Pituitary dwarfism

Summary

Pituitary dwarfism (MONDO:0006909) is a disease and 4 clinical trials. Top therapeutic interventions include somatropin. A subtype of adrenal gland disorder — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Clinical trials: 4

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	pituitary dwarfism
Mondo ID	MONDO:0006909
EFO	EFO:1001109
MeSH	D004393
SNOMED CT	367460001
UMLS	C0013338
MedGen	8506
MedDRA	10035083
Is cancer (heuristic)	no

Disease family

This is a subtype of adrenal gland disorder. 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 › endocrine system disorder › adrenal gland disorder › pituitary dwarfism

Related subtypes (17): medulloadrenal hyperfunction, adrenal cortex disorder, adrenal medullary hyperplasia, pseudoleprechaunism syndrome, Patterson type, apparent mineralocorticoid excess, autoimmune polyendocrine syndrome type 1, adrenomyodystrophy, corticosteroid-binding globulin deficiency, Congenital adrenal insufficiency with 46, XY sex reversal OR 46,XY disorder of sex development-adrenal insufficiency due to CYP11A1 deficiency, adrenogenital syndrome, hypoaldosteronism disease, primary pigmented nodular adrenocortical disease, adrenoleukodystrophy, adrenal gland neoplasm, ectopic ACTH secretion syndrome, endogenous Cushing syndrome, isolated micronodular adrenocortical disease

Subtypes (2): short stature due to partial GHR deficiency, psychosocial short stature

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

5 approved, 6 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Arginine Hydrochloride	Approved (phase 4)
Lonapegsomatropin	Approved (phase 4)
Somapacitan	Approved (phase 4)
Somatrogon	Approved (phase 4)
Somatropin	Approved (phase 4)
Albusomatropin	Phase 3 (in late-stage trials)
Arginine	Phase 3 (in late-stage trials)
Atropine	Phase 3 (in late-stage trials)
Clonidine	Phase 3 (in late-stage trials)
Macimorelin	Phase 3 (in late-stage trials)
Somavaratan	Phase 3 (in late-stage trials)

Clinical trials & evidence

Clinical trials

Clinical trials: 4.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE3	2
Not specified	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00102817	PHASE3	COMPLETED	Somatropin (Norditropin) in Insulin-like Growth Factor (IGF) Deficient Children
NCT00109733	PHASE3	COMPLETED	Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency
NCT01009905	Not specified	COMPLETED	An Observational Study (Registry) Assessing Treatment Outcomes and Safety for Children and Adults Who Are Prescribed Norditropin® (Human Growth Hormone)
NCT01504802	Not specified	COMPLETED	Pharmacodynamics of CNP During Growth Hormone Treatment

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
SOMATROPIN	4	3