NCT02677246

Denosumab for Prevention of Bone Complications After Bone Marrow Transplantation in Children

Phase 1 Trial of Denosumab for Prevention of Bone Complications After Allogenic Hematopoietic Stem Cell Transplantation in Children

• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: St. Justine's Hospital · Academic / Other
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

Children treated by bone marrow transplantation (BMT) experience bone toxicity. Those bone damages are caused by both a decrease of bone formation and an increase of bone destruction after BMT.For long term survivors, bone complications are major determinants of impaired quality of life. No standard treatment currently exists to prevent those bone injuries. Denosumab is a treatment which specifically blocks bone destruction for 4 to 6 months in adults. This trial will study whether it is safe to prescribe Denosumab to children after BMT in the aim of preventing bone complications.

Detailed description

Participant will receive one subcutaneous dose of Denosumab within 2 weeks after confirmation of bone marrow engraftment. The dose of treatment will be escalated between participants. Denosumab is supposed to be effective for several months (4 to 6 months). The biologic activity of Denosumab will be followed by the measure in the blood of a biomarker of bone destruction called CTX. This biomarker is supposed to decrease after Denosumab infusion, reflecting the blockade of bone destruction by the treatment. Bone density will be assessed by a radiologic test named DXA which a standard test. All blood and radiologic tests mandated by the study will be done at the same time as standard follow-up after bone marrow transplant. Thus, participants will not have extra visits at the outpatient clinic, or extra blood punctures, for the specific purpose of the study. Participants will be followed for 36 months after bone marrow transplantation.For experiencing graft versus host disease, a second dose of Denosumab will be allowed, followed by subsequent doses evry 4 to 6 months till a maximum of 4 doses within 24months after bone marrow transplantation.

Conditions

• Disorder Related to Bone Marrow Transplantation

Interventions

Type	Name	Description
DRUG	Denosumab	Denosumab, one sub-cutaneous infusion, given within 2 weeks after engraftment defined as neutrophils \> 0,5x10(9)/L and chimerism ≥ 10% of donor origin. Before starting Denosumab several requirements have to be met: 1) Calcemia and Phosphatemia in the normal ranges, 2) adequate Calcium and Vitamin D intakes according to age. Biological activity of Denosumab measured by CTX blood level, expected to last for 4 to 6 months after one infusion. Possibility of subsequent infusions at the same dose level, every 5 to 6 months, for patient remaining on corticotherapy, provided no DLT occurred and provided CTX blood level decreased under the required threshold, after the previous infusions of Denosumab

Timeline

• Start date: 2016-12-01
• Primary completion: 2018-09-04
• Completion: 2018-09-04
• First posted: 2016-02-09
• Last updated: 2018-09-06

Locations

1 site across 1 country: Canada