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Status unknownNCT03259152D-GctUpdated Aug 24, 2017

Characteristics and Mechanism of Denosumab-treated Giant Cell Tumor of Bone

A Phase 3 interventional study of Denosumab in Giant Cell Tumor of Bone, sponsored by Hebei Medical University Third Hospital. Status unknown at 1 site in China. Open to participants aged 14 Years to 60 Years. Per ClinicalTrials.gov, last updated 2017-08-24.

Sponsored by Hebei Medical University Third Hospital · Phase 3, Interventional, and Prevention

The sponsor has not verified this record recently (last verified May 2017), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 3
Study type
Interventional
Enrollment
30
Allocation
Randomized
Ages
14 Years to 60 Years
Sex
All
01

Study summary

Giant cell tumor of bone (GCTb) is a primary, osteolytic, benign tumor of the bone. Surgery is the commonly used treatment. Discovery of RANKL and its human monoclonal antibody, denosumab, led to use of denosumab for treatment of GCT. The aim of this study was to evaluate clinical and pathological results of treatment of relapsed or refractoriness GCT with denosumab and to assess adverse effect profile and recurrence rate.

Read the detailed description

Giant cell tumor of bone (GCTb) is an aggressive, benign bone tumor. GCTb, which was first defined by Cooper and Travers, can produce pulmonary metastasis, albeit rarely (1-6%). GCTb constitutes 5% of primary bone tumors and 20% of benign bone tumors. Histologically, the tumour consists of a proliferation of mononuclear cells, accompanied by a population of non-neoplastic osteoclast-like giant cells and mononuclear osteoclast precursors. Currently, it is thought that proliferating neoplastic cells produce a number of cytokines and mediators, including the receptor activator of nuclear factor κ-B-ligand (RANK-RANKL) system, that recruit osteoclast precursors and induce their maturation into multinucleated osteoclast. The standard management of GCTb is based on surgery with several local adjuvant treatments like methacrylate cement, phenol or cryotherapy to reduce the risk of recurrence, while bisphosphonates are used in some cases to decrease bone resorption and for pain relief in inoperable tumours or metastatic disease. In the last 5 years the use of denosumab, a fully human monoclonal antibody already licensed for postmenopausal osteoporosis and prevention of skeletal related events in bone metastases from solid tumours, has been introduced in the treatment strategy of GCTb. In this study we examined the clinical, radiological, histological and underlying mechanism features of a series of GCTb, before and after denosumab administration, comparing baseline and resection specimens. Moreover, we examined the safety of the drug and on the angiogenesis through the determination of microvascular density (MVD).

02

Conditions studied

  • Giant Cell Tumor of Bone

Keywords

  • Giant cell tumor of bone
  • Denousmab
  • Pathologic
  • Clinical
  • Mechansim
03

Who can participate

Ages eligible
14 Years to 60 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Giant cell tumor of bone patients confirmed by clinical, medical imaging and Pathology.

Exclusion criteria

Exclusion Criteria:

  • (1) less than 14 patients; 2) pregnant patients; 3) A patient who receives other medications during treatment.
04

Study design

Phase
Phase 3
Primary purpose
Prevention
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
30 participants (estimated)

Study arms

  • Experimental
    Pre-Denosumab GctB

    Specimens obtained during biopsy

    Drug: Denosumab

  • Experimental
    Post-Denosumab GctB

    Specimen after administration of Denosumab

    Drug: Denosumab

Interventions

  • DrugDenosumab

    Denosumab (trade names Prolia and Xgeva) is a human monoclonal antibody for the treatment of osteoporosis, treatment-induced bone loss, metastases to bone, and giant cell tumor of bone.Denosumab is a RANKL inhibitor, which works by preventing the development of osteoclasts which are cells that break down bone.

    Also known as: Xgeva or Prolia

05

What researchers measure

Primary outcomes

  1. Molecular analysis (Immunohistochemistry for RANKL, RANK, OPG,Col-I, VEGF)

    For collagen RANKL (Receptor Activator for Nuclear Factor-κ B Ligand), RANK (Receptor Activator for Nuclear Factor-κ B), OPG (Osteoprotegerin), Col-I (type I Collagen), VEGF (Vascular Endothelial Growth Factor) immunohistochemistry, sections were deparaffinized, rehydrated, and immunostained with a SA1024 SABC-POD kit and Kit-0017 DAB detection kit. Briefly, antigen retrieval was performed, and endogenous peroxidases were then inactivated prior to incubation with primary antibodies overnight at 4°C. This was followed by incubation with a biotinylated secondary antibody and a streptavidin-biotin complex peroxidase solution. Diaminobenzidine (DAB) chromogen was applied and counterstained with hematoxylin for antibody detection. Images were captured by a microscope system at 400-magnification. The integrated optical density values of each factor were semiquantitatively analyzed using Imaging Pro Plus 6.0 software.

    Time frame: 6 month

  2. Molecular analysis (RT-PCR for RANKL, RANK, OPG,Col-I, VEGF)

    Tissures mRNA was extracted using TRIzol reagent (Invitrogen, Carlsbad, CA). The RNA concentration and quality were assessed using a Quawell Q5000 spectrophotometer (Quawell, San Jose, CA). Reverse transcription PCR was performed using a Gene Amp 7700 Sequence Detection System (Applied Biosystems, Foster City, CA) and custom-designed, validated primers for Col1α1, Col2α1, Aggrecan, MMP-13, and ADAMTS-4. GAPDH was used as the housekeeping gene. Relative gene expression changes were reported using the 2(-Delta Delta C(T)) method as previously described. The experiment was repeated in triplicate to ensure accuracy.

    Time frame: 6 month

Secondary outcomes

  1. Visual Analog Score - Pain evaluation

    Visual analog scale \[VAS\] is a measure of pain intensity. It is a continuous scale comprised of a horizontal (called horizontal visual analogue scale) or,vertical called vertical visual analog scale usually 10 cm or 100 mm length \[both the gradations are used\]. It is anchored by two verbal descriptors, one for each symptom extreme. For pain intensity, the scale is most commonly anchored by "no pain" (score of 0) and "pain as bad as it could be" or "worst imaginable pain" (score of 100 \[on 100-mm scale\]

    Time frame: 6 months

  2. Hematology test - Tartrate Resistant Acid Phosphatase

    Tartrate-resistant acid phosphatase, a bone resorption marker, is secreted from osteoclasts and this marker is reported to be high in patients with giant cell tumor of bone. We investigated the effects of denosumab and the usefulness of a tartrate-resistant acid phosphatase as a monitoring marker in the management of a refractory giant cell tumor of bone. Tartrate-resistant acid phosphatase secretion was measured in the patient's serum to monitor the response to denosumab, and a rapid normalization of the marker was observed after the first denosumab administration.

    Time frame: 6 months

  3. Follow-up for recrudescence

    Patients were followed up regularly for local or systemic tumor recurrence by X-ray, CT, MRI, ECT. The follow-up period was 3 months.

    Time frame: 6 month to 1 year

  4. Morphological change - HE (Hematoxylin-Eosin) staining

    For histological analysis of the adjacent intervertebral disc and fusion mass, the tissures of the Giant cell tumor of bone were fixed in 10% neutral buffered formalin, decalcified in 10% EDTA-2Na for 3 months, and then embedded in paraffin. They were subsequently cut into 5-mm sections with cationic slides. Slides of the tissures of the Giant cell tumor of bone were stained with H\&E and captured by a microscope system (BX53; Olympus, Tokyo, Japan).

    Time frame: 6 month

  5. Micro-vessel density or area by IHC - stained slides

    IHC - stained for VEGF images were used for microvessel density (MVD) and vascular bud relative area analysis. MVD was measured by counting the number of cartilage endplate vascular buds (an average of cephalic and caudal vascular buds). The ratio of vascular bud area to the total endplate area was measured for vascular bud relative area analysis using the grid method. MVD and vascular bud relative area analyses were repeated at least three times for enhanced accuracy

    Time frame: 6 month

  6. Imaging changes by X-ray, CT, MRI, ECT.

    The patients' clinical information, images from radiographs, CT and MRI before and after Denosumab-treatment were recorded and analyzed. Tumor volume was measured on coronal, transverse, and sagital MRI or CT scansof the lesion; and maximum height, width, and depth were recorded; and the volume was calculated using the formula of an ellopsoid mass volume = \[(π/6) × height × width × depth\]. If CT or MRI were not available, tumor volume was measured on two-plane radiograghs.

    Time frame: 6 month

Other outcomes

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    The CTCAE v.4 criteria was used to evaluate late toxicity for all patients. Toxicity scores were recalculated for patients treated before publication of CTCAE v4.0 scale.

    Time frame: 3 year

06

Study locations

1 of 1 sites recruiting
  • Zhuang Zhou
    Shijiazhuang, Hebei 050000, China
    • Zhuang Zhou, Ph.D · Contact · 39094572@qq.com · +86 18833130669
    • Guochuan Zhang, M.D. · Contact · +86 13932110889
    Recruiting
07

References and documents

Publications

  • Girolami I, Mancini I, Simoni A, Baldi GG, Simi L, Campanacci D, Beltrami G, Scoccianti G, D'Arienzo A, Capanna R, Franchi A. Denosumab treated giant cell tumour of bone: a morphological, immunohistochemical and molecular analysis of a series. J Clin Pathol. 2016 Mar;69(3):240-7. doi: 10.1136/jclinpath-2015-203248. Epub 2015 Sep 3. PubMed 26338802 ↗
  • Deveci MA, Paydas S, Gonlusen G, Ozkan C, Bicer OS, Tekin M. Clinical and pathological results of denosumab treatment for giant cell tumors of bone: Prospective study of 14 cases. Acta Orthop Traumatol Turc. 2017 Jan;51(1):1-6. doi: 10.1016/j.aott.2016.03.004. Epub 2016 Oct 24. PubMed 27784623 ↗
  • Rutkowski P, Ferrari S, Grimer RJ, Stalley PD, Dijkstra SP, Pienkowski A, Vaz G, Wunder JS, Seeger LL, Feng A, Roberts ZJ, Bach BA. Surgical downstaging in an open-label phase II trial of denosumab in patients with giant cell tumor of bone. Ann Surg Oncol. 2015 Sep;22(9):2860-8. doi: 10.1245/s10434-015-4634-9. Epub 2015 Jun 2. PubMed 26033180 ↗

Individual participant data

Plan to share: No — We have not yet planned to to make individual participant data (IPD) available to other researchers.

08

Registry details

Key details

Study ID
NCT03259152
Lead sponsor
Hebei Medical University Third Hospital
Responsible party
Sponsor
First posted
Aug 23, 2017
Start date
May 1, 2016
Primary completion
May 1, 2019 (estimated)
Completion
May 1, 2019 (estimated)
Last update
Aug 24, 2017

Study contacts

Zhuang Zhou, Ph.D
Contact
39094572@qq.com
+86 18833130669
Guochuan Zhang, M.D.
Contact
+86 13932110889
Zhuang Zhou, Ph.D
principal investigator · Hebei Medical University Third Hospital

Oversight

Data monitoring committee
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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