AraC for Newly Diagnosed Adult Langerhans Cell Histiocytosis

Study Purpose

Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages from neonates to the elderly. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone. This regimen has never been proven effective for adults in a prospective study, since the only prospective trial evaluating the efficacy of a vinblastine/prednisone regimen in adults was prematurely closed due to unacceptable toxicities. A retrospective study showed an advantage for cytarabine monotherapy compared with vinblastine/prednisone in bone LCH patients. This phase 2, prospective, single-center study is designed to evaluate the efficacy and safety of cytarabine monotherapy in adults with newly diagnosed MS-LCH or LCH with multifocal single system (SS-m) involvement.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years - 75 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - • Histologically confirmed diagnosis of LCH.
  • - Patients were newly diagnosed or did not receive prior systemic treatment of LCH (patients who had received radiotherapy alone were allowed).
  • - Age ≥18 years and ≤75 years.
  • - LCH involved multisystem or multifocal single system.
  • - Eastern Cooperative Oncology Group (ECOG) performance status 0-2.
  • - Patients must have adequate renal, liver, and bone marrow function as defined by the following criteria: - Absolute neutrophil count ≥1500 cells per mm3 or ≥500 cells per mm3 in the case of known hematopoietic system involvement by LCH.
  • - Platelet count ≥100000 cells per mm3 or ≥20000 cells per mm3 in the case of known hematopoietic system involvement by LCH.
  • - Creatinine clearance [according to Cockcroft formula] ≥60 mL/min.
  • - Aspartate aminotransferase and alanine aminotransferase ≤2·5×upper limit of normal [ULN], and total bilirubin ≤2·5×ULN; or ≤10×ULN in the case of known liver involvement by LCH.
  • - No active or untreated infection.
  • - No cardiac abnormalities.
  • - Subject provide written informed consent.
  • - A female is eligible to enter and participate in this study if she is of: - Non-childbearing potential including ω Any female who has had a surgical procedure rendering her incapable of becoming pregnant.
ω Subjects have experienced total cessation of menses for more than 1 year and be greater than 45 years in age. ⎫ Childbearing potential, including any female who has had a negative serum pregnancy test within 2 weeks prior to the first dose of study treatment, and agrees to use adequate contraception. Male subjects must use an effective barrier method of contraception during the study and for 90 days following the last course of MA if sexually active with a childbearing potential

Exclusion Criteria:

  • - • Non-langerhans cell histiocytosis.
  • - Patients had concurrent malignancies.
  • - Patients who had received any treatment except radiotherapy for LCH.
  • - History of myocardial infarction, or unstable angina, or New York Heart Association (NYHA) Grade III-IV within 6 months prior to Day 1.
  • - Women who were pregnant or of childbearing potential.
  • - Known HIV seropositive, active hepatitis C infection, and/or hepatitis B (defined as HCV RNA ≥103 copies or HBV DNA ≥103 copies at screening).
  • - Major surgical procedure within 28 days prior to the first dose of study treatment.
  • - Presence of uncontrolled infection.
  • - Evidence of active bleeding or bleeding diathesis.
  • - Any serious and/or unstable pre-existing medical, psychiatric, or other condition that could interfere with subject's safety, provision of informed consent, or compliance to study procedures.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT04121819
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Peking Union Medical College Hospital
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Jian Li, MD
Principal Investigator Affiliation Peking Union Medical College Hospital
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other
Overall Status Recruiting
Countries China
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Langerhans Cell Histiocytosis
Arms & Interventions

Arms

Experimental: AraC

cytarabine 100mg/m2 d1-5 subcutaneous

Interventions

Drug: - Cytarabine

cytarabine 100mg/m2 d1-5 subcutaneous

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

International Sites

Peking Union Medical College Hospital, Beijing, China

Status

Recruiting

Address

Peking Union Medical College Hospital

Beijing, ,

Site Contact

Xinxin Cao

caoxinxin@pumch.cn

69155027

Terms of Service

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