New hope for kids with rare immune disorder: international trial tests drug cocktails

NCT ID NCT02205762

Disease control Sponsor: North American Consortium for Histiocytosis Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tests several drug combinations and stem cell transplants in 1,400 children with Langerhans cell histiocytosis, a rare immune disease. The goal is to prevent the disease from coming back and reduce lasting problems like hormone deficiencies and brain issues. Researchers are comparing different treatments based on how severe the disease is.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: combination of prednisone, vinblastine, mercaptopurine, indomethacin, methotrexate, cytarabine, cladribine, and hematopoietic stem cell transplant
What this could lead to: If successful, this could establish safer, more effective treatment protocols for children with LCH, reducing reactivation rates and long-term damage.
What could go wrong: This is a large but still early-phase trial combining many drugs; side effects from chemotherapy and transplant risks are significant, and results may not apply to all patients.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

1,400 people

The number who actually took part.

Start date

Nov 2016

Expected to finish

Jul 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Stratum I * Patients must be less than 18 years of age at the time of diagnosis. * Patients must have histological verification of the diagnosis of Langerhans cell histiocytosis according to the criteria described in Section 6.1 * Signed informed consent form * Stratum II * Patients of Stratum I who have: * Progressive disease (AD worse) in non-risk organs after 6 weeks (Initial Course * AD intermediate or worse in non-risk organs or AD better in risk organs after 12 weeks (Initial Course 2) * Disease progression (AD worse) in non-risk organs at any time during continuation treatment * Active disease at the end of Stratum I treatment * Disease reactivation in non-risk organs at any time after completion of Stratum I treatment * Stratum III * Patients from Stratum I who fulfill the following criteria: * AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2). * Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as * Hb \<70 g/L (\<7.0 g/dl) and/or transfusion dependency * PLT \<20 x109/L (20,000/μL) and/or transfusion dependency (both criteria have to be fulfilled) AND/OR * Liver dysfunction (or digestive involvement with protein loss) * Total protein \<55 g/L or substitution dependency * Albumin \<25 g/L or substitution dependency (at least one of the two criteria to be fulfilled) * Stratum IV * Patients from Stratum I or Stratum III who fulfill the following criteria: * AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2) of Stratum I OR * AD worse after the 2nd and 3rd 2-CdA/Ara-C course, and those AD worse or AD intermediate after the 4th 2-CdA/Ara-C course of Stratum III AND * Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as defined in Table XI (see Section 10.3.1). * Informed consent: All patients or their legal guardians (if the patient is \<18 years of age) must sign an Ethics or institutional Review Board approved consent form indicating their awareness of the investigational nature and the risks of this study. When appropriate, younger patients will be included in all discussions in order to obtain assent. * Adequate organ function: Patients should have adequate hepatic, renal, cardiac and pulmonary function to undergo reduced intensity HCT based upon local institutional guidelines, or at a minimum meet requirements noted in eligibility checklist Appendix A-VIII\_1. However, significant hepatic and pulmonary dysfunction, if secondary to underlying LCH disease activity, will not exclude patients from protocol enrollment and should be discussed with the National PI Coordinator and the Coordinating Principal Investigator. * Stratum V * All patients with verified diagnosis of LCH and MRI findings consistent with ND-CNSLCH irrespective of previous treatments (also those not registered to other Strata ofLCH-IV). * Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion in the hypothalamus-pituitary axis). In patients with already established diagnosis of LCH and radiologic finding of CNS lesions compatible with LCH, a biopsy of the lesion is not obligatory. In all other cases a biopsy of the lesion is needed for inclusion into the study * Stratum VI \-- Patients with newly diagnosed SS-LCH and localization other than "multifocal bone",isolated tumorous CNS lesion, or isolated "CNS-risk" lesion. * Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as consent for longterm follow-up has not been withheld. Exclusion Criteria: * Stratum I * Pregnancy (patients of child-bearing age must be appropriately tested before chemotherapy) * LCH-related permanent consequences (e.g. vertebra plana, sclerosing cholangitis, lung fibrosis, etc.) in the absence of active disease * Prior systemic therapy * Stratum II * Patients with progressive disease in risk organs * Permanent consequences (e.g. sclerosing cholangitis, lung fibrosis, etc.) without evidence of active LCH in the same organ or in any other locations * No written consent of the patient or his/her parents or legal guardian * Stratum III * The presence of any of the following criteria will exclude the patient from the study: * Isolated sclerosing cholangitis without evidence of active hepatic LCH as the only evidence of risk organ involvement. * Inadequate renal function as defined by serum creatinine \> 3x normal for age * Stratum IV * Pulmonary failure (requiring mechanical ventilation) not due to active LCH. * Isolated liver sclerosis or pulmonary fibrosis, without active LCH. * Uncontrolled active life-threatening infection. * Decreased renal function with a GFR of less than 50ml/1.73m2/min. * Pregnancy or active breast feeding * Failure to provide signed informed consent * Stratum VI * Patients with SS-LCH who have an isolated tumorous CNS lesion (they are eligible for Stratum V), * Patients with isolated "CNS-risk" or multifocal bone lesions (they are eligible for Stratum I, Group 2)

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Conditions

The condition(s) this trial relates to.

Histiocytosis, Langerhans-Cell Langerhans cell histiocytosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Langerhans cell histiocytosis

Contacts and locations

Locations

Akron Children's Hospital

Akron, Ohio, 44308, United States
American Family Children's Hospital University of Wisconsin

Madison, Wisconsin, 53792, United States
Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611-2991, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Carolinas Medical Center, Levine Children's Hospital

Charlotte, North Carolina, 28203, United States
Children's Healthcare of Atlanta, Emory

Atlanta, Georgia, 30342, United States
Children's Hospital at Montefiore

The Bronx, New York, 10467, United States
Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Medical Center Dallas, UT Southwestern

Dallas, Texas, 75235, United States
Children's Mercy Hospitals

Kansas City, Kansas, 64108, United States
Children's Minnesota

Minneapolis, Minnesota, 55404, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Children's of Alabama

Birmingham, Alabama, 35233, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States
Columbia University / Herbert Irving Cancer Center

New York, New York, 10032, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States
Greenville Health System BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Johns Hopkins University

Baltimore, Maryland, 21287, United States
Madigan Army Medical Center

Tacoma, Washington, 98431, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10170, United States
Mount Sinai Hospital

New York, New York, 10029, United States
Phoenix Children's Hospital

Phoenix, Arizona, 85006, United States
Providence Sacred Heart Children's Hospital

Spokane, Washington, 99204, United States
Rainbow Babies & Children's Hospital, University Hospitals

Cleveland, Ohio, 44106, United States
Riley Hospital for Children - Indiana University

Indianapolis, Indiana, 46202, United States
Russell J Ebeid Children's Hospital (Promedica)

Toledo, Ohio, 43606, United States
SUNY Upstate Medical University

Syracuse, New York, 13210, United States
St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
UCSF Benioff Children's Hospital of Oakland

Oakland, California, 94609, United States
UCSF Helen Diller Family Cancer Center

San Francisco, California, 94158-0106, United States
UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States
University of Kentucky A.B.Chandler Medical Center

Lexington, Kentucky, 40536, United States
University of Louisville, Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Valley Children's Healthcare

Madera, California, 93636, United States
Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

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