Long-term Outcome After Vemurafenib / BRAF Inhibitors Interruption in Erdheim-chester Disease
Status unconfirmed
Conditions studied: Erdheim-Chester Disease
In brief
Erdheim-Chester disease (ECD) is a rare non-Langerhans cell histiocytosis. Diagnosis of ECD is based on clinical symptoms, imaging and histology with infiltration of tissues by foamy CD68 positive CD1a negative histiocytes. Because half of the ECD patients carry a BRAFV600E mutation, we recently proposed vemurafenib, an inhibitor of mutant BRAF, as a possible targeted therapy. We have treated more than10 patients with refractory ECD with life-threatening manifestations associated with the BRAFV600E mutation and observed a short and long term efficacy. However, vemurafenib may have several side effects and long term administration of this drug has not been evaluated. In other diseases such as melanoma, duration of administration is usually shorter, due to bad prognosis of the disease and progression despite treatment. As in long-term follow-up, ECD patients with vemurafenib seem to have a stable disease, we want to evaluate the possibility of treatment interruption as this is what we do in our current practice. Other BRAF inhibitors, such as dabrafenib, have recently been proposed for treating BRAF mutated histiocytoses. Other BRAF inhibitor interruption treatment should also be prospectively evaluated.
Key facts
- Study ID
- NCT02089724
- Run by
- Groupe Hospitalier Pitie-Salpetriere
- People needed
- 20
- Starts
- 2014-03-01
- Expected to finish
- 2019-04-01
- Last updated by the study team
- 2016-10-28
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Age superior or equal to 18 years
- Clinical and radiological presentation concordant with ECD
- Presence of histological proof of ECD
- Treatment with vemurafenib or other BRAF inhibitor
- Agreement to participate
You may not qualify if…
- Pregnancy
- Patients who exceed the safe weight limit of the PET/CT bed (220 kg) or who cannot fit through the PET/CT bore (diameter 70 cm).
Where it is running
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (enrolling)
- AP-HP, Groupe Hospitalier Pitié-Salpêtrière — Paris, France (enrolling)
Full record on ClinicalTrials.gov
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