Overview
Acromegaly is a rare endocrine disorder caused by chronic excessive secretion of growth hormone (GH), most commonly due to a benign pituitary adenoma (somatotropinoma). When GH overproduction occurs before the closure of the epiphyseal growth plates in children, it results in gigantism; when it occurs in adults after growth plate fusion, it leads to acromegaly. The condition is characterized by the gradual enlargement of the hands, feet, and facial features (including the jaw, nose, and brow ridge), as well as soft tissue swelling, joint pain, and thickening of the skin. The disease name derives from the Greek words for 'extremity' and 'enlargement.' Acromegaly affects multiple body systems. Cardiovascular complications include hypertension, cardiomyopathy, and increased risk of heart failure. Metabolic effects include insulin resistance and diabetes mellitus. Patients may also experience sleep apnea, headaches, visual field defects (due to pituitary tumor compression of the optic chiasm), hyperhidrosis, carpal tunnel syndrome, arthropathy, and fatigue. Colon polyps and an increased risk of colorectal neoplasia have also been reported. The disease is insidious, with an average delay of 7–10 years between symptom onset and diagnosis. Treatment options include transsphenoidal surgical resection of the pituitary adenoma, which is the first-line therapy for most patients. Medical therapies include somatostatin receptor ligands (such as octreotide and lanreotide), the GH receptor antagonist pegvisomant, and dopamine agonists (such as cabergoline) in selected cases. Stereotactic radiosurgery or conventional radiotherapy may be used when surgery and medical therapy are insufficient. The goal of treatment is to normalize GH and insulin-like growth factor 1 (IGF-1) levels, reduce tumor size, and manage comorbidities. With appropriate treatment, many of the complications can be controlled, though some features such as bony changes are irreversible.
Clinical phenotype terms— hover any for plain English:
Sporadic
Usually appears on its own, not inherited from a parent
Adult
Begins in adulthood (age 18 or older)
FDA & Trial Timeline
10 eventsBaşakşehir Çam & Sakura City Hospital
Debiopharm International SA — PHASE3
Alexion Pharmaceuticals, Inc. — PHASE2
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
IRCCS San Raffaele
IRCCS San Raffaele
Erasmus Medical Center — NA
IRCCS San Raffaele
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
4 availableSandostatin LAR
Long-term maintenance therapy in acromegalic patients who have had an inadequate response to surgery and/or radiotherapy, or for whom surgery and/or radiotherapy, is not an option.
Somatuline Depot
the long-term treatment of acromegalic patients who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy
Palsonify
treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option
Somavert
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
Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Financial Resources
3 resourcesTravel Grants
No travel grants are currently matched to Acromegaly.
Community
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Start the conversation →Latest news about Acromegaly
Disease timeline:
New recruiting trial: The Longitudinal Approach to Acromegaly: A Pattern of Treatment and Comparative Effectiveness Research
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Genetics of Endocrine Tumours - Familial Isolated Pituitary Adenoma - FIPA
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: KETOgenic Diet Therapy in Patients With ACROmegaly
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Cellular, Molecular and Clinical Determinants of Bone Strength in in Vivo and Human Models of GH Excess.
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Korean Regulatory Post Marketing Surveillance for Somavert
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Study of MAR002 in Healthy Men
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: A Study to Assess the Efficacy and Safety of Debio 4126 in Participants With Acromegaly Previously Treated With Somatostatin Analogs
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: The RApid Switch From 1st Generation Somatostatin Analogues to PaSireOtiDe In Acromegaly
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Developing a Simple Recognition System of Acromegaly
A new clinical trial is recruiting patients for Acromegaly
New recruiting trial: Diet in the Management of Acromegaly
A new clinical trial is recruiting patients for Acromegaly
Caregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Acromegaly
What is Acromegaly?
Acromegaly is a rare endocrine disorder caused by chronic excessive secretion of growth hormone (GH), most commonly due to a benign pituitary adenoma (somatotropinoma). When GH overproduction occurs before the closure of the epiphyseal growth plates in children, it results in gigantism; when it occurs in adults after growth plate fusion, it leads to acromegaly. The condition is characterized by the gradual enlargement of the hands, feet, and facial features (including the jaw, nose, and brow ridge), as well as soft tissue swelling, joint pain, and thickening of the skin. The disease name deriv
How is Acromegaly inherited?
Acromegaly follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Acromegaly typically begin?
Typical onset of Acromegaly is adult. Age of onset can vary across affected individuals.
Are there clinical trials for Acromegaly?
Yes — 17 recruiting clinical trials are currently listed for Acromegaly on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Acromegaly?
25 specialists and care centers treating Acromegaly are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Acromegaly?
8 patient support programs are currently tracked on UniteRare for Acromegaly. See the treatments and support programs sections for copay assistance, eligibility, and contact details.