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 \[Control group\].

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

118

participants targeted

Target at P50-P75 for phase_2

Timeline

17mo left

Started Nov 2023

Typical duration for phase_2

Geographic Reach

1 country

1 active site

Status

recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

3.9 years study duration

Study Progress64%

Nov 2023Sep 2027

First Submitted

Initial submission to the registry

September 27, 2023

Completed

15 days until next milestone

First Posted

Study publicly available on registry

October 12, 2023

Completed

23 days until next milestone

Study Start

First participant enrolled

November 4, 2023

Completed

3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2027

Expected

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2027

Last Updated

February 1, 2024

Status Verified

January 1, 2024

Enrollment Period

3.9 years

First QC Date

September 27, 2023

Last Update Submit

January 31, 2024

Conditions

Langerhans Cell Histiocytosis of Bone

Keywords

Langerhans cell histiocytosisBoneSingle-systemChildrenAdolescents

Outcome Measures

Primary Outcomes (1)

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

Secondary Outcomes (3)

Local control rate
2 years
Pain relief
2 years
Adverse effect rate
2 years

Study Arms (2)

Oral prednisone

EXPERIMENTAL

Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months

Drug: Prednisone

Regular observation

NO INTERVENTION

Regular observation and follow up without medication

Interventions

PrednisoneDRUG

For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)

Oral prednisone

Eligibility Criteria

Age2 Years - 17 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

You may not qualify if:

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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Shanghai Changzheng Hospitallead
First Affiliated Hospital of Zhejiang Universitycollaborator
Peking University Third Hospitalcollaborator
National Sun Yat-sen Universitycollaborator
Beijing Children's Hospitalcollaborator
Henan Cancer Hospitalcollaborator
Tianjin Hospitalcollaborator
First Affiliated Hospital, Sun Yat-Sen Universitycollaborator
Second Hospital of Jilin Universitycollaborator
Peking University People's Hospitalcollaborator
Jinling Hospital, Chinacollaborator
The First Affiliated Hospital of Nanchang Universitycollaborator
The Third Affiliated Hospital of Southern Medical Universitycollaborator

Study Sites (1)

Shanghai Changzheng Hospital

Shanghai, Shanghai Municipality, 200003, China

RECRUITING

Related Publications (6)

Stull MA, Kransdorf MJ, Devaney KO. Langerhans cell histiocytosis of bone. Radiographics. 1992 Jul;12(4):801-23. doi: 10.1148/radiographics.12.4.1636041.
PMID: 1636041BACKGROUND
Gadner H, Minkov M, Grois N, Potschger U, Thiem E, Arico M, Astigarraga I, Braier J, Donadieu J, Henter JI, Janka-Schaub G, McClain KL, Weitzman S, Windebank K, Ladisch S; Histiocyte Society. Therapy prolongation improves outcome in multisystem Langerhans cell histiocytosis. Blood. 2013 Jun 20;121(25):5006-14. doi: 10.1182/blood-2012-09-455774. Epub 2013 Apr 15.
PMID: 23589673BACKGROUND
Mitchell P, Ezeokoli EU, Borici N, Schleh E, Montgomery N. Treatment and Outcomes of Unifocal and Multifocal Osseous Pelvic Langerhans Cell Histiocytosis Lesions in a Pediatric Population. Cureus. 2022 Aug 27;14(8):e28470. doi: 10.7759/cureus.28470. eCollection 2022 Aug.
PMID: 36176854BACKGROUND
Baptista AM, Camargo AF, de Camargo OP, Odone Filho V, Cassone AE. Does adjunctive chemotherapy reduce remission rates compared to cortisone alone in unifocal or multifocal histiocytosis of bone? Clin Orthop Relat Res. 2012 Mar;470(3):663-9. doi: 10.1007/s11999-011-2162-x.
PMID: 22052526BACKGROUND
Zhong N, Xu W, Meng T, Yang X, Yan W, Xiao J. The surgical strategy for eosinophilic granuloma of the pediatric cervical spine complicated with neurologic deficit and/or spinal instability. World J Surg Oncol. 2016 Dec 7;14(1):301. doi: 10.1186/s12957-016-1063-6.
PMID: 27923375BACKGROUND
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.
PMID: 34459688BACKGROUND

MeSH Terms

Conditions

Histiocytosis, Langerhans-Cell

Interventions

Prednisone

Condition Hierarchy (Ancestors)

Lung Diseases, InterstitialLung DiseasesRespiratory Tract DiseasesHistiocytosisLymphatic DiseasesHemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

PregnadienediolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic Compounds

Study Design

Study Type: interventional
Phase: phase 2
Allocation: RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: CROSSOVER
Sponsor Type: OTHER
Responsible Party: PRINCIPAL INVESTIGATOR
PI Title: Professor, Chief Surgeon

Study Record Dates

First Submitted

September 27, 2023

First Posted

October 12, 2023

Study Start

November 4, 2023

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Last Updated

February 1, 2024

Record last verified: 2024-01

Data Sharing

IPD Sharing: Will share
Shared Documents: STUDY PROTOCOL, ICF
Time Frame: 1 year

Locations

CN(1)

Brief Summary

Trial Health

Trial Health Score

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (3)

Study Arms (2)

Oral prednisone

Regular observation

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Publications (6)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Design

Study Record Dates

Data Sharing

Locations