EFFICACY AND SAFETY OF ZOLEDRONATE VERSUS PLACEBO ON PAIN AT WEEK 12 IN PEDIATRIC PATIENTS WITH CHRONIC RECURRENT MULTIFOCAL OSTEOMYELITIS

NCT07668583 · tracked via the Priya Life Science France tracker

Sponsor

Assistance Publique - Hôpitaux de Paris

Phase

Phase 2

Started

2026-09-15

Last updated

2026-06-25

Condition(s) studied

Osteomyelitis Chronic

Investigational drug(s) / intervention(s)

Zoledronic Acid / Zoledronate 4 MG/100 ML Intravenous SolutionNaCL 0.9% Intravenous Solution

Zoledronic Acid / Zoledronate 4 MG/100 ML Intravenous Solution: Baseline : 0.025 mg/kg ; Week 4 : 0.05 mg/kg ; Week 6 : 0.05 mg/kg

NaCL 0.9% Intravenous Solution: Baseline, Week 4 \& Week 6

Study summary

Chronic recurrent multifocal osteomyelitis (CRMO) is a rare auto-inflammatory bone disease that primarily affects children and/or adolescents at a median age of 10 years. Until now, there is no consensus regarding the treatment of CRMO. Non-steroidal anti-inflammatory drugs (NSAIDs) are considered the first line of therapy with remission in approximately 30% of cases. If unsuccessful, several treatments are tried in addition to NSAIDs, including bisphosphonates and anti-TNFs.

The effectiveness of bisphosphonates (including zoledronate) has been reported in clinical cases and/or retrospective series. They are said to be particularly effective in multifocal forms, mandibular and/or vertebral involvement, but no controlled trials have been conducted. Bisphosphonates have even been proposed as first-line therapy in spinal involvement. The only prospective study, is a phase II trial currently underway in Denmark to study the efficacy of zoledronate (NCT02594878) versus placebo in SAPHO (acronym, standing for Synovitis - Acne - Pustulosis - Hyperostosis - Osteitis) patients considered to be a very similar form of CRMO occurring in adults.

In this context, this study proposes evaluate the efficacy of zoledronate compared to placebo in reducing pain at week 12 in children aged ≥4 and \<17 years with NSAID-resistant CRMO. Zoledronate will be administered in three escalating doses: 0.025 mg/kg at baseline (W0), 0.05 mg/kg at week 12 (W12), and 0.05 mg/kg at week 24 (W24). In addition to pain reduction, improvements in MRI findings will be observed, biological markers of inflammation, and quality of life in the zoledronate group. Although subjective, pain reduction remains the most widely used criterion in clinical practice to assess therapeutic efficacy. Zoledronate efficacy will therefore be assessed by the change in standardized pain score (0-10 scale) from baseline to week 12 as the primary endpoint, with additional pain assessments at weeks 4, 24, and 36 as secondary endpoints.

Eligibility

Sex

ALL

Min age

4 Years

Max age

17 Years

Healthy volunteers

Inclusion Criteria: * aged ≥4 and \<17 years for whom: * Physician-confirmed diagnosis of CRMO according to Jansson's criteria, with compatible MRI findings. Having lesions on MRI within 12 weeks prior to inclusion and clinically active disease defined by at least one of 2 criteria: patient/parent VAS (pain) superior or equal to 30/100 and/or physician VAS superior or equal to 30/100 after failure of at least 4 weeks of NSAIDs at a stable dose * Written informed consent signed by the parents (the child may sign the consent if they wish, but their signature is not mandatory) * Having had a dental review within 3 months prior to inclusion, with completion of any necessary invasive dental work before the first dose of zoledronate. Exclusion Criteria: * History of malignancy or current tumour * History of seizure * Current infectious osteomyelitis * Contraindication to the study drug * Hypersensitivity to the active substance, to other bisphosphonates, or to any excipient (sodium hydroxide, hydrochloric acid for pH adjustment, water for injection) ; * Hypocalcemia ; * Severe renal impairment with creatinine clearance \< 35 ml/min ; * Prior treatment with bisphosphonates and/or biotherapy within 6 months prior to inclusion. * History of HIV, HBV, or HCV infection. * Congenital or acquired prolonged QT interval (\>0.44 seconds) on ECG. * Clinically significant vertebral deformities, including vertebral fracture and/or angular kyphosis with risk of spinal cord compression. * Suspected or confirmed tuberculosis. * History of renal or hepatic insufficiency. * Already enrolled in another interventional study. * Pregnant or breastfeeding participants * Not affiliated with the French social security system. * Patient or legal guardians with limited understanding of the French language * Serum 25-hydroxy vitamin D level \<30 ng/mL at screening. Participants may be re-screened after correction of vitamin D deficiency.

Primary outcome measure(s)

Change in pain score from baseline to week 12 — week 12
Change in standardized pain score (0-10 scale) from baseline to week 12, using Visual Analog Scale VAS 0-10/ 0 = no pain ; 10 = worst imaginable pain

Trial sites (1)

Facility	City	Region	Status
Kremlin-Bicêtre Hospital	Le Kremlin-Bicêtre	Val De Marne

More Assistance Publique - Hôpitaux de Paris trials in France

Fusion Ultrasound for Diagnosis and Monitoring of Endometriosis Lesions Active, not recruiting

neonAtal motoR paTtErn autoMatIc analySis Recruiting

Characterisation of Intestinal Microbiota of Patients With Spondyloarthritis or Rheumatoid Arthritis Recruiting

Effect of Adding Vasopressin on Cardiac Output, Blood Pressure, Diuresis, and Tissue Perfusion Indices in Patients With Septic Shock Starting soon

Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy : An Early Phase Study Recruiting

Other trials for the same condition

Evaluation of the Efficiency of the Bone Substitute Cerament-G Locally Delivering Gentamicin in the Treatment of Chronic Osteomyelitis of Long Bones Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07668583 on ClinicalTrials.gov ↗ ← All trials in France