Using Denosumab to treat Fibrous Dysplasia and McCune-Albright Syndrome in adults
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD): a Randomized Double-blind Placebo-controlled Trial
This study is testing if Denosumab can help adults with Fibrous Dysplasia and McCune-Albright Syndrome feel less pain and improve their condition compared to a placebo.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 82 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Leiden University Medical Center Academic / other |
| Drugs / interventions | Denosumab |
| Locations | 1 site (Leiden) |
| Trial ID | NCT05966064 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are adults over 18 with symptomatic Fibrous Dysplasia or McCune-Albright Syndrome who experience significant pain not relieved by standard treatments.
Not a fit: Patients with pain unrelated to Fibrous Dysplasia or those with uncontrolled endocrine diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce pain and improve quality of life for patients suffering from Fibrous Dysplasia and McCune-Albright Syndrome.
How similar studies have performed: Previous studies have shown promising results with Denosumab in similar conditions, suggesting potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Leiden
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.