There are ongoing ECD trials/studies that are currently accepting ECD patients.

Even if you or your loved one are not located near a trial/study center, you or the treating doctor are encouraged to talk to one of the researchers involved in a trial/study before treatment.  This will allow you to benefit from some of the leading research in the fast-changing world of ECD treatments.

Studies

ECD research involves observational studies and clinical trials.  Observational studies do not test treatments but rather observe a situation to develop a hypothesis that may then be tested through a clinical trial.  Both studies and clinical trials need patients to become involved to perform the research necessary to learn more about ECD. 

Clinical Trials

There are currently clinical trials open for ECD treatment.  The advantages to a patient for entering a trial include:

1. Treatment, or portions of the treatment, might be provided free to the patient. (Sometimes, testing and travel may also be provided during the trial. Patients should always ask what costs are included in the trial.)
2. Follow-up appointments are very thorough, and side effects are closely monitored and treated. (Where there are known side effects of treatment, the trial protocols are set up to take immediate action should these be encountered.)
3. Often, if a patient begins treatment outside of a trial using a particular drug, the patient may be ineligible to enter a trial for that particular treatment later. For this reason, it is often advantageous to ask about trials before beginning treatment. However, any patient interested in entering a trial is encouraged to ask whether they are eligible at any time, no matter their circumstances.

Advantages to the ECD community as a whole when a patient is entered into a trial include:

1. Trials can lead to FDA/governmental approval for the treatment of ECD. With government approval, payers will be more likely to approve payment for the treatment.
2. With a limited number of ECD patients, it is very important that data on the treatment be captured centrally to aid in the understanding of things such as (a) how effective the treatment is and under what circumstances, (b) how long should patients be kept on the treatment, (c) what, if any, indicators exist for possible treatment issues that might arise, etc.

Learn More About Clinical Trials

New drugs in the United States are tested in clinical trials (research studies) to determine that they are safe, can successfully treat a particular medical condition, and what the suggested dosage should be. The US Food and Drug Administration (FDA) reviews the clinical trial results to determine if the drug is a safe and effective treatment. When the FDA approves a treatment for a particular condition, access to that treatment becomes much easier for the majority of patients. Similar processes are used in other countries.

Clinical trials are a key research tool for advancing medical knowledge and patient care. ECD Clinical trials determine whether a treatment is safe and effective for ECD patients. Clinical trials produce the best data available for healthcare decision-making.

Purpose

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results. A clinical trial protocol describes what types of patients can participate in the research, in other words, who is eligible. Each trial must include only people who fit the patient traits for that study (the eligibility criteria). Eligibility criteria differ from trial to trial. They include factors such as a patient’s age and gender, the type and stage of the disease, and whether the patient has had specific treatments or has other health problems.

What to Expect

During a clinical trial, doctors, nurses, social workers, and other healthcare providers might be part of your treatment team. They will monitor your health closely. You may have more tests and medical exams than you would have if you had not participated in a clinical trial. Some people will need to travel or stay in hospitals to participate in clinical trials. ECD Clinical trials are currently occurring in medical centers and doctors’ offices in the US and Europe.

Benefits

Taking part in a clinical trial can have many benefits. For example, you may gain access to new treatments before they’re widely available. If a new treatment is proven to work and you’re in the group getting it, you might be among the first to benefit. You also will have the support of a team of healthcare providers, who will likely monitor your health closely.

Help Others

Even if you don’t directly benefit from the results of the clinical trial you take part in, the information gathered can help others and add to scientific knowledge. People who take part in clinical trials are vital to the process of improving medical care. Many people volunteer because they want to help others.

Clinical trials do have risks and some downsides, such as the following.

• The new strategies and treatments being studied aren’t always better than the current standard of care.
• Even if a new approach benefits some participants, it may not work for you.
• A new treatment may have side effects or risks doctors don’t know about or expect.
• Health insurance and providers don’t always cover all patient care costs for clinical trials. If you’re considering taking part in a clinical trial, find out ahead of time about costs and coverage for the treatment and travel to the medical center.

You should learn about the risks and benefits of any clinical trial before you agree to take part in the trial. Talk with your doctor about specific trials you are interested in.

Once the FDA is satisfied that the drug works and is safe, it and the drug maker create a report called the drug label. This report provides very specific information about the drug. The FDA approves the report (label), which provides the necessary information to health professionals who prescribe or sell the drug.

Off-Label Drug Use

In the US, when a drug is used in a way that is different from that described in the FDA-approved drug label, it’s said to be an “off-label” use. This can mean that the drug is:

• Used for a different disease or medical condition
• Given in a different way (such as by a different route)
• Given in a different dose than in the approved label

Some ECD treatments are FDA-approved, and some are not. Those that are not approved for ECD are considered off-label use. Off-label ECD treatments are treatments that are approved for use for other diseases but are not approved for ECD treatment. This is legal in the United States and many other countries. Off-label drug use is common in the treatment of rare diseases.

The biggest problem with off-label drug use is obtaining approval from a payer (insurance company) to reimburse for off-label drug use. Many insurance companies will not pay for an expensive drug that’s used in a way that’s not listed on the approved drug label. They do this on the grounds that its use is “experimental” or “investigative.” This financial burden can place an enormous strain on patients and families, especially when the cost of off-label treatments runs very high.

In cancer treatment, these issues have been largely addressed through 1993 federal legislation that requires insurance to cover medically appropriate cancer therapies. This law includes off-label uses if the treatment has been tested in careful research studies and described in well-respected drug reference books or medical journals. In 2008, Medicare rules were changed to cover more off-label uses of cancer treatment drugs.

Still, health insurance coverage laws and regulations are complex. If your doctor is thinking about off-label drug use, you and your doctor should carefully check your health plan’s coverage. If you are denied coverage, it might help if the doctor sends the insurer copies of peer-reviewed journal articles or other respected sources that support the off-label use. The ECD Global Alliance is happy to help medical teams with this. (Please see our page Filing Payer Appeals.)

Current Open ECD Studies & Trials

	#	NCT#	Name of Study	Purpose	Type of Study	Selection	Age Requirement	Care Center / Contact
	1	NCT05915208	Histiocytic Disorder Follow-up Study	To understand the health problems experienced by individuals with ECD and other histiocytic disorders.	Surveys only Observational Retrospective	English survey completion (online, on phone, or on paper) from the comfort of your home.	18 – 89 years old	University of Alabama at Birmingham Birmingham, AL USA Gaurav Goyal, MD histio@uabmc.edu +1-866-438-1640
	2	NCT03329274	Registry for Patients with Erdheim-Chester Disease and Other Histiocytoses	Determine what kinds of health problems are caused by histiocytosis, what happens as a result of different treatments, and how ECD affects people's lives, their feelings and their attitudes.	Surveys & medical records Observational Prospective	Erdheim-Chester Disease, Langerhans cell histiocytosis, Rosai Dorfman disease, or another histiocytic neoplasm	18 years and older	MSKCC New York City, NY USA Eli Diamond, MD diamone1@mskcc.org +1- 212-610-0243
	3	NCT03990428	Supportive Care Needs of Caregivers of People with Erdheim-Chester Disease and Other Histiocytic Diseases	Determine the supportive care needs of informal caregivers of people with Erdheim-Chester disease and other histiocytic diseases	Online surveys Observational Prospective	Self id’d unpaid support person for ECD, LCH, RDD, or JXG patient	18 years and older	MSKCC New York City, NY USA Eli Diamond, MD diamone1@mskcc.org +1- 212-610-0243
	4	NCT03017820	VSV-hIFNbeta-NIS in Treating Patients With Relapsed or Refractory Multiple Myeloma, Acute Myeloid Leukemia or Lymphoma	Studies the best dose and side effects of recombinant vesicular stomatitis virus carrying the human NIS and IFN beta genes as a way to to kill cancer cells without damaging normal cells. For patients whose prior treatment did not help	Clinical Trial Interventional Treatment	Relapsed/ refractory to prior ECD treatments	18 years and older	Mayo Clinic in Rochester Rochester, MN USA Nora Bennani, MD Ronald Go, MD go.ronald@mayo.edu
	5	NCT04640779	Low-Dose Selinexor and Choline Salicylate for Non-Hodgkin or Hodgkin Lymphoma, Histiocytic/Dendritic Cell Neoplasm, or Relapsed or Refractory Multiple Myeloma	Evaluates the side effects and best dose of choline salicylate given together for patients whose prior treatment did not help.	Clinical Trial Interventional Treatment	Relapsed/ refractory to prior ECD treatments	18 years and older	Mayo Clinic in Rochester Rochester, MN USA Jonas Paludo, M.D. Ronald Go, MD go.ronald@mayo.edu
	6	NCT06153173	Mirdametinib in Histiocytic Disorders	Determine if treatment with mirdametinib in patients with histiocytic disorders will be better than current treatments and with fewer side effects.	Clinical Trial Interventional Treatment	Diagnosis of histiocytic neoplasm confirmed by a Cincinnati Children's Hospital pathologist. Exception for those with isolated pituitary/CNS disease where biopsy is not feasible.	2 years and older	Children's Hospital Medical Center Cincinnati, Ohio USA Monica Trapp monica.trapp@cchmc.org Ashish Kumar, MD, PhD +1-513-803-8574
	7	n/a	LCH and Vitamin D	To investigate changes in the skeletal related quality of life (QoL) among adult LCH patients following a 3-month cholecalciferol supplementation to achieve or maintain 25(OH)D levels ≥ 30 ng/ml (75 nmol/L).	Clinical Trial Interventional Adjunct Therapy	Histiocytosis diagnosis	All ages?	251 Hellenic Air Force & VA General Hospital Greece Makras Polyzois, MD info@adultlch.gr 30-2107463606
	8	NCT06411821	Phase 2 Trial of Ulixertinib for Patients with Histiocytic Neoplasms	Clinical trial of oral ERK inhibitor	Clinical Trial Interventional Treatment	Any histiocytic neoplasm	18 years and older	MSKCC NYC, NY USA Eli Diamond, MD diamone1@mskcc.org
	9	NCT04079179	Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders (NACHO-COBI)	Study of cobimetinib in children and adults with LCH and other histiocytic diorders.	Clinical Trial Interventional Treatment	Relapsed/ refractory to prior ECD treatments	6 months and older	NACHO (Baltimore, Dallas, DC, Houston, Madison, Memphis, Orange, ??) Texas Children’s Hospital Houston, TX USA Carl Allen, MD ceallen@texaschildrens.org +1-832-822-4242
	10	NCT05768178	Vemurafenib in Combination With Cobimetinib in Adult Patients With BRAF Positive Cancers.	Evaluate the efficacy of vemurafenib and cobimetinib in adult patients with rare cancers with BRAF V600 mutations.	Clinical Trial Interventional Treatment	BRAF V600E	16 years and older	Multiple UK Hospitals Including Leeds General Infirmary Leeds, UK Martin Elliott, MBBS Martin.elliott1@nhs.net 0113 392 8779
	11	NCT02925234	The Drug Rediscovery Protocol (DRUP Trial) (DRUP)	A drug-access program, in which patients are treated with registered targeted therapy matched to their molecular tumor profile.	Clinical Trial	Histologically-proven disease	18 years and older	Erasmus MC Rotterdam, Netherlands M.J.A. de Jonge, MD, PhD E.E. Voest, prof. 0031205129111 DRUP@nki.nl