Pilot study of hydroxyurea and methotrexate for recurrent Langerhans cell histiocytosis

JRCT ID: jRCTs031220324

Registered date:08/09/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Langerhans cell histiocytosis
• Date of first enrollment: 08/09/2022
• Target sample size: 10
• Study type: Interventional
• Intervention(s): Treatment of patients with recurrent Langerhans cell histiocytosis with hydroxyurea and methotrexate for up to 48 weeks

Outcome(s)

• Primary Outcome: Rate of achieving Non-Active Disease (NAD) on physical and imaging findings associated with recurrent LCH lesions by the end of 24 weeks of study treatment

Secondary Outcome

1. Rate of treatment response at the end of 6 weeks of study treatment (GR, PR, SD, PD)
2. Rate of NAD, AD-r, AD-p, AD-s, and AD-p at the end of 12 weeks (week 13), 24 weeks (week 25), (week 32 [week 33]), (week 40 [week 41]), and 48 weeks (end of study drug administration) of study treatment (week 13)
3. Percentage of patients receiving concomitant methotrexate
4. Frequency of adverse events associated with hydroxyurea administration
5. Frequency of adverse events associated with concomitant administration of hydroxyurea and methotrexate
6. Number of days of hospitalization and outpatient visits during the study period
7. Swimmer plots of the patients
8. Relationship between blood concentration of hydroxyurea and achievement of NAD, AD-r, AD-s, and AD-p at the end of 12 weeks (week 13), 24 weeks (week 25), and 48 weeks (end of study drug administration) of study treatment

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: Not applicable
• Gender: Both

Include criteria

Relapsed patients with Langerhans cell histiocytosis
1. Capsule formulation must be able to be taken internally.
2. Histopathologically diagnosed with LCH at the time of initial or recurrent disease.
3. The LCH disease is recurrent (regardless of the number of recurrences). *Central diabetes insipidus alone is excluded.
4. Treatment for the recurrence has not been initiated (local treatment is excluded).
5. No LCH involvement of risk organs (liver, spleen, hematopoietic organs).
6. No hemophagocytic lymphohistiocytosis (HLH) complication at the time of relapse
7. Adequate hepatic, renal, cardiac, and pulmonary function at study entry.
8. Patients have a general status of 0 to 2 on the Performance status (PS) scale.

Exclude criteria

1. Cases with recurrence of Central diabetes insipidus alone
2. Cases with active infection
3. Patients with active multiple cancers
4. Pregnant or lactating women
5. Patients of childbearing potential
6. Patients with active bleeding at the time of enrollment
7. Patients with active CNS symptoms at the time of enrollment (excluding symptoms caused by LCH itself)
8.Patients who have received hydroxyurea for LCH
9. Other cases deemed inappropriate by the investigators/associate investigators.

Related Information

• Primary Sponsor: Shioda Yoko
• Source(s) of Monetary Support: National Center for Child Health and Development