Polyostotic fibrous dysplasia

Summary

Polyostotic fibrous dysplasia (MONDO:0008274) is a disease and 14 clinical trials. Top therapeutic interventions include alendronic acid, burosumab, and pegvisomant. A subtype of fibrous dysplasia — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Clinical trials: 14

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	polyostotic fibrous dysplasia
Mondo ID	MONDO:0008274
MeSH	D005359
Orphanet	93276
ICD-10-CM	Q78.1
ICD-11	771587091
NCIT	C34610
SNOMED CT	36517007
UMLS	C0016065
MedGen	5180
GARD	0004213
MedDRA	10036120
Is cancer (heuristic)	no

Also known as: fibrous dysplasia of bone · PFD · polyostotic fibrous dysplasia of bone

Data availability: 1 cell line.

Disease family

This is a subtype of fibrous dysplasia. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by body system or component › musculoskeletal system disorder › skeletal system disorder › bone disorder › bone remodeling disease › fibrous dysplasia › polyostotic fibrous dysplasia

Related subtypes (2): monostotic fibrous dysplasia, panostotic fibrous dysplasia

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

Drugs indicated for this disease

0 approved, 1 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Pegvisomant	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Alendronic Acid, Anastrozole, Fulvestrant.

Clinical trials & evidence

Clinical trials

Clinical trials: 14.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	7
PHASE2	4
PHASE3	1
PHASE2/PHASE3	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00017927	PHASE3	COMPLETED	A Study of the Effects of Pegvisomant on Growth Hormone Excess in McCune-Albright Syndrome
NCT00445575	PHASE2/PHASE3	COMPLETED	Effect of Risedronate on Bone Morbidity in Fibrous Dysplasia of Bone
NCT00001181	PHASE2	COMPLETED	Testolactone for the Treatment of Girls With LHRH Resistant Precocious Puberty
NCT00001728	PHASE2	COMPLETED	Alendronate to Treat Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
NCT01791842	PHASE2	COMPLETED	TOCILIZUMAB IN FIBROUS DYSPLASIA OF BONE
NCT05509595	PHASE2	COMPLETED	Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia
NCT00006174	PHASE1	COMPLETED	Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
NCT07476768	Not specified	NOT_YET_RECRUITING	PAINDYS_Characterizing Pain in Fibrous Dysplasia of Bone/McCune-Albright Syndrome: an Exploratory Pilot Study
NCT07569731	Not specified	RECRUITING	Fibrous Dysplasia: An Epidemiological and Correlational Evaluation of Multimodal Data
NCT00001851	Not specified	COMPLETED	Bone Marrow Injection to Replace Diseased Bone in Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
NCT00001973	Not specified	COMPLETED	Studies on Abnormal Bone From Patients With Polyostotic Fibrous Dysplasia and McCune Albright Syndrome
NCT03838991	Not specified	COMPLETED	Epigenetic Regulation in Fibrous Dysplasia of Bone: mirDYS Study.
NCT05422833	Not specified	COMPLETED	Effectiveness of Medical Management of Fibrous Dysplasia of Bone.
NCT06177327	Not specified	UNKNOWN	Hepato-pancreato-biliary Abnormalities in Fibrous Dysplasia of Bone/McCune Albright Syndrome

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
ALENDRONIC ACID	4	3
BUROSUMAB	4	1
PEGVISOMANT	4	1
RISEDRONIC ACID	4	1
TESTOLACTONE	4	1
CHEMBL166839	0	1