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Risk-specific multicenter phase II clinical trial for Langerhans cell histiocytosis in children and young adults. (JPLSG-LCH-19-MSMFB)

Risk-specific multicenter phase II clinical trial for Langerhans cell histiocytosis in children and young adults (JPLSG-LCH-19-MSMFB)

Shioda Yoko

National Center for Child Health and Development

2-10-1 Okura,Setagaya-ku,Tokyo

shioda-y@ncchd.go.jp

Shioda Yoko

National Center for Child Health and Development

2-10-1 Okura,Setagaya-ku,Tokyo

+81-3-3416-0181

shioda-y@ncchd.go.jp

Recruiting

June. 01, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

(1) Histologically LCH has been diagnosed (confirmed by institutional diagnosis).
(2) The age at registration is less than 40 years old.
(3) PS (Performance Status) >= 10 (Lansky score is used for those under 20 years old, and Karnofsky score is used for those over 20 years old).
(4) LCH with Multi-system type or Multifocal-bone type.
(5) Newly diagnosed untreated cases (surgical treatment and steroid treatment are not applicable).
(6) Samples can be submitted to the central diagnostic facility to enable central diagnosis at a later date.
(7) Sufficient explanation and written consent have been obtained for the patient or one's substitute. If the patient is 16 years of age or older, investigator must also obtain written consent from the patient.

(1) A case with hepatic / renal / cardiac dysfunction that interferes with study treatment. However, if the observed abnormality is caused by LCH and it is predicted that treatment with LCH is likely to improve it, or if it is judged to be physiological jaundice, it is not considered to be an abnormality that violates the exclusion criteria. For evaluation, test values within 14 days prior to the case registration date are used.
(2) Accompanied by intracranial hemorrhage (CTCAE ver.5.0; grade 3 or higher) that interferes with treatment.
(3) A case who have central nervous system degeneration.
(4) Have a history of malignant neoplasm or have multiple cancers in the past.
(5) Children weighing less than 2500 g at the time of registration.
(6) HIV antibody positive.
(7) HBs antigen positive or HCV antibody positive.
(8) Women who are pregnant or may become pregnant.
(9) When the principal investigator and the doctor who shares the research judge that participation in this study is not appropriate.

Both

Multi-system disease and multifocal bone disease with Langerhans cell histiocytosis

Dexamethasone and intrathecal injection (cytarabine) for multi-system disease, zoledronic acid for multifocal bone disease.

Langerhans cell histiocytosis

D006646

- 3 year event free survival rate by risk group of under 20 years old.
- 3 year event free survival rate of 20 years old and over.

- Event-free survival (EFS) (overall, by risk, by generation, by BRAFV600E gene mutation pattern at diagnosis, by BRAFV600E gene mutation pattern in bone marrow at diagnosis, by peripheral blood BRAFV600E gene mutation pattern at diagnosis, cases in which GR / PR was obtained 6 weeks after the start of treatment, by reduction rate of BRAF V600E gene mutation in bone marrow / peripheral blood during treatment)
- Overall survival (OS) (overall, by risk, by generation, by BRAFV600E gene mutation pattern at diagnosis, by BRAFV600E gene mutation pattern in bone marrow at diagnosis, by peripheral blood BRAFV600E gene mutation pattern at diagnosis, by Initial treatment responsiveness, by reduction rate of BRAF V600E gene mutation in bone marrow / peripheral blood during treatment)
- Progression-free survival (PFS) (overall, by risk, by generation, by BRAFV600E gene mutation pattern at diagnosis, by BRAFV600E gene mutation pattern in bone marrow at diagnosis, by peripheral blood BRAFV600E gene mutation pattern at diagnosis, cases in which GR / PR was obtained 6 weeks after the start of treatment, by reduction rate of BRAF V600E gene mutation in bone marrow / peripheral blood during treatment)
- Relapse rate (overall, by risk, by generation, by BRAFV600E gene mutation pattern at diagnosis, by BRAFV600E gene mutation pattern in bone marrow at diagnosis, by peripheral blood BRAFV600E gene mutation pattern at diagnosis, cases in which GR / PR was obtained 6 weeks after the start of treatment, by reduction rate of BRAF V600E gene mutation in bone marrow / peripheral blood during treatment)
-Disease assessment and response rate in each treatment phase (overall, by risk)
- Correlation between changes in LCH disease activity score (DAS) and clinical prognosis
- Incidence of central nervous system-related late complications (overall, by risk, by generation, by BRAFV600E gene mutation pattern at diagnosis, by BRAFV600E gene mutation pattern in bone marrow at diagnosis, by peripheral blood BRAFV600E gene mutation pattern at diagnosis, by Initial treatment responsiveness, by reduction rate of BRAF V600E gene mutation in bone marrow / peripheral blood during treatment)
- Incidence of adverse events due to CTCAE ver5.0
- Treatment completion rate, number of Intrathecal injections, number of zoledronic acid administration

+81-52-951-1111

Feb. 22, 2021

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