Somatropin for Growth Disorders

Not currently recruiting at 1 trial location

Overseen By Andrew Dauber, MD

Age: Any Age

Sex: Male

Trial Phase: Phase 2

Sponsor: Children's National Research Institute

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1 year

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called somatropin, designed to help individuals with a specific growth disorder. The focus is on a single patient with a rare mutation affecting growth hormone function, resulting in higher levels of a protein that binds growth hormone. The goal is to determine if increasing doses of somatropin can enhance growth by adjusting a growth-related factor in the body. The ideal participant has this specific genetic mutation affecting growth hormone. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the study team.

Is there any evidence suggesting that Somatropin is likely to be safe for humans?

Research has shown that somatropin, a type of growth hormone treatment, is usually well-tolerated. In a large study, only 3.1% of patients experienced side effects potentially related to the treatment, indicating that most people did not encounter serious problems. Another study found that a similar growth hormone was effective and safe for children in everyday situations.

Concerns exist about long-term risks, such as an increased chance of health issues in adulthood, but overall, growth hormone treatments are considered safe. These findings suggest that while somatropin is mostly safe, monitoring for side effects during treatment remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about Somatropin for growth disorders because it offers a targeted approach by focusing on maintaining specific IGF-1 levels. Unlike traditional growth hormone therapies that might not precisely control hormone levels, Somatropin allows for dose adjustments to ensure a safer and potentially more effective treatment. This tailored approach could lead to improved growth outcomes while minimizing side effects, making it a promising option for individuals with growth disorders.

What evidence suggests that Somatropin might be an effective treatment for growth disorders?

Studies have shown that somatropin, a type of growth hormone, effectively treats growth hormone deficiencies. Patients using somatropin have experienced noticeable increases in height over time. Specifically, significant growth occurred in different dosage groups. This trial will involve participants receiving escalating doses of somatropin until maintaining an IGF-1 level between the mean and +2 standard deviations. This treatment increases levels of insulin-like growth factor-1 (IGF-1), which is important for growth and development. Although some concerns exist about long-term effects, the treatment is generally considered effective for helping children grow when they have growth hormone issues. ^{6 7 8 9 10}

Who Is on the Research Team?

Andrew Dauber, MD

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

This trial is specifically for a single patient who has signed consent and agreed to follow the study procedures. The patient must have a particular mutation in the GHR gene that causes high levels of GHBP, which affects growth.

Inclusion Criteria

Provision of signed and dated informed consent form

Stated willingness to comply with all study procedures and availability for the duration of the study

I have a mutation in the GHR gene causing high GHBP levels.

Exclusion Criteria

There are no exclusion criteria for this study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

The participant receives escalating doses of growth hormone to achieve an IGF-1 level above the mean

1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Somatropin

Trial Overview The trial is testing escalating doses of Somatropin, a growth hormone, on this unique patient. The goal is to adjust the dosage until it raises insulin-like growth factor-1 above average and then observe how well the treatment stimulates growth.

How Is the Trial Designed?

Group I: Growth hormone Experimental Treatment 1 Intervention

The participant will receive escalating dose of growth hormone until an IGF-1 level is maintained between the mean and +2 standard deviations.

Somatropin is already approved in European Union, United States, Canada, Japan, Switzerland for the following indications:

Approved in European Union as Genotropin for:

• Growth hormone deficiency
• Turner syndrome
• Chronic kidney disease
• Short stature homeobox-containing gene (SHOX) deficiency
• Small for gestational age (SGA) with no catch-up growth

Approved in United States as Humatrope for:

• Growth hormone deficiency
• Turner syndrome
• Idiopathic short stature
• Short stature associated with Noonan syndrome
• Short stature associated with Turner syndrome in pediatric patients whose epiphyses are closed

Approved in Canada as Norditropin for:

• Growth hormone deficiency
• Turner syndrome
• Chronic kidney disease
• Short stature homeobox-containing gene (SHOX) deficiency
• Small for gestational age (SGA) with no catch-up growth

Approved in Japan as Nutropin for:

• Growth hormone deficiency
• Turner syndrome
• Chronic kidney disease

Approved in Switzerland as Omnitrope for:

• Growth hormone deficiency
• Turner syndrome
• Chronic kidney disease
• Short stature homeobox-containing gene (SHOX) deficiency
• Small for gestational age (SGA) with no catch-up growth

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Published Research Related to This Trial

In a Phase IV study involving 103 children aged 6 to 17, Norditropin NordiFlex was found to be significantly easier to use than previous growth hormone therapy devices, with a median ease-of-use score of 7.5 (P < 0.001).

Approximately 64.4% of patients preferred Norditropin NordiFlex, and 41% were able to self-inject, indicating that this device may enhance patient autonomy and improve adherence to growth hormone therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

User assessment of Norditropin NordiFlex(®), a new prefilled growth hormone pen: a Phase IV multicenter prospective study. Tauber, M., Jaquet, D., Jesuran-Perelroizen, M., et al.[2022]

In a study involving 199 children with growth hormone deficiency, treatment with Genotropin significantly increased height velocity from 3-4 cm/year to about 10 cm/year after 6 to 9 months.

The study found that administering the hormone in 6-7 injections per week resulted in greater height gains (2-3 cm/year more) compared to 3 injections, and the treatment showed low immunogenicity, with only 2% of children developing detectable antibodies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical experience with Genotropin worldwide: an update March 1987. Gunnarsson, R., Wilton, P.[2019]

In a phase III study involving 70 prepubertal children with growth hormone deficiency, long-term treatment with Omnitrope (0.03 mg/kg/day) resulted in significant growth improvements over 4 years, including an average height increase of 31.1 cm.

Omnitrope was found to be safe and well tolerated, with significant increases in serum levels of IGF-1 and IGFBP-3, indicating effective stimulation of growth-related hormones.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of the recombinant human growth hormone Omnitrope® in the treatment of Spanish growth hormone deficient children: results of a phase III study. López-Siguero, J., Borrás Pérez, MV., Balser, S., et al.[2013]

In a year-long study involving 16 children with growth hormone deficiency, both methionyl-somatotropin (Somatonorm) and recombinant-somatotropin (Genotropin) significantly improved growth rates, with no significant difference between the two treatments.

While no serious adverse effects were reported, four children treated with Somatonorm developed low levels of antibodies against growth hormone, whereas none of the children receiving Genotropin showed this response.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Results of the treatment of growth hormone deficiency with methionine-somatotropin or recombinant somatotropin]. Dorantes-Alvarez, LM.[2006]

The PATRO Adults study is a large, long-term observational study designed to assess the safety and efficacy of somatropin (Omnitrope(®)) in adults with growth hormone deficiency, involving multiple centers across Europe.

This study will provide valuable insights into the long-term effects of Omnitrope(®) on various health factors, including diabetes development, tumor recurrence, and overall quality of life, while monitoring adverse events and safety outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rationale and design of PATRO Adults, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope(®) for the treatment of adult patients with growth hormone deficiency. Beck-Peccoz, P., Minuto, F., Leal-Cerro, A., et al.[2021]

The PATRO Children study, involving 1837 patients across 184 sites in 10 European countries, is monitoring the long-term safety and efficacy of somatropin (Omnitrope(®)) in children requiring growth hormone treatment, with initial results showing no confirmed cases of diabetes or malignancies.

Efficacy data from the study are consistent with previous findings, indicating that Omnitrope(®) is safe for use in children, including those with specific conditions like Prader-Willi syndrome, and will provide further insights into its long-term safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Design of, and first data from, PATRO Children, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope(®) in children requiring growth hormone treatment. Pfäffle, R., Schwab, KO., Marginean, O., et al.[2021]

Citations

1. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK596750/

Somatropin for Growth Hormone Deficiency - NCBI Bookshelf There are some concerns regarding long-term outcomes of GH treatment related to an increase in insulin resistance and an increased risk of cancer. It remains to ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35405011/

Efficacy and Safety of Weekly Somatrogon vs Daily ... The efficacy of once-weekly somatrogon was noninferior to once-daily somatropin, with similar safety and tolerability profiles.

3. sciencedirect.com sciencedirect.com/science/article/pii/S1043661823001615

Efficacy, safety, quality of life, adherence and cost ... We evaluated the efficacy, safety, adherence, quality of life (QoL) and cost-effectiveness of long-acting growth hormone (LAGH) vs daily growth hormone (GH) ...

4. novomedlink.com novomedlink.com/growth-related-disorders/products/treatments/norditropin/efficacy-safety/growth-hormone-deficiency.html

Norditropin® Efficacy & Safety: Growth Hormone Deficiency Adjusted mean increases in HSDS over the 2-year period were 0.81, 1.57, and 1.73 in the 0.025, 0.05, and 0.1 mg/kg/day dose groups, respectively1 ...

5. aetna.com aetna.com/cpb/medical/data/100_199/0170.html

Growth Hormone (GH) and Growth Hormone Antagonists There are several brands of GH (somatropin) on the market, and there is a lack of reliable evidence that any brand of GH is more effective than others for any ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9693805/

Safety and Efficacy of Pediatric Growth Hormone Therapy A total of 23 163 AEs was reported for 14.4% of patients; 3108 AEs were assessed by the investigators as potentially treatment related for 3.1% of patients.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT01088412

Observational Study of Somatropin Treatment in Children GeNeSIS is an open-label, multinational, multicenter, observational study to evaluate the safety and effectiveness of Humatrope treatment.

8. academic.oup.com academic.oup.com/jcem/article/106/6/1728/6133442

Long-Term Safety of Growth Hormone Treatment in Childhood Growth hormone (GH) treatment has a generally good safety profile; however, concerns about increased mortality risk in adulthood have been raised.

9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10918591/

Safety and Effectiveness of a Biosimilar Recombinant Human ... This final analysis of the PATRO Children study indicates that biosimilar rhGH is well tolerated and effective in real-world clinical practice.

10. novomedlink.com novomedlink.com/growth-related-disorders/products/treatments/norditropin/efficacy-safety/overview.html

Norditropin® Efficacy & Safety: Overview The safety and efficacy of Norditropin® have been evaluated in clinical studies and in an observational study in a real-world setting (ANSWER program).