 33%. Adverse events were observed in 32% of subjects, with mood symptoms being most common. There were no serious adverse events related to the study. Conclusion: We conclude that high dose oral dexamethasone is well tolerated by adults with idiopathic nephrotic syndrome and may have some efficacy.

scholarly journals ACTR-68. FEASIBILITY STUDY OF THE EMULATE THERAPEUTICS™ VOYAGER SYSTEM IN PATIENTS WITH RECURRENT GLIOBLASTOMA (GBM)

2019 ◽  
Vol 21 (Supplement_6) ◽  
pp. vi29-vi29
Author(s):  
Garni Barkhoudarian ◽  
Michael Badruddoja ◽  
Nicholas Blondin ◽  
Ricky Chen ◽  
Sajeel Chowdhary ◽  
...  
Keyword(s):  
Overall Survival ◽  
Adverse Events ◽  
Skin Irritation ◽  
Recurrent Glioblastoma ◽  
Secondary Outcome ◽  
Open Label ◽  
Serious Adverse Events ◽  
Radio Frequency Energy ◽  
Long Term Treatment ◽  
Recurrent Gbm

Abstract BACKGROUND The EMulate Therapeutics Voyager system is an investigational non-sterile, non-invasive, non-thermal, non-ionizing, portable, home-use medical device that uses a specific, localized ultra-low radio frequency energy (ulRFE®) cognate for the treatment of brain cancer. METHODS This ongoing, open-label, multi-center study (NAT-101) is being conducted in the US and Australia in patients with recurrent GBM. There are 3 treatment groups: 32 patients treated with Voyager alone, 43 patients treated with Voyager + Investigator’s choice of anti-cancer therapy, and 21 patients treated with Voyager+lomustine+/-bevacizumab. The objective of the study is to assess if the Voyager is a safe and feasible treatment for recurrent GBM. The primary outcome measure is safety, assessed by the incidence and evaluation of adverse events (AEs) associated with the Voyager. The secondary outcome measures are progression-free survival and overall survival. RESULTS Enrollment is closed, and long-term treatment and follow-up is ongoing. 96 patients were enrolled and treated. 82 patients reported at least one AE, and 18 AEs were assessed as device-related (mild-moderate; 12 headache, 2 vomiting, 1 nausea, 1 confusion, 1 insomnia, and 1 skin irritation). 31 patients reported at least one serious AE, and none were assessed as device-related. 33% of patients treated with Voyager alone and 36% of patients treated with Voyager + chemotherapy were progression-free after 6 months. 58% of patients treated with Voyager alone and 60% of patients treated with Voyager + chemotherapy remained alive after 6 months; median overall survival is 7 months (95% CI=4.4±14.3) in patients treated with Voyager alone and 10 months (95% CI=6.7±11.5) in patients treated with Voyager + chemotherapy. CONCLUSIONS The Voyager system appears to be safe and feasible for the treatment of recurrent GBM. Given that therapy is delivered non-invasively and no device-related serious adverse events were reported, further prospective study of the investigational device is planned.

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