This trial is evaluating whether Somapacitan will improve 1 primary outcome and 23 secondary outcomes in patients with Hypophysial Dwarf. Measurement will happen over the course of Week 0, Week 52.
This trial requires 200 total participants across 2 different treatment groups
This trial involves 2 different treatments. Somapacitan is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.
In the United States, there is a low prevalence of hypophysial dwarfism. The prevalence is 1.4/1000 in the entire population, 1/1000 in patients with pituitary disease, 1/100 in patients with acromegaly and 1/100 in patients with growth hormone deficiency. The most common presentation is a pituitary dwarf-like morphology with secondary dwarfism in individuals with acromegaly and with growth hormone deficiency. The severity of primary dwarfism in acromegalics varies from mild and subclinical to severe and even lethal.
There are several treatment modalities available and the selection should be tailored for the specific patient so that the optimum treatment is performed. Surgical treatment or resection of adenomas is the most common treatment for pituitary dwarfism. The surgical removal of the pituitary gland has been replaced by the use of dopamine agonists (e.g. bromocriptine), which are more efficient in reducing the excess prolactin secretion and can be used as a long-term treatment. Doping with luteinizing hormone releasing hormone agonists (e.g. leuprorelin) can also be used.
Hypophysial dwarf is a rare skeletal disorder characterized by a very stunted head. Most cases are not associated with other clinical features, which may be helpful in diagnosing this disorder when other conditions are not evident. The underlying cause remains unknown.
Hypophysial dwarf was a rare genetic skeletal malformation with many different congenital skeletal defects, including a bowed humeri and a depressed deltoid process. Hypophysial dwarf is a genetic disease determined by mutations in the osteoblast-specific transcription factor Osterix. The Osterix gene is responsible for the dwarfism phenotype. Since Osterix mutations have not been identified in sporadic cases of human osteopetrosis, it is presumed that the genetic abnormality responsible for osteopetsrosis is not Osterix but another osteoblast regulatory molecule. The gene Osterix is found in mice while no mouse homologous gene for human Osterix exists in mice.
The data suggest that hypothalamic enlargement in the absence of pituitary enlargement in hypophycial dwarf fetuses may be a direct result of the enlargement of hypothalamic nuclei in response to extraembryonic tissues. Furthermore, prenatal hypophycial dwarf fetuses may exhibit subtle intrauterine abnormalities. Further research is needed to understand the relationship of pituitary dwarfism to other pituitary hormone deficiencies and how it contributes to the clinical phenotype of hypophilemic dwarfism.
Growth failure along with delayed bone age and facial features are the predominant features of hypophysial dwarf. Furthermore, delayed bone age (mammalian)/premature epiphyseal closure appears to be the direct cause of hypophysial dwarf. Hypophysial dwarf may also be caused by other diseases unrelated to GH signaling. Larger series seem mandatory to accurately demonstrate the spectrum of hypophysial dwarf.
Due to the limited number of published studies for both SMA and sclc, we were unable to assess the use of SMA and other anti-tumor [immunotherapy](https://www.withpower.com/clinical-trials/immunotherapy) combinations in the treatment of either of these rare cancers. However, the data presented here, and the experience we gained, will be useful for the design of future studies using combinations of SMA and other TACE-like treatments.
Every year, an estimated 4.6 to 24.6 million children are born with hypophysial dwarfism. This is a common condition, especially in the West. If you are new to the diagnosis, do not worry. It is very different from a pituitary adenoma. (See Hypophysial Dwarf vs Pituitary Adenoma.
Clinical trials for HD would be a viable solution for women who are determined to have symptomatic hypophysial dwarf and would not choose surgical intervention. If they are well informed, patients choose treatment, particularly surgery, that the benefits of can outweigh the side effects.
Results from a recent paper suggests that a novel somapacitan formulation, RMPI-2026, could be a potential therapeutic agent for somatostatin receptor-expressing tumors associated with low and normal IGF-I serum levels, as well as for diseases of the sympathetic nervous system including hypotension in hemorrhage. RMPI-2026 could thus offer a novel therapeutic approach for tumors, such as GH-secreting adenomas of the pituitary gland and other somatostatin receptor-expressing tumors as well as for diseases of the sympathetic nervous system including hypertension, glaucoma, hypertension of pregnancy and hypotension during childbirth.
We recommend that the clinician be aware of the potential risks when treating these small pituitary adenomas. Close follow-up is crucial and the long-term results are not yet fully reported but the chances of recurrence are quite high.