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ShareScore release 0.9.0
Dataset results
16 results for “C3 glomerulopathy”
Study on Efficacy and Safety of LNP023 in C3 Glomerulopathy Patients Transplanted and Not Transplanted
ClinicalTrials.gov study NCT03832114. IPD Sharing: YES. Countries: 6. Publications: 1.
Dataset related to: Therapeutic Small Interfering RNA Targeting Complement C3 in a Mouse Model of C3 Glomerulopathy
<p>The files contain all the dataset included in the manuscript divided by figures.</p> <p> </p> <p>Abstract</p> <p>Alternative pathway complement dysregulation with abnormal glomerular C3 deposits and glomerular damage is a key mechanism of pathology in C3 glomerulopathy (C3G). No disease-specific treatments are currently available for C3G. Therapeutics inhibiting complement are emerging as a potential strategy for the treatment of C3G. In this study, we investigated the effects of N-acetylgalactosamine (GalNAc) conjugated small interfering RNA (siRNA) targeting the C3 component of complement that inhibits liver C3 expression in the C3G model of mice with heterozygous deficiency of factor H (Cfh+/- mice). We showed a duration of action for GalNAc-conjugated C3 siRNA in reducing the liver C3 gene expression in Cfh+/- mice that were dosed s.c. once a month for up to 7 mo. C3 siRNA limited fluid-phase alternative pathway activation, reducing circulating C3 fragmentation and activation of factor B. Treatment with GalNAc-conjugated C3 siRNA reduced glomerular C3d deposits in Cfh+/- mice to levels similar to those of wild-type mice. Ultrastructural analysis further revealed the efficacy of the C3 siRNA in slowing the formation of mesangial and subendothelial electron-dense deposits. The present data indicate that RNA interference mediated C3 silencing in the liver may be a relevant therapeutic strategy for treating patients with C3G associated with the haploinsufficiency of complement factor H.</p>
Controlled Trial Evaluating Avacopan in C3 Glomerulopathy
ClinicalTrials.gov study NCT03301467. IPD Sharing: YES. Countries: 11. Publications: 1.
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
ClinicalTrials.gov study NCT04817618. IPD Sharing: YES. Countries: 19. Publications: 1.
Safety Study of IgAN, LN, MN, & C3 Glomerulopathy Including Dense Deposit Disease Treated With OMS721
ClinicalTrials.gov study NCT02682407. IPD Sharing: Not stated. Countries: 2. Publications: 0.
A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)
ClinicalTrials.gov study NCT03369236. IPD Sharing: Not stated. Countries: 2. Publications: 1.
Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
ClinicalTrials.gov study NCT05067127. IPD Sharing: Not stated. Countries: 19. Publications: 1.
Dataset related to: Liver factor B silencing to cure C3 glomerulopathy: Evidence from a mouse model of complement dysregulation
<p>The files contain all the dataset included in the manuscript divided by figures.</p> <p>Abstract: Uncontrolled activation of the alternative pathway (AP) of complement, due to genetic and/or acquired defects, plays a primary pathogenetic role in C3 glomerulopathy (C3G), a rare and heterogeneous disease characterised by predominant C3 fragment deposition within the glomerulus, as well as glomerular damage. There are currently no approved disease-specific treatments for C3G, but new drugs that directly counteract AP dysregulation, targeting components of the pathway, have opened promising new perspectives for managing the disease. Complement factor B (FB), which is primarily synthesised by hepatocytes, is a key component of the AP, as it drives the central amplification loop of the complement system. In this study we used a GalNAc (N-Acetylgalactosamine)-conjugated siRNA to selectively target and suppress liver FB expression in two mouse models characterised by the complete (Cfh<sup>-/-</sup> mice) or partial (Cfh<sup>+/-</sup>) loss of function of complement factor H (FH). Homozygous deletion of FH induced a severe C3G phenotype, with strong dysregulation of the AP of complement, glomerular C3 deposition and almost complete C3 consumption. Mice with a heterozygous deletion of FH had intermediate C3 levels and exhibited slower disease progression, resembling human C3G more closely. Here we showed that FB siRNA treatment did not improve serum C3 levels, nor limit glomerular C3 deposition in Cfh<sup>-/-</sup> mice, while it did normalise circulating C3 levels, reduce glomerular C3 deposits, and limit mesangial electron-dense deposits in Cfh<sup>+/-</sup> mice. The present data provide important insights into the potential benefits and limitations of FB-targeted inhibition strategies and suggest RNA interference-mediated FB silencing in the liver as a possible therapeutic approach for treating C3G patients with FH haploinsufficiency.</p>
Non-contrast Enhanced MRI in Patients With C3 Glomerulopathy (C3G) or Immune-complex Membranoproliferative Glomerulonephritis (IC-MPGN) Enrolled in the ACH471-205 Study
ClinicalTrials.gov study NCT03723512. IPD Sharing: UNDECIDED. Countries: 1. Publications: 3.
An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative
ClinicalTrials.gov study NCT05809531. IPD Sharing: Not stated. Countries: 15. Publications: 0.
Evaluation of a Renin Inhibitor, Aliskiren, Compared to Enalapril, in C3 Glomerulopathy
ClinicalTrials.gov study NCT04183101. IPD Sharing: YES. Countries: 1. Publications: 3.
Genomic rearrangements in C3 glomerulopathies
GEO Series GSE45585. Homo sapiens. 1 samples. Type: Genome variation profiling by array.
TP10 Use in Patients With C3 Glomerulopathy (C3G)
ClinicalTrials.gov study NCT02302755. IPD Sharing: Not stated. Countries: 1. Publications: 0.
Study of NM8074 in Adult C3 Glomerulopathy Patients
ClinicalTrials.gov study NCT05647811. IPD Sharing: NO. Countries: 0. Publications: 0.
Clinical Outcomes of C3 Glomerulopathy and IC-MPGN in Russia: Hybrid Retrospective - Prospective Study
ClinicalTrials.gov study NCT06851845. IPD Sharing: NO. Countries: 0. Publications: 0.
Humanized C3 Mouse: A Novel Accelerated Model of C3 Glomerulopathy
GEO Series GSE150838. Mus musculus. 27 samples. Type: Expression profiling by high throughput sequencing.
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International Brain Laboratory public data
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OpenNeuro
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