DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age18+
SponsorNatasha Appelman-Dijkstra
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
Active pregnancy wish, pregnancy or nursing
Pain not related to FD
Uncontrolled endocrine disease
Untreated vitamin D deficiency, hypocalcemia or hypophosphatemia
Trial design
Design model
Parallel
Treatments tested in this trial
Denosumab 120 Mg/1.7 Ml Inj
DrugDenosumab randomized at baseline and after 3 months at 6 and 9 months in case of open label
Placebo
Drugplacebo randomized at baseline and after 3 months
Treatment groups
82 Participants
are divided into 2 treatment groupsGroup A: DenosumabActive comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Denosumab effect on maximal pain score
Evaluation of maximal pain score changes after treatment, assessed by Brief Pain Inventory (scale 0 to 10; 0-no pain, 10 worst pain)
Time frame
at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 monthsSecondary outcomes
1
Denosumab effect on average pain scores
Evaluation of average pain score changes after treatment, assessed by Brief Pain Inventory (scale 0 to 10; 0-no pain,10 worst pain)
Time frame
at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months2
To evaluate the number of patients with 50% reduction of maximal pain (BPI)
Evaluation of the number of patients with 50% reduction of maximal pain score changes after treatment, asseses by Brief Pain Inventory (scale 0 to 10; 0-no pain, 10 worst pain)
Time frame
at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months3
Denosumab effect on quality of life
Evaluation of Denosumab effect on quality of life, assessed with validated questionnaire SF-36 (scale 0-100, higher scores indicate better health status)
Time frame
at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months4
Denosumab effect on average weekly pain score
Evaluation of Denosumab effect on average weekly pain score assessed through a pain diary with VAS score (scale 0 to 10)
Time frame
every week from baseline, through study completion, an average of 1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Natasha Appelman-Dijkstra
Lead sponsor
Leiden University Medical Center
Sponsor institution
Source ClinicalTrials.gov