scholarly journals SP017ONE-YEAR FOLLOW-UP OF A PHASE I CLINICAL TRIAL ON THE USE OF BONE MARROW MESENCHYMAL STROMAL CELLS IN ADPKD PATIENTS

2016 ◽  
Vol 31 (suppl_1) ◽  
pp. i92-i92
Author(s):  
Atieh Makhlough ◽  
Soroosh Shekarchian ◽  
Reza Moghadasali ◽  
Seyedeh Esmat Hosseini ◽  
Nasser Aghdami
Keyword(s):  
Clinical Trial ◽  
Bone Marrow ◽  
Phase I ◽  
Mesenchymal Stromal Cells ◽  
Stromal Cells ◽  
Phase I Clinical Trial

scholarly journals Endobronchial autologous bone marrow-mesenchymal stromal cells in idiopathic pulmonary fibrosis (phase I)

2021 ◽  
pp. 00773-2020
Author(s):  
Arantza Campo ◽  
José María González-Ruiz ◽  
Enrique Andreu ◽  
Ana B. Alcaide ◽  
María M. Ocón ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Adverse Events ◽  
Phase I ◽  
Mesenchymal Stromal Cells ◽  
Stromal Cells ◽  
Second Phase ◽  
Endobronchial Administration

RationaleIdiopathic pulmonary fibrosis (IPF) has a dismal prognosis. Mesenchymal stromal cells have shown benefit in other inflammatory diseases.ObjectivesEvaluate the safety and feasibility of endobronchial administration of bone marrow autologous MSCs (BM-MSC) in patients with mild-to-moderate IPF.MethodsA phase-I multicenter clinical Trial (ClinicalTrials.gov:NCT01919827) with a single endobronchial administration of autologous adult BM-MSC in patients diagnosed with mild-to-moderate IPF. In a first escalating-dose phase, 3 patients will be included sequentially in 3 dose cohorts (10×10^6, 50×10^6, and 100×10^6 cells). In a second phase, 9 patients will receive the highest tolerated dose. Follow-up with PFT, 6MWT, and SGRQ were done at 1, 2, 3, 6, and 12 months, and with a computed tomography at 3, 6, and 12 months.FindingsTwenty-one bone marrow samples were obtained from 17 patients. Three patients were excluded for treatment due to chromosome aberrations detected in MSCs after culture, and one patient died before treatment. Finally, 13 patients received the BM-MSCs infusion. No treatment related severe adverse events were observed during follow-up. Compared to baseline, the mean FVC showed an initial decline of 8.1% at three months. The number of patients without functional progression was 6 (46%) at 3 months and 3 (23%) at 12 months.ConclusionsThe endobronchial infusion of BM-MSCs did not cause immediate serious adverse events in IPF patients, but a relevant proportion of patients suffered clinical and/or functional progression. Genomic instability of BM-MSCs during culture found in three patients may be troublesome for the use of autologous MSCs in IPF patients.

scholarly journals Intravenous Bone Marrow Mononuclear Cells for Acute Ischemic Stroke: Safety, Feasibility, and Effect Size from a Phase I Clinical Trial

Stem Cells ◽  
2019 ◽  
Vol 37 (11) ◽  
pp. 1481-1491 ◽  
Author(s):  
Farhaan S. Vahidy ◽  
Muhammad E. Haque ◽  
Mohammad H. Rahbar ◽  
Hongjian Zhu ◽  
Paul Rowan ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bone Marrow ◽  
Ischemic Stroke ◽  
Phase I ◽  
Acute Ischemic Stroke ◽  
Effect Size ◽  
Mononuclear Cells ◽  
Phase I Clinical Trial ◽  
Bone Marrow Mononuclear Cells

scholarly journals Lymphocyte depletion of donor bone marrow by counterflow centrifugal elutriation: results of a phase I clinical trial

Blood ◽  
1988 ◽  
Vol 72 (4) ◽  
pp. 1168-1176
Author(s):  
JE Wagner ◽  
AD Donnenberg ◽  
SJ Noga ◽  
CA Cremo ◽  
IK Gao ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bone Marrow ◽  
Acute Leukemia ◽  
Phase I ◽  
Disease Status ◽  
Phase I Clinical Trial ◽  
Myelogenous Leukemia ◽  
Allogeneic Bone ◽  
Centrifugal Elutriation ◽  
Early Relapse

We report here the results of a phase I clinical trial using counterflow centrifugal elutriation (CCE) for the removal of donor T lymphocytes before allogeneic bone marrow transplantation (BMT). Thirty- eight patients received lymphocyte-depleted allografts from HLA- identical, MLR-nonreactive sibling donors. The patients entered onto the study were either at high risk on the basis of age (median, 39 years) or disease status (acute leukemia in early relapse [ER], chronic myelogenous leukemia [CML] in accelerated phase [AP], or therapy resistant [RES] lymphoma). All patients received a standard lymphocyte dose of 1 x 10(6) morphologic lymphocytes per kilogram ideal body weight (BW) and were maintained on cyclosporine A (CsA) for 170 days after BMT. Prompt engraftment occurred in 37 of 38 patients with a median time to absolute neutrophil count (ANC) greater than 500/microL of 18 days. Although acute graft-v-host disease (GVHD; clinical stage I or greater) was observed in 45%, it was limited to the skin in all but five patients. Survival was related to disease status at the time of BMT. Among patients with acute leukemia in first or second remission, CML in chronic phase (CP) or lymphoma in partial remission (PR), 64% are currently alive, in contrast to 31% of patients with acute leukemia in third remission or early relapse, CML in second CP or AP, or RES lymphoma. Median follow-up for all patients was 351 days (range, 105 to 711 days). We conclude that this procedure is safe and warrants further evaluation in a randomized efficacy trial.

scholarly journals Phase II Multicenter Randomized Controlled Clinical Trial on the Efficacy of Intra-articular Injection of Autologous Bone Marrow Mesenchymal Stem Cells with Platelet Rich Plasma for the Treatment of Knee Osteoarthritis

2020 ◽  
Author(s):  
José María Lamo-Espinosa ◽  
Juan F. Blanco ◽  
Mikel Sánchez ◽  
Victoria Moreno ◽  
Froilán Granero-Moltó ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bone Marrow ◽  
Clinical Trials ◽  
Knee Osteoarthritis ◽  
Phase Ii ◽  
Stromal Cells ◽  
Platelet Rich Plasma ◽  
X Ray ◽  
The Mean

Abstract Background: Mesenchymal stromal cells are a safe and promising option to treat knee osteoarthritis as previously demonstrated in different clinical trials. However, their efficacy, optimal dose and addition of adjuvants must be determined. Here, we evaluated the clinical effects of a dose of 100x106 bone marrow mesenchymal stromal cells (BM-MSCs) in combination with Platelet Rich Plasma (PRGF®) as adjuvant in a randomized clinical trial.Methods: A phase II, multicenter, randomized clinical trial with active control was conducted. Sixty patients diagnosed with knee OA were randomly assigned to 3 weekly doses of PRGF® or intraarticular administration of 100x106 cultured autologous BM-MSCs plus PRGF®. Patients were followed up for 12 months, and pain and function were assessed using VAS and WOMAC and by measuring the knee range of motion range. X-ray and magnetic resonance imaging analyses were performed to analyze joint damage.Results: No adverse effects were reported after BM-MSC administration or during follow-up. According to VAS, the mean value (SD) for PRGF® and BM-MSC with PRGF® went from 5 (1.8) to 4.5 (2.2) (p=0.389) and from 5.3 (1.9) to 3.5 (2.5) (p=0.01), respectively at 12 months.. In WOMAC, the mean (SD) baseline and 12-month overall WOMAC scores in patients treated with PRGF® was 31.9 (16.2) and 22.3 (15.8) respectively (p=0.002) while that for patients treated with BM-MSC plus PRGF® was 33.4 (18.7) and 23.0 (16.6) (p=0.053). Although statistical significances between groups have been not detected, only patients being treated with BM-MSC plus PRGF® could be considered as a OA treatment responders following OARSI criteria. X-ray and MRI (WORMS protocol) revealed no changes in knee joint space width or joint damage.Conclusions: Treatment with BM-MSC associated with PRGF® was shown to be a viable therapeutic option for osteoarthritis of the knee, with clinical improvement at the end of follow-up. Further phase III clinical trials would be necessary to confirm the efficacy. Clinical Trials.gov identifier NCT02365142. Nº EudraCT: 2011-006036-23

Phase I study of lurbinectedin (PM11083) in patients with advanced AML and MDS.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18521-e18521 ◽  
Author(s):  
Christopher Brent Benton ◽  
Jose Rodriguez Diaz -Pavon ◽  
Abhishek Maiti ◽  
Naval Guastad Daver ◽  
Farhad Ravandi ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Bone Marrow ◽  
Phase I ◽  
Single Agent ◽  
Dna Minor Groove ◽  
Oral Herpes ◽  
Risk Patients ◽  
Md Anderson ◽  
Dose Levels

e18521 Background: The FDA-approved drug trabectedin is a DNA minor groove binder (MGB) with activity against translocation-associated sarcomas. Lurbinectedin is a next-generation MGB, with activity against myeloid leukemia cells. A phase I clinical trial was initiated to determine recommended doses in MDS/AML. Further assessment of its safety, tolerability, and pharmacogenetics were studied. Methods: In total, 42 patients with relapsed/refractory MDS/AML received lurbinectedin, using a 3+3 study design. It was administered as a 1-hour IV infusion first at 3.5, 5, 6, and 7mg per dose on days 1 and 8. Subsequently, it was administered on days 1, 2, and 3, using 1, 1.5, 2, or 3mg per dose per patient. Results: Three patients had dose-limiting toxicities (DLTs), which were rhabdomyolysis (up to gr 4), hyperbilirubinemia (gr 3), and oral herpes (gr 3). All DLTs occurred in the 6 and 7mg cohorts. Adverse events included febrile neutropenia/infections (n = 3), GI (n = 6) and pulmonary (n = 2) toxicity, hyperhidrosis (n = 1), and QT prolongation (n = 1). Overall, 33 of 42 patients (79%) had reduction of blasts in peripheral blood (PB) or bone marrow (BM) at nadir, including 23 (55%) with > 50% reduction in PB blasts alone (n = 18) or both BM and PB blasts (n = 5). One patient had PR, 2 patients had morphologic leukemia-free survival, and most (n = 30) were discontinued for progressive disease. Early deaths occurred from disease-related causes, not attributable to lurbinectedin. Thirty-two patients treated at MD Anderson were analyzed for clinical characteristics and responses. Notably, among patients with follow-up bone marrow biopsy, those with a cytogenetic abnormality at chromosome 11q21-23 had significantly greater bone marrow blast reduction than those without such abnormality (change in percentage blasts: -31±14% [n = 4] vs. 8±8% [n = 16], respectively; P= 0.04). Conclusions: Lurbinectedin is generally safe and tolerated at dose levels tested. While no sustained remissions > 3 cycles were observed in these highest-risk patients, single-agent lurbinectedin was transiently leukemia suppressive for some patients, including some with abnormal chr11q or TP53 mutation. Rational combinations and situational uses of lurbinectedin are under consideration. Clinical trial information: NCT01314599.

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