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Disease
Erdheim-Chester Disease
Late-stage therapeutic developmentEmerging research
1
Publications
10
Clinical trials
2020
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Low
Key developments
- 3 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
- Q4 2027In-human CXCR4 Imaging of Hematologic and Solid Tumors Using [68Ga]-Pentixafor-PET
- Q4 2029DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.
- Q3 2030MC220806: Phase I Study Evaluating the Efficacy of CSF1R and TAM Receptor or Inhibition in Hematologic Malignancies With Q702, a Small Molecular Inhibitor
Major developments
Upcoming trial readoutImportant
Results expected Q4 20292025-11-24
Major trial resultsImportant
Results posted2025-12-10
Clinical MilestonesViewHide
Results posted
- 2025-12-10A Phase II Study of Lenalidomide for Adult Histiocyte DisordersResults posted
Results expected
- 2026-02-05In-human CXCR4 Imaging of Hematologic and Solid Tumors Using [68Ga]-Pentixafor-PETResults expected Q4 2027
- 2025-11-24DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.Results expected Q4 2029
- 2025-10-10MC220806: Phase I Study Evaluating the Efficacy of CSF1R and TAM Receptor or Inhibition in Hematologic Malignancies With Q702, a Small Molecular InhibitorResults expected Q3 2030
Recently completed
Activity timeline6
- 2026-05-08ClinicalA Phase I Trial of Bevacizumab, Temsirolimus Alone and in Combination With Valproic Acid, or Cetuximab in Patients With Advanced Malignancy and Other IndicationsTerminated
- 2026-02-05ClinicalIn-human CXCR4 Imaging of Hematologic and Solid Tumors Using [68Ga]-Pentixafor-PETResults expected Q4 2027
- 2025-12-10ClinicalA Phase II Study of Lenalidomide for Adult Histiocyte DisordersResults posted
- 2025-12-01ClinicalA Phase 2 Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cell Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders.Primary completion
- 2025-11-24ClinicalDETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial): An Umbrella-Basket Platform Trial to Evaluate the Efficacy of Targeted Therapies in Rare Adult, Paediatric and Teenage/Young Adult (TYA) Cancers With Actionable Genomic Alterations, Including Common Cancers With Rare Actionable Alterations. Treatment Arm 05: Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.Results expected Q4 2029
- 2025-10-10ClinicalMC220806: Phase I Study Evaluating the Efficacy of CSF1R and TAM Receptor or Inhibition in Hematologic Malignancies With Q702, a Small Molecular InhibitorResults expected Q3 2030
Clinical trials
The current development programme across all trial phases.
Clinical programme
10
5
1
0
Recruiting
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Most influential
Leading journals1
- Blood1
Leading researchers8
- Abdo A1
- Cao XX1
- Cohen-Aubart F1
- Collin M1
- Dagna L1
- Diamond EL1
- Durham BH1
- Estrada-Veras JI1
Affiliations (unnormalised)6
- Ariel University1
- Baylor College of Medicine1
- Cincinnati Children's Hospital Medical Center1
- Clinic of Histiocytic Neoplasms1
- Columbia University Medical Center1
- Dana-Farber Cancer Institute1
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A rare form of non-Langerhans-cell histiocytosis (HISTIOCYTOSIS, NON-LANGERHANS-CELL) with onset in middle age. The systemic disease is characterized by infiltration of lipid-laden macrophages, multinucleated giant cells, an inflammatory infiltrate of lymphocytes and histiocytes in the bone marrow, and a generalized sclerosis of the long bones.
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.