Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence
January 31, 2024 updated by: Jianru Xiao, Shanghai Changzheng Hospital
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
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].
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
118
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Shanghai
-
Shanghai, Shanghai, China, 200003
- Recruiting
- Shanghai Changzheng Hospital
-
Contact:
- Shaohui He, MD;PhD
- Phone Number: 086+18221376577
- Email: heshaohui1025@163.com
-
Contact:
- Chen Ye, MD
- Phone Number: 086+17621628515
- Email: chenye@usst.edu.cn
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
Accepts Healthy Volunteers
No
Description
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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
|
Experimental: Oral prednisone
Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months
|
For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)
|
|
No Intervention: Regular observation
Regular observation and follow up without medication
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Progression-free survival
Time Frame: 2 years
|
Any evidence proving the disease progression
|
2 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Local control rate
Time Frame: 2 years
|
radiological examinations of local lesion status
|
2 years
|
|
Pain relief
Time Frame: 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.
|
2 years
|
|
Adverse effect rate
Time Frame: 2 years
|
side effects related to the prednisone
|
2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 4, 2023
Primary Completion (Estimated)
September 30, 2027
Study Completion (Estimated)
September 30, 2027
Study Registration Dates
First Submitted
September 27, 2023
First Submitted That Met QC Criteria
October 9, 2023
First Posted (Actual)
October 12, 2023
Study Record Updates
Last Update Posted (Actual)
February 1, 2024
Last Update Submitted That Met QC Criteria
January 31, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Respiratory Tract Diseases
- Lymphatic Diseases
- Lung Diseases
- Lung Diseases, Interstitial
- Histiocytosis, Langerhans-Cell
- Histiocytosis
- Physiological Effects of Drugs
- Anti-Inflammatory Agents
- Antineoplastic Agents
- Glucocorticoids
- Hormones
- Hormones, Hormone Substitutes, and Hormone Antagonists
- Antineoplastic Agents, Hormonal
- Prednisone
Other Study ID Numbers
- 2023SL049
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
YES
IPD Sharing Time Frame
1 year
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- ICF
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
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