scholarly journals VEROnA Protocol: A Pilot, Open-Label, Single-Arm, Phase 0, Window-of-Opportunity Study of Vandetanib-Eluting Radiopaque Embolic Beads (BTG-002814) in Patients With Resectable Liver Malignancies (Preprint)

2019 ◽  
Author(s):  
Laura Beaton ◽  
Henry F J Tregidgo ◽  
Sami A Znati ◽  
Sharon Forsyth ◽  
Matthew J Clarkson ◽  
...  
Keyword(s):  
Liver Metastases ◽  
Standard Of Care ◽  
Intermediate Stage ◽  
Window Of Opportunity ◽  
Current Standard ◽  
Open Label ◽  
Liver Malignancies ◽  
Surgical Specimen ◽  
Resected Liver ◽  
Phase 0

BACKGROUND Transarterial chemoembolization (TACE) is the current standard of care for patients with intermediate-stage hepatocellular carcinoma (HCC) and is also a treatment option for patients with liver metastases from colorectal cancer. However, TACE is not a curative treatment, and tumor progression occurs in more than half of the patients treated. Despite advances and technical refinements of TACE, including the introduction of drug-eluting beads-TACE, the clinical efficacy of TACE has not been optimized, and improved arterial therapies are required. OBJECTIVE The primary objectives of the VEROnA study are to evaluate the safety and tolerability of vandetanib-eluting radiopaque embolic beads (BTG-002814) in patients with resectable liver malignancies and to determine concentrations of vandetanib and the N-desmethyl metabolite in plasma and resected liver following treatment with BTG-002814. METHODS The VEROnA study is a first-in-human, open-label, single-arm, phase 0, window-of-opportunity study of BTG-002814 (containing 100 mg vandetanib) delivered transarterially, 7 to 21 days before surgery in patients with resectable liver malignancies. Eligible patients have a diagnosis of colorectal liver metastases, or HCC (Childs Pugh A), diagnosed histologically or radiologically, and are candidates for liver surgery. All patients are followed up for 28 days following surgery. Secondary objectives of this study are to evaluate the anatomical distribution of BTG-002814 on noncontrast-enhanced imaging, to evaluate histopathological features in the surgical specimen, and to assess changes in blood flow on dynamic contrast-enhanced magnetic resonance imaging following treatment with BTG-002814. Exploratory objectives of this study are to study blood biomarkers with the potential to identify patients likely to respond to treatment and to correlate the distribution of BTG-002814 on imaging with pathology by 3-dimensional modeling. RESULTS Enrollment for the study was completed in February 2019. Results of a planned interim analysis were reviewed by a safety committee after the first 3 patients completed follow-up. The recommendation of the committee was to continue the study without any changes to the dose or trial design, as there were no significant unexpected toxicities related to BTG-002814. CONCLUSIONS The VEROnA study is studying the feasibility of administering BTG-002814 to optimize the use of this novel technology as liver-directed therapy for patients with primary and secondary liver cancer. CLINICALTRIAL ClinicalTrial.gov NCT03291379; https://clinicaltrials.gov/ct2/show/NCT03291379 INTERNATIONAL REGISTERED REPORT DERR1-10.2196/13696

scholarly journals VEROnA Protocol: A Pilot, Open-Label, Single-Arm, Phase 0, Window-of-Opportunity Study of Vandetanib-Eluting Radiopaque Embolic Beads (BTG-002814) in Patients With Resectable Liver Malignancies

10.2196/13696 ◽  
2019 ◽  
Vol 8 (10) ◽  
pp. e13696
Author(s):  
Laura Beaton ◽  
Henry F J Tregidgo ◽  
Sami A Znati ◽  
Sharon Forsyth ◽  
Matthew J Clarkson ◽  
...  
Keyword(s):  
Liver Metastases ◽  
Standard Of Care ◽  
Intermediate Stage ◽  
Window Of Opportunity ◽  
Current Standard ◽  
Open Label ◽  
Liver Malignancies ◽  
Surgical Specimen ◽  
Resected Liver ◽  
Phase 0

Background Transarterial chemoembolization (TACE) is the current standard of care for patients with intermediate-stage hepatocellular carcinoma (HCC) and is also a treatment option for patients with liver metastases from colorectal cancer. However, TACE is not a curative treatment, and tumor progression occurs in more than half of the patients treated. Despite advances and technical refinements of TACE, including the introduction of drug-eluting beads-TACE, the clinical efficacy of TACE has not been optimized, and improved arterial therapies are required. Objective The primary objectives of the VEROnA study are to evaluate the safety and tolerability of vandetanib-eluting radiopaque embolic beads (BTG-002814) in patients with resectable liver malignancies and to determine concentrations of vandetanib and the N-desmethyl metabolite in plasma and resected liver following treatment with BTG-002814. Methods The VEROnA study is a first-in-human, open-label, single-arm, phase 0, window-of-opportunity study of BTG-002814 (containing 100 mg vandetanib) delivered transarterially, 7 to 21 days before surgery in patients with resectable liver malignancies. Eligible patients have a diagnosis of colorectal liver metastases, or HCC (Childs Pugh A), diagnosed histologically or radiologically, and are candidates for liver surgery. All patients are followed up for 28 days following surgery. Secondary objectives of this study are to evaluate the anatomical distribution of BTG-002814 on noncontrast-enhanced imaging, to evaluate histopathological features in the surgical specimen, and to assess changes in blood flow on dynamic contrast-enhanced magnetic resonance imaging following treatment with BTG-002814. Exploratory objectives of this study are to study blood biomarkers with the potential to identify patients likely to respond to treatment and to correlate the distribution of BTG-002814 on imaging with pathology by 3-dimensional modeling. Results Enrollment for the study was completed in February 2019. Results of a planned interim analysis were reviewed by a safety committee after the first 3 patients completed follow-up. The recommendation of the committee was to continue the study without any changes to the dose or trial design, as there were no significant unexpected toxicities related to BTG-002814. Conclusions The VEROnA study is studying the feasibility of administering BTG-002814 to optimize the use of this novel technology as liver-directed therapy for patients with primary and secondary liver cancer. Trial Registration ClinicalTrial.gov NCT03291379; https://clinicaltrials.gov/ct2/show/NCT03291379 International Registered Report Identifier (IRRID) DERR1-10.2196/13696

scholarly journals Management of patients with intermediate stage hepatocellular carcinoma

2020 ◽  
Vol 12 ◽  
pp. 175883592097084
Author(s):  
David Prince ◽  
Ken Liu ◽  
Weiqi Xu ◽  
Minjiang Chen ◽  
Jin-Yu Sun ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Standard Of Care ◽  
Intermediate Stage ◽  
Tumor Burden ◽  
Current Standard ◽  
Current Management ◽  
Heterogenous Group ◽  
Patient Subgroups ◽  
Significant Health ◽  
Systemic Therapies

Hepatocellular carcinoma (HCC) causes a significant health burden globally and its impact is expected to increase in the coming years. Intermediate stage HCC, as defined by the Barcelona Clinic Liver Cancer (BCLC) system stage B, represents up to 30% of patients at diagnosis and encompasses a broad spectrum of tumor burden. Several attempts have been made to further subclassify this heterogenous group. The current standard of care recommended by BCLC for intermediate stage HCC patients is transarterial chemoembolization (TACE), with modest outcomes reported. While refinements have been made to TACE technique and patient selection, it remains non-curative. In the real-world setting, only 60% of patients with intermediate stage HCC receive TACE, with the remainder deviating to a range of other therapies that have shown promise in select patient subgroups. These include curative treatments (resection, ablation, and liver transplantation), radiotherapy (stereotactic and radioembolization), systemic therapies, and their combination. In this review, we summarize the classifications and current management for patients with intermediate stage HCC as well as highlight recent key developments in this space.

scholarly journals Phase 0 and window of opportunity clinical trial design in neuro-oncology: a RANO review

2020 ◽  
Vol 22 (11) ◽  
pp. 1568-1579 ◽  
Author(s):  
Michael A Vogelbaum ◽  
Daria Krivosheya ◽  
Hamid Borghei-Razavi ◽  
Nader Sanai ◽  
Michael Weller ◽  
...  
Keyword(s):  
Brain Tumor ◽  
Trial Design ◽  
Human Study ◽  
Clinical Trial Design ◽  
Drug Level ◽  
New Drugs ◽  
Window Of Opportunity ◽  
Surgical Specimen ◽  
Number Of Patients ◽  
Phase 0

Abstract Glioblastoma is a devastating disease with poor prognosis. Few effective chemotherapeutics are currently available, and much effort has been expended to identify new drugs capable of slowing tumor progression. The phase 0 trial design was developed to facilitate early identification of promising agents for cancer that should undergo accelerated approval. This design features an early in-human study that enrolls a small number of patients who receive subtherapeutic doses of medication with the goals of describing pharmacokinetics through drug blood level measurements and determining intratumoral concentrations of the investigational compound as well as pharmacodynamics by studying the biochemical and physiological effects of drugs. In neuro-oncology, however, the presence of the blood–brain barrier and difficulty in obtaining brain tumor tissue warrant a separate set of considerations. In this paper, we critically reviewed the protocols used in all brain tumor related in-human phase 0 and phase 0–like (“window of opportunity”) studies between 1993 and 2018, as well as ongoing clinical trials, and identified major challenges in trial design as applied to central nervous system tumors that include surgical specimen collection and storage, brain tumor drug level analysis, and confirmation of drug action. We therefore propose that phase 0 trials in neuro-oncology should include (i) only patients in whom a resection of the tumor is planned, (ii) use of clinical doses of an investigational agent, (iii) tissue sampling from enhancing and non-enhancing portions of the tumor, and (iv) assessment of drug-specific target effects. Standardization of clinical protocols for phase 0/window of opportunity studies can help accelerate the development of effective treatments for glioblastoma.

scholarly journals High dose oral and intravenous rifampicin for improved survival from adult tuberculous meningitis: a phase II open-label randomised controlled trial (the RifT study)

2018 ◽  
Vol 3 ◽  
pp. 83 ◽  
Author(s):  
Fiona V. Cresswell ◽  
Kenneth Ssebambulidde ◽  
Daniel Grint ◽  
Lindsey te Brake ◽  
Abdul Musabire ◽  
...  
Keyword(s):  
Randomised Controlled Trial ◽  
Phase Ii ◽  
Controlled Trial ◽  
Standard Of Care ◽  
Hiv Positive ◽  
Current Standard ◽  
Open Label ◽  
Dose Oral ◽  
Randomised Controlled ◽  
Positive Population

Background: Tuberculous meningitis (TBM) has 44% (95%CI 35-52%) in-hospital mortality with standard therapy in Uganda. Rifampicin, the cornerstone of TB therapy, has 70% oral bioavailability and ~10-20% cerebrospinal fluid (CSF) penetration.  With current WHO-recommended TB treatment containing 8-12mg/kg rifampicin, CSF rifampicin exposures frequently fall below the minimal inhibitory concentration for M. tuberculosis. Two Indonesian phase II studies, the first investigating intravenous rifampicin 600mg and the second oral rifampicin ~30mg/kg, found the interventions were safe and resulted in significantly increased CSF rifampicin exposures and a reduction in 6-month mortality in the investigational arms. Whether such improvements can be replicated in an HIV-positive population remains to be determined. Protocol: We will perform a phase II, open-label randomised controlled trial, comparing higher-dose oral and intravenous rifampicin with current standard of care in a predominantly HIV-positive population. Participants will be allocated to one of three parallel arms (I:I:I): (i) intravenous rifampicin 20mg/kg for 2-weeks followed by oral rifampicin 35mg/kg for 6-weeks; (ii) oral rifampicin 35mg/kg for 8-weeks; (iii) standard of care, oral rifampicin 10mg/kg/day for 8-weeks. Primary endpoints will be: (i) pharmacokinetic parameters in plasma and CSF; (ii) safety. We will also examine the effect of higher-dose rifampicin on survival time, neurological outcomes and incidence of immune reconstitution inflammatory syndrome. We will enrol 60 adults with suspected TBM, from two hospitals in Uganda, with follow-up to 6 months post-enrolment. Discussion: HIV co-infection affects the bioavailability of rifampicin in the initial days of therapy, risk of drug toxicity and drug interactions, and ultimately mortality from TBM. Our study aims to demonstrate, in a predominantly HIV-positive population, the safety and pharmacokinetic superiority of one or both investigational arms compared to current standard of care. The most favourable dose may ultimately be taken forward into an adequately powered phase III trial. Trial registration: ISRCTN42218549 (24th April 2018)

First-line use of cabazitaxel in chemotherapy-naive patients with metastatic castration-resistant prostate cancer (mCRPC): A three-arm study in comparison with docetaxel.

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. TPS4696-TPS4696
Author(s):  
Stephane Oudard ◽  
Lisa Sengelov ◽  
Paul N. Mainwaring ◽  
Antoine Thiery- Vuillemin ◽  
Christine Theodore ◽  
...  
Keyword(s):  
Progression Free Survival ◽  
Standard Of Care ◽  
Phase Iii ◽  
Clinical Activity ◽  
Castration Resistant Prostate Cancer ◽  
First Line ◽  
Current Standard ◽  
Open Label ◽  
Measurable Disease ◽  
Ecog Ps

TPS4696^ Background: Docetaxel (D) in combination with prednisone (P) as first-line (1L) chemotherapy in patients (pts) with mCRPC is the current standard of care. However, treatment is not curative and D-resistant disease typically develops. Cabazitaxel (Cbz) is a novel taxane active in D-sensitive and -resistant tumor models. Clinical activity of Cbz plus P (CbzP) was demonstrated in the Phase III TROPIC study in mCRPC pts previously treated with a D-containing regimen; CbzP showed a significant overall survival (OS) benefit vs mitoxantrone plus prednisone (median OS 15.1 vs 12.7 months; HR 0.70; P < 0.0001). Therefore, it is of interest to determine if CbzP provides an OS advantage vs DP in 1L mCRPC pts. Methods: The phase III FIRSTANA study (NCT01308567) is a randomized, open-label, multinational trial in 1L mCRPC pts, designed to compare the efficacy of Cbz 25 mg/m² IV Q3W (Arm A) and Cbz 20 mg/m² IV Q3W (Arm B) vs D 75 mg/m2 IV Q3W (Arm C). P 10 mg PO QD is to be given concomitantly. Pts are stratified by ECOG PS (0–1 vs 2), measurable disease (yes/no) and region (depending on availability of Cbz as 2L). Pts with ECOG PS ≤ 2, histologically/cytologically confirmed metastatic prostate adenocarcinoma, with no prior chemotherapy and with disease progression following medical or surgical castration are eligible. The primary endpoint is OS. Secondary endpoints include progression-free survival (PFS) (PCWG2 criteria), radiologic PFS, tumor response in measurable disease (RECIST 1.1), PSA response and PSA PFS, pain response and pain PFS, time to occurrence of any skeletal-related events, safety profile and health-related quality of life. Cbz pharmacokinetics and pharmacogenomics will be assessed in pt subgroups. Pts will be treated until progression, unacceptable toxicity or pt request. Planned enrollment is 1,170 pts; study size was calculated to achieve 90% power for OS. Study start was in May 2011; at January 2012, 219 pts were enrolled. The first DMC meeting recommended continuing the study without change.

Phase III, Open-Label, Randomized Study Comparing Concurrent Gemcitabine Plus Cisplatin and Radiation Followed by Adjuvant Gemcitabine and Cisplatin Versus Concurrent Cisplatin and Radiation in Patients With Stage IIB to IVA Carcinoma of the Cervix

2011 ◽  
Vol 29 (13) ◽  
pp. 1678-1685 ◽  
Author(s):  
Alfonso Dueñas-González ◽  
Juan J. Zarbá ◽  
Firuza Patel ◽  
Juan C. Alcedo ◽  
Semir Beslija ◽  
...  
Keyword(s):  
External Beam Radiotherapy ◽  
Randomized Study ◽  
Locally Advanced ◽  
Progression Free Survival ◽  
Standard Of Care ◽  
Phase Iii ◽  
Karnofsky Performance Score ◽  
Current Standard ◽  
Open Label ◽  
Grade 3

Purpose To determine whether addition of gemcitabine to concurrent cisplatin chemoradiotherapy and as adjuvant chemotherapy with cisplatin improves progression-free survival (PFS) at 3 years compared with current standard of care in locally advanced cervical cancer. Patients and Methods Eligible chemotherapy- and radiotherapy-naive patients with stage IIB to IVA disease and Karnofsky performance score ≥ 70 were randomly assigned to arm A (cisplatin 40 mg/m2 and gemcitabine 125 mg/m2 weekly for 6 weeks with concurrent external-beam radiotherapy [XRT] 50.4 Gy in 28 fractions, followed by brachytherapy [BCT] 30 to 35 Gy in 96 hours, and then two adjuvant 21-day cycles of cisplatin, 50 mg/m2 on day 1, plus gemcitabine, 1,000 mg/m2 on days 1 and 8) or to arm B (cisplatin and concurrent XRT followed by BCT only; dosing same as for arm A). Results Between May 2002 and March 2004, 515 patients were enrolled (arm A, n = 259; arm B, n = 256). PFS at 3 years was significantly improved in arm A versus arm B (74.4% v 65.0%, respectively; P = .029), as were overall PFS (log-rank P = .0227; hazard ratio [HR], 0.68; 95% CI, 0.49 to 0.95), overall survival (log-rank P = .0224; HR, 0.68; 95% CI, 0.49 to 0.95), and time to progressive disease (log-rank P = .0012; HR, 0.54; 95% CI, 0.37 to 0.79). Grade 3 and 4 toxicities were more frequent in arm A than in arm B (86.5% v 46.3%, respectively; P < .001), including two deaths possibly related to treatment toxicity in arm A. Conclusion Gemcitabine plus cisplatin chemoradiotherapy followed by BCT and adjuvant gemcitabine/cisplatin chemotherapy improved survival outcomes with increased but clinically manageable toxicity when compared with standard treatment.

scholarly journals The CIPAZ study protocol: an open label randomised controlled trial of azithromycin versus ciprofloxacin for the treatment of children hospitalised with dysentery in Ho Chi Minh City, Vietnam

2020 ◽  
Vol 5 ◽  
pp. 214
Author(s):  
Thomas C. Darton ◽  
Tran Thi Hong Chau ◽  
Christopher M. Parry ◽  
James I. Campbell ◽  
Nguyen Minh Ngoc ◽  
...  
Keyword(s):  
Study Protocol ◽  
Bacillary Dysentery ◽  
Controlled Trial ◽  
Standard Of Care ◽  
Ho Chi Minh City ◽  
Ethical Review ◽  
Current Standard ◽  
Open Label ◽  
Children's Hospital ◽  
Children’S Hospital

Background: Diarrhoeal disease remains a common cause of illness and death in children <5 years of age. Faecal-oral infection by Shigella spp. causing bacillary dysentery is a leading cause of moderate-to-severe diarrhoea, particularly in low and middle-income countries. In Southeast Asia, S. sonnei predominates and infections are frequently resistant to first-line treatment with the fluoroquinolone, ciprofloxacin. While resistance to all antimicrobials is increasing, there may be theoretical and clinical benefits to prioritizing treatment of bacillary dysentery with the azalide, azithromycin. In this study we aim to measure the efficacy of treatment with azithromycin compared with ciprofloxacin, the current standard of care, for the treatment of children with bacillary dysentery. Methods and analysis: We will perform a multicentre, open-label, randomized controlled trial of two therapeutic options for the antimicrobial treatment of children hospitalised with dysentery. Children (6–60 months of age) presenting with symptoms and signs of dysentery at Children’s Hospital 2 in Ho Chi Minh City will be randomised (1:1) to treatment with either oral ciprofloxacin (15mg/kg/twice daily for 3 days, standard-of-care) or oral azithromycin (10mg/kg/daily for 3 days). The primary endpoint will be the proportion of treatment failure (defined by clinical and microbiological parameters) by day 28 (+3 days) and will be compared between study arms by logistic regression modelling using treatment allocation as the main variable. Ethics and dissemination: The study protocol (version 1.2 dated 27th December 2018) has been approved by the Oxford Tropical Research Ethics Committee (47–18) and the ethical review boards of Children's Hospital 2 (1341/NĐ2-CĐT). The study has also been approved by the Vietnamese Ministry of Health (5044/QĐ-BYT). Trial registration: Clinicaltrials.gov: NCT03854929 (February 26th 2019).

TACE 2: A randomized placebo-controlled, double-blinded, phase III trial evaluating sorafenib in combination with transarterial chemoembolisation (TACE) in patients with unresectable hepatocellular carcinoma (HCC).

2012 ◽  
Vol 30 (15_suppl) ◽  
pp. TPS4150-TPS4150 ◽  
Author(s):  
Tim Meyer ◽  
Richard Fox ◽  
Deborah Bird ◽  
Anthony Watkinson ◽  
Nigel Hacking ◽  
...  
Keyword(s):  
Study Drug ◽  
Progression Free Survival ◽  
Standard Of Care ◽  
Intermediate Stage ◽  
Primary Outcome Measure ◽  
Phase Iii ◽  
Secondary Outcome ◽  
Current Standard ◽  
Double Blind ◽  
Phase Iii Trial

TPS4150 Background: TACE is regarded as the standard of care for patients with intermediate stage HCC (BCLC B), while sorafenib (S) is the current standard for advanced disease (BCLC C). The combination of S with TACE for intermediate disease is rational since sorafenib inhibits the action of VEGF which may be induced by tumour hypoxia following TACE, and S also has direct anti-tumour effect. The combination has been shown to be safe in and active in phase I and II studies. Phase III trials are now required to determine whether TACE + S is superior to TACE alone in terms of survival. Methods: We are conducting a double blind multicentre trial to determine if the addition of S to TACE is superior to TACE alone. This is a UK NCRI trial sponsored by University College London (UCL) (EudraCT 2008-005073-36). Patients with intermediate stage HCC are randomised 1:1 to continuous S (400mg BD) or placebo (P). Key inclusion criteria include unresectable HCC confined to the liver, patent main portal vein, ECOG PS ≤1 and Child Pugh A liver score. After randomisation patients commence study drug and TACE is performed at 2-5 weeks using drug eluting beads loaded with 150mg doxorubicin (Biocompatibles UK Ltd) according to a standard protocol. Further TACE is performed at the discretion of the investigator based on radiological response and patient tolerance. The primary outcome measure is progression free survival and central, external, real time radiology review is required to confirm progression. Secondary outcome measures are overall survival, time to progression, toxicity, disease control, quality of life and number of TACE procedures performed in 12 months. The target recruitment is 412 in order to detect a hazard ratio of 0.72 using a 2-sided level of significance of α=0.05 with 85% power. The trial was reviewed by the IDMC after the presentation of the SPACE trial (Lencioni et al J Clin Oncol 30, 2012). The IDMC concurred with the conclusion of the SPACE investigators that the combination is tolerated and results required confirmation in a phase III trial. Thus recruitment into the TACE2 trial will continue as planned.

Phase Ib study of brentuximab vedotin (BV) with low-dose total skin electron beam (LD-TSEB) for treatment of mycosis fungoides (MF) and Sezary syndrome (SS).

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e20037-e20037
Author(s):  
Shiyu Song ◽  
Beata Holkova ◽  
Nitai Mukhopadhyay ◽  
Daeryl Williamson ◽  
Molly Dickinson
Keyword(s):  
Short Duration ◽  
Assessment Tool ◽  
Response Rate ◽  
Skin Toxicity ◽  
Standard Of Care ◽  
Skin Lesions ◽  
Brentuximab Vedotin ◽  
Current Standard ◽  
Open Label ◽  
Duration Of Response

e20037 Background: MF is generally not curable, causing severe symptoms and eventually leading to patient (pt) death. LD-TSEB is currently used for diffuse skin lesions but with short duration of response. BV is effective and approved for MF but also limited by short duration of response. In this study we proposed to evaluate the feasibility of combining these 2 treatments by assessing the safety and response rate/duration in these pts. Methods: Pts with stage IB - IVA CD30+ MF or SS who were candidates for TSEB were recruited for a 2-cohort open-label phase 1b trial. Cohort A included pts with earlier stage disease; cohort B included pts with more advanced disease. BV was given at 1.8 mg/kg 3 wks before initiation of TSEB and then every 3 wks. Pts in Cohort A received 3 doses of BV; pts in Cohort B continued BV until disease progression or unacceptable toxicity, up to 2 years. Standard 12 Gy TSEB was administered in 6 fractions (2/wk) beginning 3 wks after the first dose of BV. The Modified Severity Weighted Assessment Tool (mSWAT) was used to determine skin involvement at baseline and during and after treatment. Skindex-16 was used to assess pt-reported symptoms and toxicities. Pts were seen weekly during TSEB, monthly after TSEB, and every 3 months after the last dose of BV for response evaluation and time to progression (TTP). Results: Five pts were enrolled and treated in this study. Two were enrolled to Cohort A, and 3 were enrolled to Cohort B. One pt in Cohort B expired before completing study treatment due to comorbidity unrelated to MF. The other pt data is listed in the table. None of the pts developed grade 4 or 5 toxicities; pt 1 developed recurrent neuropathy and stopped BV after 5 cycles. Conclusions: As BV became approved for the indication, accrual slowed, and the trial was stopped with very few pts. These pt cohorts are heavily treated before trial entry, making conclusions regarding response hard to generalize. However, the combination of BV and TSEB is well tolerated with no significant increase in skin toxicity. Response rate seems comparable to current standard of care, though with no improvement in duration of response over BV alone. Clinical trial information: NCT02822586 . [Table: see text]

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