Somatropin for Growth Disorders

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

Trial Summary

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.

What data supports the effectiveness of the drug Somatropin for growth disorders?

Research shows that Somatropin, including brands like Omnitrope and Genotropin, effectively increases growth rates in children with growth hormone deficiency. Studies report significant improvements in height, with growth rates increasing from around 3-4 cm/year to about 9-10 cm/year after treatment. ^{1 2 3 4 5}

How is the drug Somatropin unique for treating growth disorders?

Somatropin is a recombinant human growth hormone that is used for both children and adults with growth hormone deficiency, offering a long-term treatment option with proven safety and efficacy. It is available in different formulations, including liquid and freeze-dried, which may provide flexibility in administration. ^{2 6 7 8 9}

What is the purpose of this trial?

This is a prospective interventional study designed for a single patient with a dominant-negative mutation in the growth hormone receptor gene (GHR) which results in increased levels of growth hormone binding protein (GHBP). The patient will receive escalating doses of growth hormone titrated to achieve an insulin like growth factor-1 level above the mean and then growth response to therapy will be monitored.

Research Team

Andrew Dauber, MD

Principal Investigator

Children's National Research Institute

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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 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]

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]

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 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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

User assessment of Norditropin NordiFlex(®), a new prefilled growth hormone pen: a Phase IV multicenter prospective study. [2022]

2. United States 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. [2013]

3. United States 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. [2021]

4. Sweden pubmed.ncbi.nlm.nih.gov

Clinical experience with Genotropin worldwide: an update March 1987. [2019]

5. Mexico pubmed.ncbi.nlm.nih.gov

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

6. United States 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. [2021]

7. Germany pubmed.ncbi.nlm.nih.gov

Systematic review of the clinical effectiveness of Genotropin (somatropin) in children with short stature. [2019]

8. Germany pubmed.ncbi.nlm.nih.gov

PATRO children, a multi-center, non-interventional study of the safety and effectiveness of Omnitrope&#174; (somatropin) treatment in children: update on the United States cohort. [2021]

9. England pubmed.ncbi.nlm.nih.gov

Comparison of the pharmacokinetics, safety and tolerability of two concentrations of a new liquid recombinant human growth hormone formulation versus the freeze-dried formulation. [2021]