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Long-term Outcome After Vemurafenib / BRAF Inhibitors Interruption in Erdheim-chester Disease

NCT02089724 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 20

Last updated 2016-10-28

No results posted yet for this study

Summary

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.

Conditions

  • Erdheim-Chester Disease

Sponsors & Collaborators

Principal Investigators

  • Julien Haroche, MD, PhD · Groupe Hospitalier Pitié-Salpêtrière

  • Fleur Cohen Aubart, MD, PhD · Groupe Hospitalier Pitié-Salpêtrière

  • Eli L. Diamond, MD, PhD · Memorial Sloan Kettering Cancer Center

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-03-31
Primary Completion
2016-12-31
Completion
2019-04-30

Countries

  • United States
  • France

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02089724 on ClinicalTrials.gov