Histiocytosis Program

Clinical Trials

The Histiocytosis Program at Children's National is one of a few comprehensive histiocytosis programs in the United States, and we're leading the way to find new and better treatments for children with histiocyte disorders. We have an active histiocytosis clinical trial program that offers access to cutting-edge therapies for newly diagnosed, relapsed and refractory patients

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NACHO-COBI: A Phase II Study to Assess the Safety and Efficacy of Cobimetinib in Refractory Langerhans Cells Histiocytosis, LCH-Associated Neurodegenerative Disease, and Other Histiocytic Disorders

ClinicalTrials.gov Identifier: NCT04079179

Disease Type:

Langerhans cell histiocytosis (LCH), LCH-ND, and other histiocytic disorders with activating MAPK pathway mutations

Key Eligibility Criteria:

This trial is an open-label, limited-center Phase 2 study to evaluate the safety and efficacy of cobimetinib (a MEK inhibitor) in histiocytic disorders with MAPK pathway activation.
Specific groups will include in children and young adults with active Langerhans Cell Histiocytosis (Group 1)
Any patients who develop LCH-associated neurodegenerative disease (Group 2)
Children and young adults with other histiocytic disorders associated with activating MAPK pathway mutations (Group 3) (this group is closed)
Adults with histiocytic disorders (Group 4)

Sponsor:

NACHO

Investigator:

Birte Wistinghausen, MD

NACHO-BIO: A Translational Biology Platform to Advance Understanding of Pathogenesis and Improve Outcomes for Patients with Histiocytic Disorders

Disease Type:

Patients with confirmed or suspected diagnosis of LCH, JXG, ECD, MFRH, RDD, HLH, malignant histiocytosis or other histiocytic diseases

Key Eligibility Criteria:

Patients with confirmed or suspected diagnosis of LCH, JXG, ECD, MFRH, RDD, HLH, malignant histiocytosis or other histiocytic diseases that agree to have their samples stored and used for future research including genetic studies

Sponsor:

NACHO

Investigator:

Birte Wistinghausen, MD

LCH-IV: LCH-IV is an international, multicenter, prospective clinical Phase II study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years)

ClinicalTrials.gov Identifier: NCT02205762

Disease Type

Langerhans cell histiocytosis

Key Eligibility Criteria:

The eligibility criteria are organized into seven strata, with consent determined by the patient’s diagnosis:

Stratum I: Newly diagnosed <18yo first-line treatment for MS-LCH (multi- system) patients (Group 1) and patients with SS-LCH (single-system) with multifocal bone or “CNS-risk” lesions (Group 2-closed in the US)
Stratum II Second-line treatment for non-risk patients (patients without risk organ involvement who fail first-line therapy or have a reactivation after completion of first-line therapy)
Stratum III: Salvage treatment for risk LCH (patients with dysfunction of risk organs who fail first-line therapy)
Stratum IV: Patients from Stratum I or III with: AD worse in risk organs after 6 or 12 weeks, AD worse after 2nd/3rd or worse/intermediate after 4th 2-CdA/Ara-C course, Presence of severe organ dysfunction
Stratum V: Monitoring and treatment of isolated tumorous and neurodegenerative CNS-LCH
Statum VI: Natural history and management of “other” SS-LCH (patients who do not need systemic therapy at the time of diagnosis)
Stratum VII: Long-term Follow up (all patients irrespective of previous therapy will be followed for reactivation or permanent consequences once complete disease resolution has been achieved and the respective protocol treatment completed)

Sponsor:

NACHO

Investigator:

Stephan Ladisch, MD