Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis

• Status: Active, not recruiting
• Trial end date: 2023-11-01
• Target enrollment: 0
• Participant gender: All

Summary

The study is looking at the efficacy of subcutaneously administrated denosumab 60 mg every 6 months versus placebo after 3 years, by analyze of lumbar spine bone mineral density (BMD) in systemic mastocytosis.

Investigators hypothesize that use of denosumab subcutaneously in patients with osteoporosis related to systemic mastocytosis is effective and safe to improve bone mineral density and prevent new bone events, based on targeted specific RANKL secretion by mast cells and short half-life of denosumab.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Collaborator: CEREMAST
• Treatments: Denosumab

Criteria

Inclusion Criteria:

• Male or female >/= 18 years of age at time of informed consent

• Willingness and ability to sign informed consent, comply with scheduled visits, treatment plan, laboratory tests and other study procedures.

• Patient with Indolent systemic or cutaneous mastocytosis according to WHO criteria (Appendix 4) with any specific treatment including corticosteroid, chemotherapy and immunomodulating drugs.

• Patient with:

• osteoporosis defined as bone mineral density T score ≤ -2.5 at the lumbar spine, OR

• osteopenia defined as BMD T-score >-2,5 and ≤ -1 at the lumbar spine and low energy fracture (defined as fractures that are associated with decreased bone mineral density. Are excluded fractures of skull, face, mandible, metacarpals, fingers, or toes, pathologic fracture, and fracture that are associated with severe trauma).

(in case of osteoarthritis at the lumbar spine, the T score at left femoral neck or total left hip can be used to define osteoporosis or osteopenia)

Exclusion Criteria:

• Patient with aggressive mastocytosis or/and Associated Hematologic Non-Mastocytosis Disease (AHNMD)

• Patient with conditions that influence bone metabolism (primitive hyperparathyroidism, hyperaldosteronism, hypercorticism, etc …)

• Patient treated with intravenous bisphosphonate within 1 year prior to enrolment or with any other antiosteoporotic treatment within 3 months before enrolment. (per os bisphosphonate, strontium ranelate) Calcium and vitamin supplementation will be accepted

• Patient previously treated with denosumab

• Patient with hypocalcemia and/or hypo25-hydroxyvitamin D level non substituted prior enrolment

• Woman without contraceptive treatment if of childbearing age.

• Pregnant or breastfeeding woman

• Patient with contraindication to denosumab

• Patient with medical, psychiatric or other conditions that may interfere with patient safety

• Patient with dental problem that need any dental surgery within 6 months after enrolment.

• Patient with clearance of creatinine less than 30 mL/min/1,73m2 (MDRD) or patient receiving dialysis