NCT05966064 DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
| NCT ID | NCT05966064 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Natasha Appelman-Dijkstra |
| Condition | Fibrous Dysplasia |
| Study Type | INTERVENTIONAL |
| Enrollment | 82 participants |
| Start Date | 2023-06-13 |
| Primary Completion | 2027-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 82 participants in total. It began in 2023-06-13 with a primary completion date of 2027-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
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
Contact & Investigator
Natasha Appelman-Dijsktra, MD, PhD
PRINCIPAL INVESTIGATOR
Leiden University Medical Center
Frequently Asked Questions
Who can join the NCT05966064 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Fibrous Dysplasia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05966064 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT05966064 currently recruiting?
Yes, NCT05966064 is actively recruiting participants. Contact the research team at n.m.appelman-dijkstra@lumc.nl for enrollment information.
Where is the NCT05966064 trial being conducted?
This trial is being conducted at Leiden, Netherlands.
Who is sponsoring the NCT05966064 clinical trial?
NCT05966064 is sponsored by Natasha Appelman-Dijkstra. The principal investigator is Natasha Appelman-Dijsktra, MD, PhD at Leiden University Medical Center. The trial plans to enroll 82 participants.