Safety and Tolerability of I-040302 in Children and Young Adults With Solitary Bone Cysts

Study Overview
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Brief Summary
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This is a randomised, controlled, open-label study of a single intralesional administration of I-040302 or a single bone marrow aspirate or a single steroid injection (methylprednisolone). Subjects will undergo screening with X-ray and magnetic resonance imaging (MRI) in the diagnosis of a solitary bone cyst.

Official Title
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A Phase 2 Randomised, Open-label, Multi-centre Ascending Dose Study of the Efficacy, Safety and Tolerability of I-040302 Versus Control Injection (Bone Marrow Aspirate or Steroids) in Children and Young Adults With Solitary Bone Cysts

Conditions
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Bone Cysts

Intervention / Treatment
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* Drug: I-040302
* Other: Standard of Care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects 6 - 16 years of age Diagnostic evidence of unicameral bone cysts based on one or more of a combination of any of the following methods: Cyst fluid examination Plain radiographs MRI Subjects with the following types of cysts: Intact cysts that are either growing or at risk of fracture, requiring surgical intervention Persistence of a cyst cavity following cyst fracture that has received conservative treatment/s to repair the fracture Persistence of a cyst cavity despite repeated interventions Subjects with bone cyst volumes < 30 mL Subjects must be appropriately communicative to verbalise pain. Subjects must be able to understand and be willing to comply with the protocol procedures. Subjects who have provided written informed consent to participate in the study The legally authorised representative of the child must give written informed consent after the child has agreed to participate in the study. Exclusion Criteria: A history of/or presence of active cancer Family history of retinoblastoma Indication of aneurysmal bone cyst on MRI, cyst aspiration or histology Possible presence of osteosarcoma or uncertain histology Systemic or localised infection at time of surgery Evidence of immune-suppression Evidence of hypercalcemia Cyst volume > 30 mL Fracture present in the cortical bone surrounding the cyst Evidence of rapid venous drainage from the cyst determined by rapid disappearance of X-ray contrast medium during the surgical process. Suspected or known evidence of allergic reactions towards any of the components of I-040302 Known clinically significant organ or systemic diseases such that, in the opinion of the investigator, the significance of the disease will compromise the subject's participation in the study Pregnant or lactating females Participation in another clinical trial within 3 months prior to trial start

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>I-040302 doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34 | Drug: I-040302<br>* doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34<br>|
| Active Comparator: 2<br>Standard of care (bone marrow aspirate or steroids) | Other: Standard of Care<br>* Bone marrow aspirate or steroids<br>* Other names: Bone marrow aspirate or steroids;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Radiographic images |  | At 6 months and throughout the study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blood parameters |  | At 6 months and throughout the study | 
| Vital signs |  | At 6 months and throughout the study | 
| Questionnaires of life questionnaire |  | At 6 months and throughout the study | 
| Pharmacoeconomic evaluation |  | At 6 months and throughout the study | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Osteogenic Gel, Solitary Bone Cysts