Erdheim-Chester Disease Clinical Trial
Official title:
Open-label, Single-arm, Phase II, Prospective, Pilot Study of Tocilizumab in Patients With Erdheim-Chester Disease
The study we propose is a pilot phase II, interventional, treatment, open-label, single-arm,
efficacy/safety clinical trial of a 6-month treatment with tocilizumab (8 mg/kg once
monthly) in adult patients with extraskeletal Erdheim-Chester disease (ECD). Efficacy will
be assessed as the effect of the treatment on the size of the measurable lesions, as
evaluated by bidimensional measurements, and by the impact of the treatment on symptom
control and on patient quality of life. Safety of the treatment will be determined by the
analysis of adverse events and of the relevant safety laboratory parameters.
Secondary objectives of the study will be:
1. to better characterize the mechanisms underlying such disease and the possible response
to the treatment. In particular: i) we will investigate the immunophenotypic and
histomorphologic features of ECD histiocytes; ii) we will gather gene expression data
from peripheral blood immune cells to better characterize their functional status, to
define their transcriptional fingerprints and their possible modulation as a result of
tocilizumab treatment; iii) we will assess the production of soluble mediators and the
expression of activation molecules by monocytes derived from ECD patients, as well as
after stimulation with selected inflammatory cytokines; iv) we will investigate the ex
vivo and in vitro impact of tocilizumab treatment on those markers, for possible use as
a predictor or indicator of response to treatment.
2. to investigate the metabolic pattern in ECD patients before and after tocilizumab
treatment as evaluated by fluoro-d-glucose positron emission tomography (FDG-PET) and
to verify if it can be an indicator of ECD activity.
n/a
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT06317246 -
Subtypes and Prognostic Factors in Erdheim-Chester Disease
|
N/A | |
| Recruiting |
NCT05915208 -
Histiocytic Disorder Follow-up Study
|
||
| Recruiting |
NCT03329274 -
Registry for Patients With Erdheim-Chester Disease and Other Histiocytoses
|
||
| Recruiting |
NCT05093335 -
In-Human CXCR4 Imaging of Blood Cancers Using [68Ga]-Pentixafor-PET
|
Early Phase 1 | |
| Completed |
NCT02608619 -
Uptake and Biodistribution of 18F-fluorocholine in Histiocytic Disorders by PET Imaging and Biopsy Measurement
|
||
| Recruiting |
NCT02089724 -
Long-term Outcome After Vemurafenib / BRAF Inhibitors Interruption in Erdheim-chester Disease
|
N/A | |
| Active, not recruiting |
NCT01552434 -
Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease
|
Phase 1 | |
| Recruiting |
NCT05768178 -
DETERMINE Trial Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.
|
Phase 2/Phase 3 | |
| Recruiting |
NCT06332183 -
GWAS and EWAS in Patients With Erdheim-Chester Disease
|
||
| Terminated |
NCT04198818 -
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors
|
Phase 1/Phase 2 | |
| Withdrawn |
NCT03794297 -
Dabrafenib and Trametinib in Treating Patients With Erdheim Chester Disease With BRAF V600 Mutations
|
Phase 2 | |
| Active, not recruiting |
NCT05092815 -
The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
|
Phase 2 | |
| Recruiting |
NCT04079179 -
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
|
Phase 2 |