DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD) (NCT05966064) | Clinical Trial Compass
RecruitingPhase 4
DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Netherlands82 participantsStarted 2023-06-13
Plain-language summary
Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease, consisting of the replacement of normal bone tissue with fibrous tissue. FD lesions may be isolated in one or more bones or may be associated with endocrinopathies in McCune-Albright syndrome. Bone lesions constitute of weak bone tissue, leading to higher risk of fractures, pain and decreased quality of life. There is no cure for FD lesions and current therapies failed to soothe patients' complaints or to display any effect on progression of the lesions on imaging. However, the RANKL-inhibitor Denosumab demonstrated encouraging results in mouse models and in off-label clinical use, leading to clinical, biochemical and radiographical improvements.
Study's aim is to investigate whether 3-monthly Denosumab will improve the clinical, radiological and biochemical manifestations of FD bone lesions.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Denosumab effect on maximal pain score
Timeframe: at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months