Langerhans Cell Histiocytosis of BonePhase 2NCT06078969
Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence
A Phase 2 study testing Prednisone in people with Langerhans Cell Histiocytosis of Bone, run by Shanghai Changzheng Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Langerhans Cell Histiocytosis of Bone
- What is being tested?
- Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 1 siteChina
- Study dates
- 4 Nov 2023to 30 Sep 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 9Show fewer
- Having taken glucocorticoid in the past two weeks;
- Not capable of swallowing tablets;
- Without signed informed consent inform.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Shanghai Changzheng Hospital
Shanghai, Shanghai Municipality, 200003
Shaohui He, MD;PhD, 086+18221376577, heshaohui1025@163.com
Chen Ye, MD, 086+17621628515, chenye@usst.edu.cn
About this study
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
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].
- Conditions
- Langerhans Cell Histiocytosis of Bone
- Being tested
- Prednisone
- Keywords
- Langerhans cell histiocytosis, Bone, Single-system, Children, Adolescents
- Medical subject headings
- Histiocytosis, Langerhans-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)
| Study group | Role | What they receive |
|---|---|---|
| Oral prednisone | Experimental | Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months Receives: Drug: Prednisone |
| Regular observation | No intervention | Regular observation and follow up without medication |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression-free survival
Any evidence proving the disease progression
Measured: 2 years
Secondary outcomes (3)
Local control rate
radiological examinations of local lesion status
Measured: 2 years
Pain relief
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.
Measured: 2 years
Adverse effect rate
side effects related to the prednisone
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2023
- Study start4 Nov 2023
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Langerhans Cell Histiocytosis of BoneStudies of the same condition, open and under way.
- More trials run by Shanghai Changzheng HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).
- Masking
- None (open label)
- Enrolment
- 118 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Changzheng Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- First Affiliated Hospital of Zhejiang University, Peking University Third Hospital, National Sun Yat-sen University, Beijing Children's Hospital, Henan Cancer Hospital, Tianjin Hospital, First Affiliated Hospital, Sun Yat-Sen University, Second Hospital of Jilin University, Peking University People's Hospital, Jinling Hospital, China, The First Affiliated Hospital of Nanchang University, The Third Affiliated Hospital of Southern Medical University
- Responsible party
- Principal investigator, Jianru Xiao, Shanghai Changzheng Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06078969
- Sponsor's protocol ID
- 2023SL049
- Status last verified
- Jan 2024
- First submitted
- 27 Sep 2023
- Last updated
- 1 Feb 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
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.
PubMed 1636041Gadner 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.
PubMed 23589673Mitchell 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.
PubMed 36176854Baptista 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.
PubMed 22052526Zhong 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.
PubMed 27923375Tang 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.
PubMed 34459688
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06078969
- Acronym / sponsor ID
- 2023SL049
- Conditions
- Langerhans Cell Histiocytosis of Bone
- Keywords
- Langerhans cell histiocytosis, Bone, Single-system, Children, Adolescents
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 118 participants (estimated)
- Lead sponsor
- Shanghai Changzheng HospitalUniversity, hospital or other
- Collaborators
- First Affiliated Hospital of Zhejiang University, Peking University Third Hospital, National Sun Yat-sen University, Beijing Children's Hospital, Henan Cancer Hospital, Tianjin Hospital, First Affiliated Hospital, Sun Yat-Sen University, Second Hospital of Jilin University, Peking University People's Hospital, Jinling Hospital, China, The First Affiliated Hospital of Nanchang University, The Third Affiliated Hospital of Southern Medical University
- Study start
- 4 Nov 2023 (actual)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 12 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).
| Arm | Type | Receives |
|---|---|---|
| Oral prednisone | Experimental | Drug: PrednisoneOral prednisone, 0.5mg/kg/day, first 5 days/month, 6months |
| Regular observation | No intervention | –Regular observation and follow up without medication |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PrednisoneFor the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months) | Drug | Oral prednisone |
Primary outcome
Progression-free survival
Time frame: 2 years
Any evidence proving the disease progression
3 secondary outcomes
Local control rate
Time frame: 2 years
Pain relief
Time frame: 2 years
Adverse effect rate
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2024.
- Background: PubMed 1636041Stull MA, Kransdorf MJ, Devaney KO. Langerhans cell histiocytosis of bone. Radiographics. 1992 Jul;12(4):801-23. doi: 10.1148/radiographics.12.4.1636041.
- Background: PubMed 23589673Gadner 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.
- Background: PubMed 36176854Mitchell 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.
- Background: PubMed 22052526Baptista 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.
- Background: PubMed 27923375Zhong 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.
- Background: PubMed 34459688Tang 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.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Prednisone in people with Langerhans Cell Histiocytosis of Bone, run by Shanghai Changzheng Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression-free survival (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06078969, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Feb 2024.