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

Starting soon · Phase 2 · Has a placebo group

Conditions studied: Osteomyelitis Chronic

In brief

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.

Key facts

Study ID: NCT07668583
Run by: Assistance Publique - Hôpitaux de Paris
People needed: 30
Starts: 2026-09-15
Expected to finish: 2029-12-31
Last updated by the study team: 2026-06-25

Who can join

Age: 4 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.

Where it is running

Kremlin-Bicêtre Hospital — Le Kremlin-Bicêtre, Val De Marne, France

Full record on ClinicalTrials.gov

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