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Thalidomide, Cyclophosphamide and Dexamethasone for Recurrent/Refractory Adult Langerhans Cell Histiocytosis

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ClinicalTrials.gov Identifier: NCT04120519
Recruitment Status : Recruiting
First Posted : October 9, 2019
Last Update Posted : October 9, 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 most commonly in children. Because of the rarity of LCH in adults and a lack of prospective randomized trials, the treatment strategy for adults is mostly based on pediatric protocols. The overall response rate of therapy based on vinblastine plus prednisone in adults is lower than in children and the treatment tends to show higher toxicity.There is little data to guide therapy after frontline treatment. In a phase 2 trial, thalidomide as monotherapy gave a 70% response rate in recurrent/refractory low risk LCH but there were no responses in six high risk children. We want to analyze the efficacy and toxicity of thalidomide combined with dexamethasone and cyclophosphamide regimens in the treatment of recurrent/refractory LCH among adult patients at our hospital.

Condition or disease Intervention/treatment Phase
Langerhans Cell Histiocytosis Drug: thalidomide combined with dexamethasone and cyclophosphamide Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: thalidomide combined with dexamethasone and cyclophosphamide
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Thalidomide, Cyclophosphamide and Dexamethasone for Adult Patients With Recurrent/Refractory 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: TCD
thalidomide 100mg qn cyclophosphamide 300mg/m2 d1,8,15 dexamethasone 40mg d1,8,15,22
Drug: thalidomide combined with dexamethasone and cyclophosphamide
TCD




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

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



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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 recurrent/refractory or at least receive one line of systemic treatment of LCH
    • 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 were newly diagnosed 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): NCT04120519


Contacts
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Contact: Xinxin caoyang, MD 69155027 caoxinxin@pumch.cn

Locations
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China
Peking Union Medical College Hospital Recruiting
Beijing, China
Contact: Xinxin Cao         
Sponsors and Collaborators
Peking Union Medical College Hospital
Investigators
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Study Director: Jian Li Peking Union Medical College Hospital
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Responsible Party: Cao Xinxin, Associate Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT04120519    
Other Study ID Numbers: PUMCHRRLCH-1
First Posted: October 9, 2019    Key Record Dates
Last Update Posted: October 9, 2019
Last Verified: October 2019

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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
recurrent/refractory
Additional relevant MeSH terms:
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Histiocytosis, Langerhans-Cell
Histiocytosis
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Thalidomide
Dexamethasone
Cyclophosphamide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Leprostatic Agents
Anti-Bacterial Agents
Anti-Infective Agents