One Year Follow-Up for the Phase I MTD Study of Ultratrace Iobenguane I 131 in Patients With Malignant Pheochromocytoma/Paraganglioma (Pheo)

Abstract Context: The natural history of malignant pheochromocytoma or paragangliomas (MPP) remain unknown. Objective: The primary aim of this study was to define progression-free survival at 1 year in therapy-naive patients with MPP. Secondary objectives were to characterize MPP and to look for prognostic parameters for progression at 1 year. Design and Setting: The files of MPP followed up between January 2001 and January 2011 in two French Endocrine Networks were retrospectively reviewed. Therapy-naive patients were enrolled. Main Outcome Measures: The main outcome was progression-free survival at 1 year in therapy-naive MPP patients according to Response Evaluation Criteria In Solid Tumors 1.1 criteria. Results: Ninety files (46 men, 44 women, mean age of 47.5 ± 15 years) were reviewed on site by one investigator. MPP characteristics were as follows: presence of an adrenal primary, a mitotic count exceeding 5 per high power field, hypertension, inherited disease, and presence of bone metastases in 50%, 22%, 60%, 49%, and 56% patients, respectively. Fifty-seven of the 90 patients with MPP (63%) were classified as therapy-naive. The median follow-up of these 57 patients was 2.4 years (range, 0.4–5.7). At 1 year, progression-free survival was 46% (CI 95: 33–59). Twenty-six of 30 (87%) patients with progression at 1 year had exhibited progressive disease at the first imaging workup performed after a median of 5.7 months. No prognostic parameter was identified. Conclusions: Half of the therapy-naive patients with MPP achieved stable disease at 1 year. In symptom-free patients with MPP, a wait-and-see antitumor policy seems appropriate as first line. Modality for a prospective follow-up is proposed.

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Abstract 20536: ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration (ALLSTAR): the One Year Phase I Results

Circulation ◽

10.1161/circ.130.suppl_2.20536 ◽

2014 ◽

Vol 130 (suppl_2) ◽

Author(s):

Raj Makkar ◽

Richard Schatz ◽

Jay Traverse ◽

Andrew Hamer ◽

Katherine Beattie ◽

...

Keyword(s):

Phase I ◽

Infarct Size ◽

De Novo ◽

Acute Myocarditis ◽

Safety Data ◽

Left Ventricular ◽

Intracoronary Infusion ◽

Scar Size ◽

One Year

Introduction: In CADUCEUS, autologous cardiosphere-derived cells decreased infarct size and increased viable tissue in post-myocardial infarction (MI) subject s. Hypothesis: The first-in-human Phase I ALLSTAR trial was designed to test the safety and feasibility of intracoronary infusion of allogeneic cardiosphere-derived cells (CAP-1002) in patients with an anterior MI, within the prior 12 months with a scar size >15% by Magnetic Resonance Imaging (MRI). Methods: A total of 14 adult subjects (mean age = 55.6 yr; range 40-66 years) with a recent (28 - 90 days; n=9) or chronic (91-365 days; n=5) anterior MI (mean infarct size 25.4%; range 15.3% - 32.7%) and LV dysfunction (mean LVEF 42%; range 26.7% - 55.1%) were prospectively enrolled and infused with CAP-1002 (n=4 at 12.5M dose; n=10 at 25M dose) via stop-flow intracoronary infusion. Primary safety endpoints were: MACE events (recurrent MI, hospitalization or ER treatment for heart failure, Left Ventricular Assist Device placement or heart transplantation), acute myocarditis, death due to arrhythmias or unwitnessed death. Humoral and cellular immunologic responses were assessed via single antigen bead and ELISpot assays. Results: No pre-specified safety endpoint occurred. Only two adverse events were treatment-related, both transient hypotension related to nitroglycerin. There were no clinically significant rises in peri-procedural cardiac enzymes. Donor specific antibodies (DSAs) were present in four subjects prior to infusion; one resolved and three presistent at 6 months of follow up. De novo DSA’s developed in four subjects, three resolved during follow up and one persisted at 6 months of follow up. All DSA levels were low (MFI<5000). ELISpot revealed no de novo cellular immune responses. One year follow-up safety data as well as full 6 month and available 12 month MRI results on scar size and LVEF will be presented. Conclusions: Intracoronary infusion of allogeneic cardiosphere-derived cells (CAP-1002) appears to be safe and feasible. On the basis of the Phase 1 findings, the ALLSTAR trial has proceeded to a Phase II randomized, double-blind trial, powered to assess reduction of scar size by MRI.

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Safety, Immunogenicity and Duration of Protection of the RTS,S/AS02D Malaria Vaccine: One Year Follow-Up of a Randomized Controlled Phase I/IIb Trial

PLoS ONE ◽

10.1371/journal.pone.0013838 ◽

2010 ◽

Vol 5 (11) ◽

pp. e13838 ◽

Cited By ~ 25

Author(s):

Pedro Aide ◽

John J. Aponte ◽

Montse Renom ◽

Tacilta Nhampossa ◽

Jahit Sacarlal ◽

...

Keyword(s):

Phase I ◽

Malaria Vaccine ◽

Randomized Controlled ◽

One Year ◽

Duration Of Protection

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One year follow-up results of phase I/II clinical trial for combined treatment of chronic wounds with dermal equivalent-assisted skin autograft

Cytotherapy ◽

10.1016/j.jcyt.2017.03.066 ◽

2017 ◽

Vol 19 (5) ◽

pp. e24

Author(s):

Dmytro Zubov ◽

Roman Vasyliev ◽

Oleg Petrenko ◽

Georgy Kozynets ◽

Olga Gubar ◽

...

Keyword(s):

Clinical Trial ◽

Phase I ◽

Chronic Wounds ◽

Combined Treatment ◽

Dermal Equivalent ◽

One Year

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CTNI-66. ONE-YEAR FOLLOW-UP DATA OF PHASE I/II STUDY OF TIRABRUTINIB IN PATIENTS WITH RELAPSED OR REFRACTORY PRIMARY CENTRAL NERVOUS SYSTEM LYMPHOMA

Neuro-Oncology ◽

10.1093/neuonc/noaa215.232 ◽

2020 ◽

Vol 22 (Supplement_2) ◽

pp. ii57-ii58

Author(s):

Kazuhiko Mishima ◽

Yoshitaka Narita ◽

Motoo Nagane ◽

Yasuhito Terui ◽

Yoshiki Arakawa ◽

...

Keyword(s):

Central Nervous System ◽

Nervous System ◽

Phase I ◽

Kinase Inhibitor ◽

Central Nervous System Lymphoma ◽

Progression Free Survival ◽

Collaborative Group ◽

Pneumocystis Jirovecii Pneumonia ◽

One Year

Abstract Primary central nervous system lymphoma (PCNSL) is known as one of the incurable brain tumors. In March 2020, Tirabrutinib (TIR), a second-generation oral Bruton’s tyrosine kinase inhibitor, was approved for the indication of relapsed or refractory PCNSL (rrPCNSL) based on the results of a phase I/II study in Japan (Trial registration: JapicCTI-173646). In this study, 44 Japanese patients with rrPCNSL were treated with TIR QD at 320 mg, 480 mg, or 480 mg in the fasted condition (480 mg fasted QD). The primary endpoint was overall response rate (ORR) assessed by an independent review committee according to International PCNSL Collaborative Group criteria. We previously reported the results of this study with data cutoff on June 13, 2019 (Nagane et al., the 61st American Society of Hematology Annual Meeting and Exposition in 2019). In the report, 17 of 44 patients were treated with TIR at 480 mg fasted QD which is an approved dose, and had ORR of 52.9%, median progression-free survival of 5.8 months, and median overall survival of not reached (median follow-up: 3.8 months). In 44 patients, ORR was similar among patients harboring either of the oncogenic mutants CARD11, MYD88, CD79B, or wild type. Throughout the whole patients, most common adverse events (AEs) at any grade were rash (32%), neutropenia (23%), leukopenia (18%), and lymphopenia (16%), and grade ≥3 AEs were neutropenia (9.1%), lymphopenia, leukopenia, and erythema multiforme (6.8% each). One patient with 480 mg QD had grade 5 AEs (pneumocystis jirovecii pneumonia and interstitial lung disease). We will present one-year follow-up data of this study at the meeting. As of data cutoff (February 25, 2020), 11 of 44 patients continued to receive TIR, including 6 patients with 480 mg fasted QD. Updated data for OS, duration of response, and time to onset of AEs will also be presented.

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ML-05 One-year follow-up data of phase I/II study of tirabrutinib in patients with relapsed or refractory primary central nervous system lymphoma

Neuro-Oncology Advances ◽

10.1093/noajnl/vdaa143.068 ◽

2020 ◽

Vol 2 (Supplement_3) ◽

pp. ii16-ii16

Author(s):

Kazuhiko Mishima ◽

Yoshitaka Narita ◽

Motoo Nagane ◽

Yasuhito Terui ◽

Yoshiki Arakawa ◽

...

Keyword(s):

Overall Survival ◽

Phase I ◽

Kinase Inhibitor ◽

Central Nervous System Lymphoma ◽

Progression Free Survival ◽

Collaborative Group ◽

Survival Duration ◽

Pneumocystis Jirovecii Pneumonia ◽

One Year

Abstract In March 2020, Tirabrutinib (TIR), a second-generation oral Bruton’s tyrosine kinase inhibitor, was approved for the indication of relapsed or refractory PCNSL (r/rPCNSL) based on the results of a phase I/II study in Japan. In this study, 44 Japanese patients with r/rPCNSL were treated with TIR QD at 320 mg, 480 mg, or 480 mg in the fasted condition (480 mg fasted QD). The primary endpoint was overall response rate (ORR) assessed by an independent review committee according to International PCNSL Collaborative Group criteria. We previously reported the results of this study with data cutoff in June 2019 (Narita et al. Neuro Oncol. 2020). In the report, 17 of 44 patients were treated with TIR at 480 mg fasted QD which is an approved dose, and had ORR of 52.9%, median progression-free survival of 5.8 months, and median overall survival of not reached (median follow-up: 3.8 months). In 44 patients, ORR was similar among patients harboring either of the oncogenic mutants CARD11, MYD88, CD79B, or wild type. Throughout the whole patients, most common adverse events (AEs) at any grade were rash (31.8%), neutropenia (22.7%), leukopenia (18.2%), and lymphopenia (15.9%), and grade ≥3 AEs were neutropenia (9.1%), lymphopenia, leukopenia, and erythema multiforme (6.8% each). One patient with 480 mg QD had grade 5 AEs (pneumocystis jirovecii pneumonia and interstitial lung disease). We will present one-year follow-up data of this study at the meeting. As of data cutoff (February 2020), 11 of 44 patients continued to receive TIR, including 6 patients with 480 mg fasted QD. Updated data for overall survival, duration of response, and time to onset of AEs will also be presented. TIR is a promising new treatment for r/rPCNSL.

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