AraC for Newly Diagnosed Adult Langerhans Cell Histiocytosis

NCT04121819 | Unknown Phase 2

• 1 other identifier: PUMCHNDLCH2
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Langerhans Cell Histiocytosis

Keywords

Langerhans cell histiocytosis; adult; newly diagnosed

Outcome Measures

Primary Outcomes (1)

• Event-free survival (EFS)

  Events were defined as a poor response to AraC, reactivation after AraC therapy and death from any cause.

  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

Secondary Outcomes (2)

• Overall response rate

  on 12 months

• Overall survival

  the duration from initiation of AraC treatment to the date of death or last follow-up, assessed up to 5 years

Study Arms (1)

AraC

EXPERIMENTAL

cytarabine 100mg/m2 d1-5 subcutaneous

Drug: Cytarabine

Interventions

Cytarabine DRUG

cytarabine 100mg/m2 d1-5 subcutaneous

AraC

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, China

RECRUITING

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell

Interventions

Cytarabine

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Jian Li, MD

  Peking Union Medical College Hospital

  STUDY DIRECTOR

Central Study Contacts

Xinxin Cao, MD

CONTACT

69155027; caoxinxin@pumch.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Model Details: AraC

Study Record Dates

• First Submitted: October 7, 2019
• First Posted: October 10, 2019
• Study Start: October 1, 2019
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2021
• Last Updated: June 8, 2021

Record last verified: 2021-06

Locations

CN (1)