Clinical trial NCT06078969: Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence
Official title: The Efficacy of Oral Prednisone in Treating Langerhans Cell Histiocytosis of Bone in Childhood and Adolescence: A Multi-center, Open-label, Randomized-controlled, Phase II Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Langerhans Cell Histiocytosis of Bone
Keywords: Langerhans cell histiocytosis
Bone
Single-system
Children
Adolescents
Lead sponsor: Shanghai Changzheng Hospital
Sponsor class: OTHER
Collaborators: name: First Affiliated Hospital of Zhejiang University
name: Peking University Third Hospital
name: National Sun Yat-sen University
name: Beijing Children's Hospital
name: Henan Cancer Hospital
name: Tianjin Hospital
name: First Affiliated Hospital, Sun Yat-Sen University
name: Second Hospital of Jilin University
name: Peking University People's Hospital
name: Jinling Hospital, China
name: The First Affiliated Hospital of Nanchang University
name: The Third Affiliated Hospital of Southern Medical University
First posted: 2023-10-12
Enrollment: 118
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).
Primary purpose: TREATMENT

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 \[Control group\].


Eligibility criteria: 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.

Primary outcomes:
measure: Progression-free survival
timeFrame: 2 years
Secondary outcomes:
measure: Local control rate
timeFrame: 2 years
measure: Pain relief
timeFrame: 2 years
measure: Adverse effect rate
timeFrame: 2 years
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Shanghai Changzheng Hospital
state: Shanghai
Study officials:
References:
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citation: Tang X, Gao J, Ma ZG, Guo X, Li Q, Wan Z, Sun JJ. Clinical and prognostic characteristics of 95 cases of Langerhans cell histiocytosis in children: a single-institute experience from 2013 to 2020. Ann Med. 2021 Dec;53(1):1537-1546. doi: 10.1080/07853890.2021.1966085.