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

Bone Cysts Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT00459641
• Other study ID #: CS I-040302/01
• Status: Withdrawn
• Phase: Phase 2
• First received: April 11, 2007
• Last updated: July 16, 2012
• Start date: December 2012
• Est. completion date: July 2019

Study information

• Verified date: July 2012
• Source: Kuros Biosurgery AG
• Contact: n/a
• Is FDA regulated: No
• Health authority: Switzerland: EthikkommissionSwitzerland: SwissmedicItaly: National Institute of HealthItaly: The Italian Medicines AgencyUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyUnited Kingdom: National Health Service
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: July 2019
• Est. primary completion date: June 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 16 Years

Eligibility

Inclusion Criteria:

1. Subjects 6 - 16 years of age

2. 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

3. 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

4. Subjects with bone cyst volumes < 30 mL

5. Subjects must be appropriately communicative to verbalise pain.

6. Subjects must be able to understand and be willing to comply with the protocol procedures.

7. Subjects who have provided written informed consent to participate in the study

8. The legally authorised representative of the child must give written informed consent after the child has agreed to participate in the study.

Exclusion Criteria:

1. A history of/or presence of active cancer

2. Family history of retinoblastoma

3. Indication of aneurysmal bone cyst on MRI, cyst aspiration or histology

4. Possible presence of osteosarcoma or uncertain histology

5. Systemic or localised infection at time of surgery

6. Evidence of immune-suppression

7. Evidence of hypercalcemia

8. Cyst volume > 30 mL

9. Fracture present in the cortical bone surrounding the cyst

10. Evidence of rapid venous drainage from the cyst determined by rapid disappearance of X-ray contrast medium during the surgical process.

11. Suspected or known evidence of allergic reactions towards any of the components of I-040302

12. 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

13. Pregnant or lactating females

14. Participation in another clinical trial within 3 months prior to trial start

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bone Cysts
• Cysts

Intervention

Drug:
• I-040302
doses of up to 1 mg, 2 mg or 4 mg of TGplPTH1-34

Other:
• Standard of Care
Bone marrow aspirate or steroids

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Kuros Biosurgery AG

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Radiographic images		At 6 months and throughout the study	No
Secondary	Blood parameters		At 6 months and throughout the study	Yes
Secondary	Vital signs		At 6 months and throughout the study	Yes
Secondary	Questionnaires of life questionnaire		At 6 months and throughout the study	Yes
Secondary	Pharmacoeconomic evaluation		At 6 months and throughout the study	No