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

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

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.

Study Overview

• Status: Withdrawn
• Conditions: Bone Cysts
• Intervention / Treatment: Drug: I-040302; Other: Standard of Care

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Kuros Biosurgery AG
• Investigators: Study Director: Virginia Jamieson, MD, Kuros Biosurgery AG, Technoparkstrasse 1, CH-8005 Zurich

Study record dates

Study Major Dates

• Study Start: December 1, 2012
• Primary Completion (Anticipated): June 1, 2014
• Study Completion (Anticipated): July 1, 2019

Study Registration Dates

• First Submitted: April 11, 2007
• First Submitted That Met QC Criteria: April 11, 2007
• First Posted (Estimate): April 12, 2007

Study Record Updates

• Last Update Posted (Estimate): July 17, 2012
• Last Update Submitted That Met QC Criteria: July 16, 2012
• Last Verified: July 1, 2012

More Information

Terms related to this study

• Keywords: Osteogenic Gel; Solitary Bone Cysts
• Additional Relevant MeSH Terms: Neoplasms; Musculoskeletal Diseases; Pathological Conditions, Anatomical; Bone Diseases; Cysts; Bone Cysts
• Other Study ID Numbers: CS I-040302/01