Using Denosumab to treat Fibrous Dysplasia and McCune-Albright Syndrome in adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial investigates the efficacy of Denosumab, a RANKL-inhibitor, in improving the clinical, radiological, and biochemical manifestations of Fibrous Dysplasia and McCune-Albright Syndrome in adults. Patients will be randomized to receive either Denosumab or a placebo, with treatment administered every three months. The study aims to assess pain levels and lesional growth, allowing patients with inadequate pain relief to continue with Denosumab in an open-label phase after six months. The trial seeks to provide a potential new treatment option for a condition that currently lacks effective therapies.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Symptomatic patients with established diagnosis of FD/MAS and closed growth plates(\>18 years)
* Pain in the region of an FD localization, not responding to adequate pain treatment and without mechanical component e.g. impending fracture
* Pain score from FD lesion for maximum or average pain on VAS ≥ 4
* Increased lesional activity defined as increased bone turnover markers (ALP, P1NP or CTX) or increased activity on Na\[18F\]-PET/CT or bone scintigraphy in at least one lesion
* Normal levels of calcium, parathyroid hormone and vitamin D (supplementation is allowed)
* Treated hypophosphatemia (defined as \>0.7 at two separate measures)
* good dental health (last check within the last 12 months)

Exclusion Criteria:

* Active pregnancy wish, pregnancy or nursing
* Pain not related to FD
* Uncontrolled endocrine disease
* Untreated vitamin D deficiency, hypocalcemia or hypophosphatemia
* Previous use of bisphosphonates or Dmab \< 6 months before inclusion ('6 months wash out')
* Previously reported severe side effects on Dmab
* Inability to fulfil study requirements
* Poor untreated dental health without intention to get treatment
* Treatment with other bone influencing drugs, such as high doses corticosteroids

Where it's running

• Leiden University Medical Center
  Leiden, Netherlands Recruiting

Study contacts

• Principal investigator: Natasha Appelman-Dijsktra, MD, PhD — Leiden University Medical Center
• Study coordinator: Natasha Appelman-Dijkstra, MD, PhD
• Email: n.m.appelman-dijkstra@lumc.nl
• Phone: +31 625301410

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.