NCT04773366 A Prospective Study for the Treatment of Children With Newly Diagnosed LCH Using a Cytarabine Contained Protocol
| NCT ID | NCT04773366 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Shanghai Children's Medical Center |
| Condition | Langerhans Cell Histiocytosis |
| Study Type | INTERVENTIONAL |
| Enrollment | 200 participants |
| Start Date | 2018-07-01 |
| Primary Completion | 2023-06-30 |
Eligibility & Interventions
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 200 participants in total. It began in 2018-07-01 with a primary completion date of 2023-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
From January 2010 to December 2014, 150 children with MS-LCH were treated in our hospital following a LCH II (Arm B) based protocol. Treatment was based on a modification of the LCH-II (Arm B) based protocol. However, the continuation treatment was extended to 56 weeks and etoposide was omitted from the continuation treatment. For the 59 patients with RO involvement (RO+) (the lungs are not considered a RO in the current study), the rapid response rate (week 6) was 61.0% and the 3-year overall survival (OS) 73.4±5.9%. Rapid responders had a better 3-year survival rate than poor responders (90.9±5.0% vs. 45.7±11.0%, P\<0.001). The 3-year OS in the current study is 10\~20% lower than the rates reported by Gadner et al. and Morimoto et al.. We have not yet adopted effective salvage therapies for RO+ patients with recurrent disease. During the time of this study, cladribine was unavailable. Second-line therapy for non-responders or patients with disease reactivation was individualized treatment based on the physician's experience. An effective salvage therapy is essential for this high-risk group. For 91without RO involvement (RO-), 78 patients (85.7%) were rapid responders at week 6. The 3-year cumulative reactivation rate was 10.7% for RO- patients. No death occurred in this subgroup, with a 3-year OS of 100% in RO- patients. Compared to the LCH II and LCH III trials, the current study had a more intensive initial treatment regimen for RO- patients. However, the addition of etoposide to prednisone and vincristine in the initial therapy did not increase the 6-week response rate for RO- patients (85.7% in this study compared to 83% in the LCH II study and 86% in the LCH III study). Surprisingly, with a relatively intense initial treatment, a relatively low 3-year cumulative reactivation rate was observed in RO- patients in the current study. This result suggests that the initial treatment intensity and duration of continuation therapy both impact disease reactivation. The intensity of induction can affect the degree of disease resolution. Insufficient treatment intensity might lead to late relapse. Similarity to that observed has been in other childhood hematological malignancies. This finding deserves to be tested in prospective clinical trials with long-term follow-up. Cytarabine has been applied for patients with LCH but has never been evaluated in our hospital prospectively. In this study, we administer a cytarabine contained protocol to patients with multisystem involvement with or without risk organs involvement. The treatment results will be compared with our historical studies.
Eligibility Criteria
Inclusion Criteria: 1. Age under 18 years 2. Newly diagnosed LCH：Morphologic identification of the characteristic LCH cells, positive staining of the lesional cells with CD1α and/or Langerin 3. No congenital immunodeficiency, HIV infection, or prior organ transplant 4. No previous chemotherapy/target therapy/radiation, if any steroid applied, total prior steroids dosage \< prednisone 280 mg/m2 Exclusion Criteria: * Patients have overwhelming infection, and a life expectancy of \< 2 weeks
Contact & Investigator
Yi-Jin Gao, MD
PRINCIPAL INVESTIGATOR
Shanghai Children's Medical Center
Frequently Asked Questions
Who can join the NCT04773366 clinical trial?
This trial is open to participants of all sexes, aged 1 Day or older, up to 18 Years, studying Langerhans Cell Histiocytosis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04773366 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 200 participants.
Is NCT04773366 currently recruiting?
Yes, NCT04773366 is actively recruiting participants. Contact the research team at sumeng@scmc.com.cn for enrollment information.
Where is the NCT04773366 trial being conducted?
This trial is being conducted at Shanghai, China.
Who is sponsoring the NCT04773366 clinical trial?
NCT04773366 is sponsored by Shanghai Children's Medical Center. The principal investigator is Yi-Jin Gao, MD at Shanghai Children's Medical Center. The trial plans to enroll 200 participants.