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DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial

Phase 4
Recruiting
Conditions
Fibrous Dysplasia/McCune Albright syndrome
10028396
Registration Number
NL-OMON53469
Lead Sponsor
eids Universitair Medisch Centrum
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
Not specified
Target Recruitment
82
Inclusion Criteria

- Being symptomatic with an 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 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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>The effect of Dmab on pain, assessed by the difference in maximum pain score<br /><br>after 6 months (2 injections) by Brief Pain Inventory</p><br>
Secondary Outcome Measures
NameTimeMethod
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