scholarly journals Safety and efficacy of intrarenal arterial autologous CD34 + cell transfusion in patients with chronic kidney disease: A randomized, open‐label, controlled phase II clinical trial

2020 ◽  
Vol 9 (8) ◽  
pp. 827-838
Author(s):  
Chih‐Chao Yang ◽  
Pei‐Hsun Sung ◽  
Ben‐Chung Cheng ◽  
Yi‐Chen Li ◽  
Yi‐Ling Chen ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Phase Ii ◽  
Phase Ii Clinical Trial ◽  
Open Label ◽  
Safety And Efficacy

scholarly journals P0182SAFETY AND EFFICACY OF INTRA-RENAL ARTERIAL AUTOLOGOUS CD34+ CELL TRANSFUSION IN PATIENTS WITH CHRONIC KIDNEY DISEASE: A RANDOMIZED, OPEN-LABEL, CONTROLLED PHASE II CLINICAL TRIAL

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Chihchao Yang ◽  
Yi-Chen Li ◽  
Honkan Yip
Keyword(s):  
Clinical Trial ◽  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Cell Therapy ◽  
Creatinine Clearance ◽  
Clinical Outcomes ◽  
Creatinine Level ◽  
Phase Ii ◽  
Open Label ◽  
One Year

Abstract Background and Aims This was a randomized, open-label, controlled phase II clinical trial to investigate the safety, efficacy and the outcomes of intra-renal artery (RA) infusion of autologous peripheral-blood-derived CD34+ cells for chronic kidney disease (CKD) (i.e., stage III or IV) patients. Method Between December 2016 and November 2018, 52 consecutive patients with CKD at stage III or IV were randomly allocated into treatment group (TG) (2.5 × 107 cells for each RA; n=26) and control group (CG) (standardized pharmacotherapy only; n=26). The primary endpoints included safety and change of creatinine level/creatinine clearance (Ccr). The secondary endpoints were 12-month combined unfavorable clinical outcomes (defined as dialysis or death), improvement in proteinuria, and CD34+cell-related adverse events. Results All patients were uneventfully discharged after CD34+ cell therapy. Flow cytometric analysis showed increases in circulating EPC (i.e., CD34+KDR+CD45dim/CD34+CD133+ CD45dim/CD31+CD133+CD45dim/CD34+CD133+KDR+/CD133+) and hematopoietic stem cell (HSC) (CD34+) populations after G-CSF treatment. Besides, Matrigel assay of angiogenesis was also significantly enhanced (all p<0.001). Renal vein blood samplings (i.e., at 0/5/10/30 min after CD34+ cell infusion) demonstrated significant progressive increases in EPC level (p for trend<0.001) among the TG patients. The baseline EPC populations did not differ between TG and CG (p>0.5). One-year combined unfavorable clinical outcomes were significantly lower in TG than those in CG [0% (0) vs. 13.3% (4), p=0.038](Figure). By 12-month after CD34+ cell therapy, circulating creatinine level, ratio of urine protein to urine creatinine, and creatinine clearance did not differ between TG and CG (all p>0.1). Conclusion CD34+ cell therapy was safe and improved one-year outcome.

REGOMA: A Randomized, Multicenter, Controlled Open-Label Phase II Clinical Trial of Regorafenib Compared to Lomustine in Relapsed Glioblastoma Patients

2018 ◽  
Author(s):  
Giuseppe Lombardi ◽  
Gian Luca De Salvo ◽  
Alba Ariela Brandes ◽  
Marica Eoli ◽  
Roberta Rudà ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Phase Ii ◽  
Phase Ii Clinical Trial ◽  
Open Label ◽  
Open Label Phase

scholarly journals Safety and efficacy of iron isomaltoside 1000/ferric derisomaltose versus iron sucrose in patients with chronic kidney disease: the FERWON-NEPHRO randomized, open-label, comparative trial

2020 ◽  
Vol 36 (1) ◽  
pp. 111-120 ◽  
Author(s):  
Sunil Bhandari ◽  
Philip A Kalra ◽  
Mario Berkowitz ◽  
Diogo Belo ◽  
Lars L Thomsen ◽  
...  
Keyword(s):  
Chronic Kidney Disease ◽  
Single Dose ◽  
Kidney Disease ◽  
Iron Deficiency ◽  
Iron Sucrose ◽  
Hypersensitivity Reactions ◽  
Open Label ◽  
Pronounced Increase ◽  
Safety And Efficacy ◽  
Significant Difference

Abstract Background The optimal intravenous (IV) iron would allow safe correction of iron deficiency at a single infusion over a short time. The FERWON-NEPHRO trial evaluated the safety and efficacy of iron isomaltoside 1000/ferric derisomaltose (IIM) in patients with non-dialysis-dependent chronic kidney disease and iron deficiency anaemia. Methods In this randomized, open-label and multi-centre trial conducted in the USA, patients were randomized 2:1 to a single dose of 1000 mg IIM or iron sucrose (IS) administered as 200 mg IV injections up to five times within a 2-week period. The co-primary endpoints were serious or severe hypersensitivity reactions and change in haemoglobin (Hb) from baseline to Week 8. Secondary endpoints included incidence of composite cardiovascular adverse events (AEs). Results A total of 1538 patients were enrolled (mean estimated glomerular filtration rate 35.5 mL/min/1.73 m2). The co-primary safety objective was met based on no significant difference in the incidence of serious or severe hypersensitivity reactions in the IIM and IS groups [0.3% versus 0%; risk difference: 0.29% (95% confidence interval: –0.19; 0.77; P > 0.05)]. Incidence of composite cardiovascular AEs was significantly lower in the IIM versus IS group (4.1% versus 6.9%; P = 0.025). Compared with IS, IIM led to a more pronounced increase in Hb during the first 4 weeks (P ≤ 0.021), and change in Hb to Week 8 showed non-inferiority, confirming that the co-primary efficacy objective was met. Conclusions Compared with multiple doses of IS, a single dose of IIM induced a non-inferior 8-week haematological response, comparably low rates of hypersensitivity reactions, and a significantly lower incidence of composite cardiovascular AEs.

scholarly journals OS2.3 Updated results of REGOMA: A randomized, multicenter, controlled open-label phase II clinical trial evaluating regorafenib in relapsed glioblastoma patients

2018 ◽  
Vol 20 (suppl_3) ◽  
pp. iii219-iii219
Author(s):  
G Lombardi ◽  
G de Salvo ◽  
R Rudà ◽  
E Franceschi ◽  
M Eoli ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Phase Ii ◽  
Phase Ii Clinical Trial ◽  
Open Label ◽  
Open Label Phase

Dual immune checkpoint blockade in advanced well-differentiated neuroendocrine tumors, a phase II clinical trial.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e16201-e16201
Author(s):  
Susan Combs Scott ◽  
Ana De Jesus-Acosta ◽  
Chen Hu ◽  
Benjamin Philip Levy ◽  
Valsamo Anagnostou ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Neuroendocrine Tumors ◽  
Phase Ii ◽  
Median Duration ◽  
Primary Endpoint ◽  
Response Rate ◽  
Objective Response ◽  
Phase Ii Clinical Trial ◽  
Open Label ◽  
Well Differentiated

e16201 Background: Limited systemic treatment options are available for progressive well-differentiated neuroendocrine tumors (NET), also called carcinoid tumors. Given emerging evidence for immunotherapy response in high grade NET including small cell lung cancer, we sought to determine the efficacy of combination immunotherapy with ipilimumab and nivolumab in patients with advanced, progressive, well-differentiated NET in an open label phase II clinical trial. Methods: Eligible patients had well-differentiated, nonfunctional NET of lung, pancreas, or GI origin that had progressed within the past 12 months after at least one line of prior therapy. Patients received nivolumab 240 mg every 2 weeks and ipilimumab 1mg/kg every 6 weeks for up to 2 years. Primary endpoint was objective response rate (ORR) by RECIST v1.1. Using a Simon’s 2-stage design, the study planned to accrue up to 56 patients. Based on published response rates to everolimus of 5%, we hypothesized that this regimen would be considered promising if the true ORR is > 15%. Results: Nine patients were enrolled prior to study closure due to funding, including 6 patients with NET of lung origin, 2 pancreatic, and 1 small bowel (Table). Median age was 71 years. All patients had distant metastatic disease at enrollment, with an average of 2 prior lines of therapy. Four of 9 patients achieved the primary endpoint of confirmed objective response, all of whom have ongoing response with a median duration of 15.4 months. Five of 9 patients, including all 4 responders, experienced immune-related toxicity requiring treatment modification or discontinuation. The trial did not accrue the target of 56 patients, however, objective response in 4 of 9 patients (ORR 44.4%, 90% CI: 16.9-74.9%) excluded the response rate target (15%). Conclusions: The impressive ORR of 44% with a median duration of response exceeding 15 months in this small clinical trial warrants further study of combination CTLA-4 and PD-1 inhibition in previously treated well-differentiated NET. Our ongoing immunologic and genomic correlative analysis in responders and non-responders will help inform future study of immunotherapy in this patient population in need of new systemic therapy approaches. Clinical trial information: NCT03420521. [Table: see text]

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