Clinical Research Directory

Optimization of the Time and Dosage of Vemurafenib in BRAF Positive Juvenile Patients With Refractory Histiocytosis

Prospective, interventional, open, randomized, single-center, non-commercial clinical trial to optimize treatment and dosage of vemurafenib in juvenile patients with histiocytosis resistant to conventional therapy and in whom the BRAF gene mutation has been found.

Optimization of the Time and Dosage of Trametinib in BRAF Negative Juvenile Patients

Prospective, interventional, open, randomized, single-center, non-commercial clinical trial to optimize treatment and dosage of trametinib in juvenile patients with histiocytosis resistant to conventional therapy and without the BRAF gene mutation or after the failure of vemurafenib treatment.

Determination of Molecular Status, the Efficacy and Safety of Fluorodeoxyglucose in PET-CT Imaging

Prospective, low intervention, open, single-center, non-commercial clinical trial to improve diagnostics in patients with histiocytosis by assessing the molecular profile of the tumor tissues, monitoring its presence in free-circulating DNA, and determining the efficacy of fluorodeoxyglucose (18F-FDG) in PET-CT imaging.

Phase 1/2 Trial of S241656 in Selected RAS/MAPK Mutation- Positive Malignancies

BDTX-4933-101 is a first-in-human, open-label, Phase 1/2 dose escalation, dose optimization and expansion study designed to evaluate the safety and tolerability of S241656 as monotherapy and in combination with other anti-cancer therapies in participants with selected advanced malignancies. The study population for the Dose Escalation part of the study comprises adults with recurrent advanced/metastatic non-small cell lung cancer (NSCLC), Gastrointestinal (GI) cancers, and other solid tumors harboring KRAS, HRAS, NRAS, BRAF, and/or CRAF (Rapidly Accelerated Fibrosarcoma (RAF1)) mutations or alterations. A dose optimization part in adults with NSCLC may follow the dose escalation phase if the sponsor, in consultation with the safety review committee, decides it is necessary to further characterize the optimal dose. However, the study may also proceed directly to the expansion phase. The study population for the Dose Expansion part of the study comprises adults with advanced/metastatic NSCLC with KRAS and/or BRAF mutations, and with Pancreatic Ductal AdenoCarcinoma (PDAC), ColoRectal Cancer (CRC), and Biliary Tract Cancer (BTC) with KRAS, HRAS, NRAS, BRAF, and/or CRAF (RAF1) mutations and alterations. All patients will self-administer S241656 orally in 28-day cycles until disease progression, toxicity, withdrawal of consent, or termination of the study.

Histiocytosis in Injecting Drug Users

The goal of this observational study is to describe a new disease condition of histiocytosis related to injecting drug use, its preconditions, symptoms, signs, findings, and prognosis in a detailed and systematic patient series in one referral center. The main question\[s\] it aims to answer are: * to find specific histologic features in different tissues to help the differential diagnosis from other histiocytoses * to describe the value of chitotriosidase activity to screen this condition * to describe the value of various tissue biopsies in confirming the diagnosis * to describe the nature of polyvinylpyrrolidone accumulation in tissue macrophages by novel special microscopic techniques * to find new tandem mass spectrometry methodology to prove polyvinylpyrrolidone accumulation in macrophages * to show that polyvinylpyrrolidone treatment activates macrophages to histiocytes and causes povidone accumulation within the cells in in vitro experiments * to evaluate the pathology of macrophage activation to histiocytes by transcriptomics The patient history will be collected from the data produced by follow-up of cases followed up in a single center. A subgroup of participants will be given an opportunity to sign informed consent to give access to/donate blood and tissue samples to search for techniques to prove polyvinylpyrrolidone storage within histiocytes, and to search for transcriptomics signal(s) in histiocytes. Researchers will compare blood and tissue samples from the biobank as controls.

Histiocytic Disorder Follow-up Study

The purpose of the study is to describe the burden of chronic health conditions, psychological dysfunction, chronic pain, healthcare utilization, worse health-related quality of life, overall mortality, and cause-specific mortality among individuals with histiocytic disorders

A Study of Memory, Thinking, and Brain Imaging in Adults With Histiocytosis

The purpose of this study is to try to understand how histopcytosis can cause symptoms or problems in the brain. The tests being done in the study will look at memory and thinking as well as brain function via MRI scan.

Adult Pulmonary Langerhans Cell Histiocytosis: a National Registry-based Prospective Cohort Study

The long-term outcomes of adult patients with pulmonary Langerhans cell histiocytosis (PLCH), particularly survival, is largely unknown. This is the first prospective study in the field evaluating the long-term outcomes of PLCH patients. This french countrywide registry-based study included a large cohort of PLCH patients followed for a sufficiently long period to address risk factors of long-term outcomes of PLCH patients.