scholarly journals An Observational, Non-Interventional Study for the Follow-up of Patients with Amyloidosis Who Received Miridesap Followed by Dezamizumab in a Phase 1 Study

2021 ◽  
Author(s):  
Duncan Richards ◽  
Helen Millns ◽  
Louise Cookson ◽  
Mary Ann Lukas
Keyword(s):  
Phase 1 ◽  
Human Study ◽  
Disease Status ◽  
Serum Amyloid ◽  
Response To Treatment ◽  
Interventional Study ◽  
Amyloid A ◽  
Amyloid Deposits ◽  
Long Term Follow Up

Abstract Background: Miridesap depletes circulating serum amyloid P (SAP) and dezamizumab (anti-SAP monoclonal antibody) targets SAP on amyloid deposits, triggering amyloid removal. In a Phase 1, first-in-human study (FIHS), progressive amyloid removal was observed in some patients after ≤3 cycles of miridesap/dezamizumab.Methods: This observational, non-interventional study in patients who received miridesap/dezamizumab during the FIHS (planned follow-up: 5 years) evaluated response to treatment based on routine assessments of disease status and key organ function. In a post hoc analysis, patients responding to treatment in the FIHS during follow-up were identified as responders and further categorized as sustained or declining responders.Results: In the FIHS, 17/23 patients were treatment responders. Of these patients, seven (immunoglobulin light chain [AL], n=6; serum amyloid A, n=1) were considered sustained responders and ten (fibrinogen-a alpha chain [AFib], n=5; AL, n=4; apolipoprotein A-I, n=1) were considered declining responders. We primarily present responder patient-level data for functional, cardiac, laboratory and imaging assessments conducted during the follow-up period, with non-responder data presented as supplementary.Conclusion: No further development of miridesap/dezamizumab is planned in amyloidosis. However, long-term follow-up of these patients may provide insight into whether active removal of amyloid deposits has an impact on disease progression. Trial registration: ClinicalTrials.gov, NCT01777243. Registered 28 January 2013, https://clinicaltrials.gov/ct2/show/study/NCT01777243.

scholarly journals Long-term Follow-up of Patients with Relapsed or Refractory Non-Hodgkin Lymphoma Treated with Venetoclax in a Phase 1, First-in-Human Study

2021 ◽  
pp. clincanres.4842.2020
Author(s):  
Matthew S. Davids ◽  
Andrew W. Roberts ◽  
Vaishalee P. Kenkre ◽  
William G. Wierda ◽  
Abhijeet Kumar ◽  
...  
Keyword(s):  
Hodgkin Lymphoma ◽  
Phase 1 ◽  
Human Study ◽  
Non Hodgkin Lymphoma ◽  
Long Term Follow Up

Intestinal spirochetosis and chronic watery diarrhea: Clinical and histological response to treatment and long-term follow up

2006 ◽  
Vol 21 (8) ◽  
pp. 1326-1333 ◽  
Author(s):  
Maria Esteve ◽  
Antonio Salas ◽  
Fernando Fernandez-Banares ◽  
Josep Lloreta ◽  
Meritxell Marine ◽  
...  
Keyword(s):  
Response To Treatment ◽  
Watery Diarrhea ◽  
Histological Response ◽  
Intestinal Spirochetosis ◽  
Long Term Follow Up

scholarly journals Amyloid deposition in granuloma of tuberculosis patients: A pilot study

2021 ◽  
Author(s):  
Shreya Ghosh ◽  
Akansha Garg ◽  
Chayanika Kala ◽  
Ashwani Kumar Thakur
Keyword(s):  
Serum Amyloid A ◽  
Amyloid Deposition ◽  
Serum Amyloid ◽  
Secondary Amyloidosis ◽  
Amyloid Formation ◽  
Tuberculosis Patients ◽  
Amyloid A ◽  
Inflammatory Conditions ◽  
Amyloid Deposits ◽  
Serum Amyloid A Protein

AbstractThe formation of granuloma is one of the characteristic feature of tuberculosis. Besides, rise in the concentration of acute phase response proteins mainly serum amyloid A is the indicator for chronic inflammation associated with tuberculosis. Serum amyloid A drives secondary amyloidosis in tuberculosis and other chronic inflammatory conditions. The linkage between serum amyloid A (SAA) protein and amyloid deposition site is not well understood in tuberculosis and other chronic inflammatory conditions. We hypothesized that granuloma could be a potential site for amyloid deposition because of the presence of serum amyloid A protein and proteases that cleave SAA and trigger amyloid formation. Based on this hypothesis, for the first time we have shown the presence of amyloid deposits in the granuloma of tuberculosis patients using the gold standard, Congo red dye staining.

scholarly journals Minimal residual hairy cell leukemia eradication with moxetumomab pasudotox: phase 1 results and long-term follow-up

Blood ◽  
2018 ◽  
Vol 131 (21) ◽  
pp. 2331-2334 ◽  
Author(s):  
Robert J. Kreitman ◽  
Martin S. Tallman ◽  
Tadeusz Robak ◽  
Steven Coutre ◽  
Wyndham H. Wilson ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Aspirate ◽  
Phase 1 ◽  
Hairy Cell ◽  
Cell Leukemia ◽  
Key Points ◽  
Long Term Follow Up ◽  
Minimal Residual

Key Points Moxetumomab pasudotox eradicated HCL MRD in >50% of CRs, even by the most sensitive measure, bone marrow aspirate flow cytometry. Elimination of MRD was significantly associated with prolonged CR duration.

scholarly journals Serum Amyloid A, Procalcitonin, Tumor Necrosis Factor-α, and Interleukin-1βLevels in Neonatal Late-Onset Sepsis

2008 ◽  
Vol 2008 ◽  
pp. 1-7 ◽  
Author(s):  
Birsen Ucar ◽  
Bilal Yildiz ◽  
M. Arif Aksit ◽  
Coskun Yarar ◽  
Omer Colak ◽  
...  
Keyword(s):  
Serum Amyloid A ◽  
Late Onset ◽  
Serum Amyloid ◽  
Amyloid A ◽  
Late Onset Sepsis ◽  
Sepsis Score ◽  
Reliable Marker ◽  
Factor Α ◽  
Necrosis Factor

Background. Sepsis is an important cause of mortality in newborns. However, a single reliable marker is not available for the diagnosis of neonatal late-onset sepsis (NLS). The aim of this study is to evaluate the value of serum amyloid A (SAA) and procalcitonin (PCT) in the diagnosis and follow-up of NLS.Methods. 36 septic and healthy newborns were included in the study. However, SAA, PCT, TNF-α, IL-1β, and CRP were serially measured on days 0, 4, and 8 in the patients and once in the controls. Töllner's sepsis score (TSS) was calculated for each patient.Results. CRP, PCT, and TNF-αlevels in septic neonates at each study day were significantly higher than in the controls (P=.001). SAA and IL-1βlevels did not differ from healthy neonates. The sensitivity and specificity were 86.8% and 97.2% for PCT, 83.3% and 80.6% for TNF-α, 75% and 44.4% for SAA on day 0.Conclusion. Present study suggests that CRP seems to be the most helpful indicator and PCT and TNF-αmay be useful markers for the early diagnosis of NLS. However, SAA, IL-1β, and TSS are not reliable markers for the diagnosis and follow-up of NLS.

scholarly journals Safety and Pharmacokinetics of a Four Monoclonal Antibody Combination against Botulinum C and D Neurotoxins

2019 ◽  
Vol 63 (12) ◽  
Author(s):  
Doris M. Snow ◽  
Kathryn Riling ◽  
Angie Kimbler ◽  
Yero Espinoza ◽  
David Wong ◽  
...  
Keyword(s):  
Phase 1 ◽  
Human Study ◽  
Amino Acid Sequences ◽  
High Potency ◽  
Human Mabs ◽  
Tier 1 ◽  
Physical Examinations ◽  
Human Igg1 ◽  
Antidrug Antibodies

ABSTRACT Botulism is caused by botulinum neurotoxin (BoNT), the most poisonous substance known. BoNTs are also classified as tier 1 biothreat agents due to their high potency and lethality. The existence of seven BoNT serotypes (A to G), which differ by 35% to 68% in amino acid sequences, necessitates the development of serotype-specific countermeasures. We present results of a phase 1 clinical study of an anti-toxin to BoNT serotypes C and D, NTM-1634, which consists of an equimolar mixture of four fully human IgG1 monoclonal antibodies (MAbs), each binding to nonoverlapping epitopes on BoNT serotypes C and D, resulting in potent toxin neutralization in rodents. This first in-human study evaluated the safety and pharmacokinetics of escalating doses of NTM-1634 administered intravenously to healthy adults. Three cohorts of eight healthy subjects received single intravenous doses of NTM-1634 at 0.33 mg/kg, 0.66 mg/kg, or 1 mg/kg or placebo. Follow-up examinations and pharmacokinetics evaluations were continued up to 121 days postinfusion. Subjects were monitored by using physical examinations, hematology and chemistry blood tests, and electrocardiograms. Pharmacokinetics parameters were estimated using noncompartmental methods. The results demonstrated that the materials were safe and well tolerated with the expected half-lives for human MAbs and with minimal antidrug antibodies detected over the dose ranges and duration of the study. (This study has been registered at ClinicalTrials.gov under identifier NCT03046550.)

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