Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

AraC for Newly Diagnosed Adult Langerhans Cell Histiocytosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04121819
Recruitment Status : Recruiting
First Posted : October 10, 2019
Last Update Posted : October 10, 2019
Sponsor:
Information provided by (Responsible Party):
Cao Xinxin, Peking Union Medical College Hospital

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Langerhans Cell Histiocytosis Drug: Cytarabine Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: AraC
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cytarabine Monotherapy for Adult Patients With Newly Diagnosed Langerhans Cell Histiocytosis: A Single Arm, Single Center, Prospective Phase 2 Study
Estimated Study Start Date : October 2019
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2021


Arm Intervention/treatment
Experimental: AraC
cytarabine 100mg/m2 d1-5 subcutaneous
Drug: Cytarabine
cytarabine 100mg/m2 d1-5 subcutaneous




Primary Outcome Measures :
  1. Event-free survival (EFS) [ Time Frame: the duration from initiation of AraC treatment to the date of a first event or date of death from any cause, whichever come first, assessed up to 5 years ]
    Events were defined as a poor response to AraC, reactivation after AraC therapy and death from any cause.


Secondary Outcome Measures :
  1. Overall response rate [ Time Frame: on 12 months ]
    the cumulative number of patients with either non-active disease or regressive disease (signs and symptoms were improved with no new lesions) after AraC therapy

  2. Overall survival [ Time Frame: the duration from initiation of AraC treatment to the date of death or last follow-up, assessed up to 5 years ]
    Overall survival



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04121819


Contacts
Layout table for location contacts
Contact: Xinxin Cao, MD 69155027 caoxinxin@pumch.cn

Locations
Layout table for location information
China
Peking Union Medical College Hospital Recruiting
Beijing, China
Contact: Xinxin Cao         
Sponsors and Collaborators
Peking Union Medical College Hospital
Investigators
Layout table for investigator information
Study Director: Jian Li, MD Peking Union Medical College Hospital
Layout table for additonal information
Responsible Party: Cao Xinxin, Associate Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT04121819    
Other Study ID Numbers: PUMCHNDLCH2
First Posted: October 10, 2019    Key Record Dates
Last Update Posted: October 10, 2019
Last Verified: October 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cao Xinxin, Peking Union Medical College Hospital:
Langerhans cell histiocytosis
adult
newly diagnosed
Additional relevant MeSH terms:
Layout table for MeSH terms
Histiocytosis, Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Cytarabine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs