DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)

Launched by NATASHA APPELMAN-DIJKSTRA · Jul 20, 2023

Keywords

ClinConnect Summary

The DeFiD clinical trial is examining whether a medication called Denosumab can help improve the symptoms and bone health of adults with Fibrous Dysplasia and McCune-Albright Syndrome (FD/MAS). These are rare conditions where normal bone is replaced by fibrous tissue, leading to weak bones, chronic pain, and a higher risk of fractures. Currently, there are no effective treatments to manage these issues, but Denosumab has shown promise in earlier studies. The goal of this trial is to see if receiving Denosumab every three months can make a difference in pain relief and improve bone health as seen on medical imaging.

To join the study, participants need to be adults over 18 years old who are experiencing significant pain from their condition that hasn’t improved with standard treatments. They also need to have certain blood markers indicating active disease. However, individuals who are pregnant, nursing, or have certain other health issues may not be eligible. Those who participate will receive regular doses of Denosumab and will be monitored closely for changes in their symptoms and bone health. This trial is actively recruiting participants, and it aims to provide new insights into treating this challenging condition.

Gender

ALL

Eligibility 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