Rare Endocrinology News

Spotlight On

Acromegaly

Acromegaly is a hormonal disorder that results from the pituitary gland producing too much growth hormone (GH)

Prevalence

5-7/100,000

16,550-23,170

US Estimated

25,675-35,945

Europe Estimated

Age of Onset

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ICD-10

E22.0

Inheritance

Autosomal dominant

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Autosomal recessive

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Mitochondrial/Multigenic

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X-linked dominant

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X-linked recessive

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Rare View

Acromegaly is a disorder that results from excess growth hormone (GH) after the growth plates have closed. The initial symptom is typically enlargement of the hands and feet. There may also be an enlargement of the forehead, jaw, and nose. Acromegaly is usually caused by the pituitary gland producing excess growth hormone. In more than 95% of cases the excess production is due to a benign tumor, known as a pituitary adenoma.

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5 Facts you should know

FACT

1

A disorder that results from excess growth hormone (GH) after the growth plates have closed.

FACT

2

The initial symptom is typically enlargement of the hands and feet.

FACT

3

There may also be an enlargement of the forehead, jaw, and nose.

FACT

4

Other symptoms may include joint pain, thicker skin, deepening of the voice, headaches, and problems with vision.

FACT

5

Complications of the disease may include type 2 diabetes, sleep apnea, and high blood pressure.

Acromegaly is also known as...

Acromegaly

Somatotroph adenoma; Growth hormone excess; Pituitary giant

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Common signs & symptoms

Arthralgia

Joint pain

Broad foot

Wide foot

Broad forehead

Increased width of the forehead

Cortical diaphyseal thickening of the upper limbs

Deep palmar crease

Deep palm line

Deep plantar creases

Deep wrinkles in soles of feet

Fatigue

Growth hormone excess

Current treatments

Octreotide

(Brand name: Sandostatin LAR)- Manufactured by Novartis Pharmaceuticals Corporation
FDA-approved indication: Reduction of growth hormone and IGF-1 (somatomedin C) in acromegaly.

Pasireotide

(Brand name: Signifor) - Manufactured by Novartis Pharmaceuticals Corporation
FDA-approved indication: June 2018, approved for the treatment of patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative. Also, December 2014, approved for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.

Lanreotide

(Brand name: Somatuline® Depot (injection)) - Manufactured by IPSEN, Inc.
FDA-approved indication: Long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy.

Pegvisomant

(Brand name: Somavert®) - Manufactured by Pfizer
FDA-approved indication: Treatment of acromegaly in patients who have had an inadequate response to surgery and/or radiation therapy and/or other medical therapies, or for whom these therapies are not appropriate

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsMore Information
A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-2) (PATHFNDR-2)A randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with non-pharmacologically treated acromegaly.Phase 3RecruitingDrug: Paltusotine
Drug: Placebo
More Info
Acromegaly Combination Treatment StudyIn this study the investigators will evaluate whether combination low dose somatostatin receptor ligand (SRL) and weekly or daily Pegvisomant will attain equivalent control of serum IGF-1 levels at a lower cost, compared to combination high dose SRL and weekly Pegvisomant.N/ARecruitingDrug: Pegvisomant
Drug: Octreotide LAR
Drug: Lanreotide
More Info
Safety and Efficacy of Pegvisomant in Children With Growth Hormone ExcessFor children with gigantism, too much growth hormone (GH) in the body causes abnormal growth and many other problems. Current treatments often don't work; no medical treatment is approved by FDA. Researchers want to see if the drug pegvisomant can help.Phase 3RecruitingDrug: PegvisomantMore Info
A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With AcromegalyThe purpose of this trial is to assess the long-term safety of CAM2029 in patients with acromegaly.Phase 3RecruitingDrug: CAM2029 (octreotide subcutaneous depot)More Info
A Trial to Assess Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With AcromegalyThe purpose of this trial is to assess the efficacy and safety of CAM2029 in patients with acromegaly.Phase 3RecruitingDrug: CAM2029 (octreotide subcutaneous depot)
Drug: Matching placebo
More Info
A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of AcromegalyA randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with acromegaly previously treated with somatostatin receptor ligand (SRL) based treatment regimens.Phase 3RecruitingDrug: Paltusotine
Drug: Placebo
More Info
A Study to Evaluate the Long-Term Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (ACROBAT Advance)A phase 2, open label, long-term extension study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with acromegaly.Phase 2Enrolling by invitationDrug: PaltusotineMore Info

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyClinical StudiesMore Information
PaltusotinePaltusotine is a promising once-daily oral nonpeptide SST2 agonist candidate for managing acromegaly and neuroendocrine tumors.Phase 3Crinetics Pharmaceuticals Inc.More InfoMore Info
CAM2029A new long-acting investigational formulation of octreotide, an octapeptide that mimics natural somatostatin pharmacologically.Phase 3CamurusMore InfoMore Info

Genetic and Rare Diseases Information Center (GARD) - PO Box 8126, Gaithersburg, MD 20898-8126 https://rarediseases.info.nih.gov