Clinical Trials

What is a Clinical Trial?

According to, a clinical study [trial] involves research using human volunteers (also called participants) that is intended to add to medical knowledge.

There are 2 types: observational studies and clinical trials. You can learn more about clinical trials and studies from these trusted sources:

Clinical Trials Support

Are you an HLH or LCH patient or caregiver looking for help finding a clinical trial or paying for travel arrangements to participate in one? The Be The Match Jason Carter Clinical Trials Search and Support may be able to help! They offer travel grants to help get you to clinical trials and can help you navigate the process along the way.

Learn more at

Phase II Clinical Trial testing cancer genes to choose treatments for children and young adults with histiocytosis that has come back (relapsed) or has not gotten better with treatment (refractory. There are 162 sites participating in this clinical study within the United States.

Learn more about this trial, here.

Registries & Gene Therapies

You've learned more about clinical trials and studies, but what about Registries and Gene Therapies? Our friends at the National Organization for Rare Disorders (NORD) explain all about registries, gene therapies and more in the videos below. Find more information and videos at

Learn more about the registries the Histiocytosis Association supports in our Grants Awarded directory.

Click an image below to view the video on NORD's YouTube channel.

Free Genomic Profiling


The Memorial Sloan Kettering Make-an-IMPACT Initiative is a rare-cancer global outreach initiative that provides free genomic profiling for patients with Langerhans Cell Histiocytosis (LCH), Erdheim-Chester Disease (ECD), Rosai-Dorfman Disease (RDD), Juvenile Xanthogranuloma (JXG), and Histiocytic Sarcoma (HS). Genomic profiling with world class, next-generation sequencing technology developed at Memorial Sloan Kettering Cancer Center aims to identify genetic mutations in these tumors. These tumors often harbor mutations in BRAF or related family of genes and may respond to drugs that target these genetic alterations. Patients who were diagnosed or treated for LCH, RDD, ECD, JX, or HS within 2 years with current active disease may be eligible to participate. If you would like to learn more, please visit the Make-an-IMPACT site or reach out to the Make-an-IMPACT team: If you are eligible and would like to participate, please complete the volunteer form.

Additional Opportunities


The Swiss-based company AB2 Bio is conducting a clinical study in North America (U.S.A. and Canada) and Germany, and recruiting patients in several regions. The aim of this Phase III study is to assess the efficacy and safety of Tadekinig alfa in pediatric and adult patients with monogenic, interleukin-18 driven autoinflammation caused by NLRC4-MAS mutation or XIAP deficiency. Update June 2021: Patients of all ages are eligible to enroll. Learn more at

Read the Study Information Sheet here.

International Rare Histiocytic Disorders Registry (IRHDR)

The Division of Haematology and Oncology at the Hospital for Sick Children is conducting a research study. This research study is being done to build up a registry of patients with rare histiocytic disorders in order to learn more about these disorders and what types of treatments are being used. This study does not involve any additional tests and it will not impact your/your child’s current treatment. If you/your child would like to participate or have any questions, please email:

Read the Study Introduction Letter here.


NACHO-COBI is a Phase II clinical trial for pediatric, young adult, and adult patients who are being cared for at a North American Consortium for Histiocytosis (NACHO) institution. Patients must be at least 6 months old to enroll. Eligible patients will receive cobimetinib as a front-line or second-line treatment for LCH, LCH-associated Neurodegenerative Disease, or other histiocytic disorders. Preclinical studies support the potential success of this class of drug in treating mutations that may cause histiocytic diseases. This study is designed to test the potential success and harmfulness of cobimetinib in patients with histiocytic diseases. Eligible patients will be prescribed to an oral (by mouth) dose of cobimetinib daily for 28 days (a cycle). If the patient can tolerate the medication without additional illness, relapse, or progressive disease, the patient can continue taking cobimetinib for up to 12 cycles. For more information please refer to the NACHO-COBI Webpage at


NACHO-HLHRUXO​ is a Phase Ib/II clinical study for the use of a response-adapted Ruxolitinib-Containing Regimen for the treatment of Hemophagocytic Lymphohistiocytosis. This study is designed to test the treatment of HLH with a new drug called ruxolitinib. The study will focus on two groups of patients: newly diagnosed patients and patients with relapsed or refractory HLH. In this study patients will receive at least 8 weeks of therapy, and will be monitored for an additional 44 weeks after therapy. Additional details can be found on the HLHRUXO Webpage at


NACHO-BIO is the biorepository study for the NACHO Consortium. This biorepository gives patients a chance to contribute samples for research at NACHO. These samples can be collected with correlated clinical data to support specific aims of clinical trials as well as future research initiatives. These samples will allow researchers to help better define the biology of histiocytic diseases and identify biomarkers that may be utilized as diagnostic or therapeutic targets in future protocols. You do not have to currently be enrolled on a NACHO Clinical Study to contribute. Learn more on the NACHO-BIO Webpage at


LCH-IV is an international, multi-center study for LCH in patients under the age of 18 years. This study is looking at combination therapy with vinblastine and prednisone as an effective therapy to treat LCH. LCH-IV is a study that is designed to tailor treatment based on features at presentation and response to treatment and therefore has seven different strata that can be enrolled. To learn more about participating in LCH-IV in the United States, visit the NACHO LCH-IV Webpage at


*Patients should speak with their medical and caregiving teams about any trial or study you may be interested in prior to enrolling.*