Somapacitan for Hypophysial Dwarf

Phase-Based Estimates
2
Effectiveness
3
Safety
Novo Nordisk Investigational Site, Raleigh, NC
Hypophysial Dwarf+1 More
Somapacitan - Drug
Eligibility
< 18
All Sexes
Eligible conditions
Hypophysial Dwarf

Study Summary

This study is evaluating whether a new medicine for children with growth hormone deficiency is better than the medicine doctors already use.

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Eligible Conditions

  • Hypophysial Dwarf
  • Dwarfism, Pituitary
  • Growth Hormone Deficiency in Children

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

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.

Week -2, Week 104
Change in FPG: Week 104
Change in HOMA: Week 104
Change in HbA1c: Week 104
Week -2, Week 156
Change in FPG: Week 156
Change in HOMA: Week 156
Change in HbA1c: Week 156
Week -2, Week 208
Change in FPG: Week 208
Change in HOMA: Week 208
Change in HbA1c: Week 208
Week -2, Week 52
Change in bone age
Change in fasting plasma glucose (FPG): Week 52
Change in glycated haemoglobin (HbA1c): Week 52
Change in homeostatic model assessment (HOMA): Week 52
Week 0, Week 104
Change in IGF-I SDS: Week 104
Change in IGFBP-3 SDS: Week 104
Week 0, Week 156
Change in IGF-I SDS: Week 156
Change in IGFBP-3 SDS: Week 156
Week 0, Week 208
Change in IGF-I SDS: Week 208
Change in IGFBP-3 SDS: Week 208
Week 0, Week 52
Change in Height Standard Deviation Score
Change in height velocity standard deviation score (HV SDS)
Change in insulin-like growth factor I (IGF-I) SDS: Week 52
Change in insulin-like growth factor binding protein 3 (IGFBP-3) SDS: Week 52
Week 0-52
Height velocity

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Norditropin® daily
Somapacitan weekly

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.

Somapacitan weekly
Drug
Participants will receive somapacitan weekly for 52 weeks (main trial period). Participants completing the main trial period in both the treatment arms ('Somapacitan weekly' and 'Norditropin® daily') will receive somapacitan weekly for 3 years (extension trial period).
Norditropin® daily
Drug
Participants will receive Norditropin® daily for 52 weeks (main trial period).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Somapacitan
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: week -2, week 208
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly week -2, week 208 for reporting.

Closest Location

Novo Nordisk Investigational Site - Raleigh, NC

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention
No prior exposure to growth hormone therapy or IGF-I treatment
Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue)
Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World Health Organisation (WHO) International Somatropin 98/574 standard
Impaired height velocity, defined as annualised height velocity below the 25th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening
Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people get hypophysial dwarf a year in the United States?

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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.

Unverified Answer

What are common treatments for hypophysial dwarf?

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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.

Unverified Answer

What is hypophysial dwarf?

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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.

Unverified Answer

Can hypophysial dwarf be cured?

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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.

Unverified Answer

What causes hypophysial dwarf?

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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.

Unverified Answer

What are the signs of hypophysial dwarf?

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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.

Unverified Answer

Is somapacitan typically used in combination with any other treatments?

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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.

Unverified Answer

What is the average age someone gets hypophysial dwarf?

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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.

Unverified Answer

Who should consider clinical trials for hypophysial dwarf?

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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.

Unverified Answer

Is somapacitan safe for people?

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Somapacitan should be administered with caution to patients treated for glaucoma while awaiting safety results from the phase 1 trial and with close monitoring during long term administration for cardiovascular health.

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What are the latest developments in somapacitan for therapeutic use?

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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.

Unverified Answer

How serious can hypophysial dwarf be?

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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.

Unverified Answer
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