Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence

Phase 2

Recruiting

• Conditions: Langerhans Cell Histiocytosis of Bone
• Interventions: Drug: Prednisone

• Registration Number: NCT06078969
• Lead Sponsor: Shanghai Changzheng Hospital

Brief Summary

Langerhans cell histiocytosis (LCH) of bone is a benign-tumor-like osteolytic lesion in childhood and adolescence, which is characterized by the aberrant activation of antigen presenting cells. Rather than the multi-system involvements of LCH, no standard or widely-accepted therapeutic regimens were established for LCH of bone. In the previous clinical practice, several LCH patients obtained remarkable pain relief after taking prednisone. Therefore, the investigators aim to conducting a multi-center, open-labelled, randomized-controlled, Phase II study to investigate the efficacy and safety of oral prednisone in treating LCH of bone in children and adolescents. The enrolled patients will be randomly recruited to the following groups: (1) Oral prednisone \[Test group); (2) Regular observation \[Control group\].

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-09-27
• Study First Submitted QC: 2023-10-09
• Study First Posted: 2023-10-12
• Study Start: 2023-11-04
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-31
• Last Update Posted: 2024-02-01
• Primary Completion: 2027-09-30
• Study Completion: 2027-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 118

Inclusion Criteria

• Pathological diagnosis of Langerhans cell histiocytosis (LCH) of bone;
• Single-system involvement (skeletal system);
• No need of surgical intervention;
• Must be able to swallow tablets;
• Signing informed consent form.

Exclusion Criteria

• Multi-system involvements (≥2 systems, including bone, liver, spleen, hematologic system, central nerve system);
• Need of surgical intervention (e.g. pathological fracture and/or spinal cord compression)
• Glucocorticoid allergy;
• Immunodeficiency;
• Severe infection;
• Insulin dependent/independent Diabetes;
• Having taken glucocorticoid in the past two weeks;
• Not capable of swallowing tablets;
• Without signed informed consent inform.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Oral prednisone	Prednisone	Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months

Primary Outcome Measures

Name	Time	Method
Progression-free survival	2 years	Any evidence proving the disease progression

Secondary Outcome Measures

Name	Time	Method
Local control rate	2 years	radiological examinations of local lesion status
Adverse effect rate	2 years	side effects related to the prednisone
Pain relief	2 years	The Visual Analogue Scale (Minimum: 0; Maximum: 10 scores), is used to evaluate the degree of pain relief. The higher scores mean a worse outcome.

Trial Locations

Locations (1)

Shanghai Changzheng Hospital; 🇨🇳 Shanghai, Shanghai, China