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
Age of Onset
ICD-10
E22.0
Inheritance
This condition does not appear to have a clear pattern of inheritance.
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.†
5 Facts you should know
FACT
A disorder that results from excess growth hormone (GH) after the growth plates have closed.
FACT
The initial symptom is typically enlargement of the hands and feet.
FACT
There may also be an enlargement of the forehead, jaw, and nose.
FACT
Other symptoms may include joint pain, thicker skin, deepening of the voice, headaches, and problems with vision.
FACT
Complications of the disease may include type 2 diabetes, sleep apnea, and high blood pressure.
Interest over time
Google searches
Common signs & symptoms
Arthralgia
Joint pain
Broad 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
| Title | Description | Phases | Status | Interventions | More 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 3 | Recruiting | Drug: Paltusotine Drug: Placebo | More Info |
| Acromegaly Combination Treatment Study | In 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/A | Recruiting | Drug: Pegvisomant Drug: Octreotide LAR Drug: Lanreotide | More Info |
| Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess | For 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 3 | Recruiting | Drug: Pegvisomant | More Info |
| A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly | The purpose of this trial is to assess the long-term safety of CAM2029 in patients with acromegaly. | Phase 3 | Recruiting | Drug: CAM2029 (octreotide subcutaneous depot) | More Info |
| A Trial to Assess Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly | The purpose of this trial is to assess the efficacy and safety of CAM2029 in patients with acromegaly. | Phase 3 | Recruiting | Drug: CAM2029 (octreotide subcutaneous depot) Drug: Matching placebo | More Info |
| A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly | 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 acromegaly previously treated with somatostatin receptor ligand (SRL) based treatment regimens. | Phase 3 | Recruiting | Drug: 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 2 | Enrolling by invitation | Drug: Paltusotine | More Info |
Top Treatments in Research
| Agent | Class/Mechanism of Action | Development Status | Company | Clinical Studies | More Information |
|---|---|---|---|---|---|
| Paltusotine | Paltusotine is a promising once-daily oral nonpeptide SST2 agonist candidate for managing acromegaly and neuroendocrine tumors. | Phase 3 | Crinetics Pharmaceuticals Inc. | More Info | More Info |
| CAM2029 | A new long-acting investigational formulation of octreotide, an octapeptide that mimics natural somatostatin pharmacologically. | Phase 3 | Camurus | More Info | More Info |
† Genetic and Rare Diseases Information Center (GARD) - PO Box 8126, Gaithersburg, MD 20898-8126 https://rarediseases.info.nih.gov