Weekly Growth Hormone for Growth Hormone Deficiency

(ELEVATE Trial)

Overseen By Peng Duan

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

Must not be taking: RhGH, IGF 1 therapy

Disqualifiers: Major medical conditions, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

162 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the efficacy and safety of weekly GenSci004 compared with daily Genotropin in treatment-naive children with growth failure due to GHD.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you have multiple hormonal deficiencies, you must be on stable replacement therapy for at least 3 months before joining the trial.

What data supports the effectiveness of the drug Genotropin for treating growth hormone deficiency?

Research shows that Genotropin significantly increases growth rates in children with growth hormone deficiency, with height velocity improving from about 3-4 cm/year to 8-10 cm/year after treatment. The effectiveness is greater with more frequent weekly doses, and very few side effects have been reported. ^{1 2 3 4 5}

Is weekly growth hormone treatment safe for humans?

The safety of growth hormone therapy has been studied extensively, showing an excellent safety profile in large studies with over 600,000 patient-years of exposure. Some concerns about increased mortality and stroke risk have been raised, but these findings are not consistently replicated across studies. Overall, growth hormone treatments like Genotropin have a favorable safety profile, with low rates of side effects such as antibody development. ^{3 6 7 8 9}

How does the drug Genotropin, GenSci004 differ from other treatments for growth hormone deficiency?

Genotropin is typically administered daily, while GenSci004 (Somatrogon) is a long-acting growth hormone designed for once-weekly administration, offering a more convenient dosing schedule compared to the daily injections required by Genotropin. ^{1 3 5 10 11}

Research Team

Bradley Miller

Principal Investigator

University of Minnesota

Eligibility Criteria

This trial is for treatment-naive children with Growth Hormone Deficiency (GHD). They must have normal growth charts for their age and sex, no major medical conditions, not be on any other investigational drugs recently, and have never used growth hormone therapies before.

Inclusion Criteria

My height is significantly shorter than average for my age and sex.

My genetic test shows I have a normal female chromosome pattern.

My child has been on stable hormone replacement for other brain-related hormone issues for over 3 months.

Exclusion Criteria

I do not have major health issues or reasons I can't take human growth hormone.

Bone age greater than or equal to chronological age

I have previously used growth hormone or IGF 1 therapy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive weekly GenSci004 or daily Genotropin for growth hormone deficiency

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Treatment Details

Interventions

• Genotropin
• GenSci004

Trial Overview The study compares the effectiveness and safety of a weekly dose of GenSci004 to a daily dose of Genotropin in children who haven't been treated for GHD. The goal is to see if taking GenSci004 once a week works as well as taking Genotropin every day.

Participant Groups

Group I: GenSci004 Experimental Treatment 1 Intervention

Group II: Genotropin Active Control 1 Intervention

Who Is Running the Clinical Trial?

Changchun GeneScience Pharmaceutical Co., Ltd.

Lead Sponsor

Trials

72

Recruited

18,700+

Dr. Lei Jin

Changchun GeneScience Pharmaceutical Co., Ltd.

Chief Executive Officer

PhD

Dr. Yuanfeng Xia

Changchun GeneScience Pharmaceutical Co., Ltd.

Chief Medical Officer

MD

Findings from Research

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 12-month phase 3 clinical trial involving 117 treatment-naïve prepubertal children with pediatric growth hormone deficiency (pGHD), both once-weekly somatrogon and once-daily somatropin significantly improved health-related quality of life (HRQoL) as measured by the QoLISSY questionnaire.

Children aged 7 and older reported greater HRQoL improvements with somatrogon compared to somatropin, suggesting that the once-weekly treatment may offer advantages in perceived quality of life, while parents tended to underestimate these improvements.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin. Loftus, J., Quitmann, J., Valluri, SR.[2023]

Recombinant human growth hormone (rhGH) has a strong safety profile, supported by extensive studies involving over 600,000 patient-years of exposure, indicating it is generally safe for children receiving this therapy.

While there have been reports of increased mortality risk associated with growth hormone therapy, these risks appear to be linked to factors like small size at birth, and further long-term safety studies are needed, especially with new long-acting growth hormone products.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Monitoring rhGH Safety: rhGH Registries, SAGhE and Future Needs. Miller, BS., Rosenfeld, RG.[2019]

A comprehensive review of safety data from growth hormone (GH) registries found no increased risk of mortality or stroke in children or adults treated with GH, providing reassurance about its long-term safety.

While there may be a heightened risk of type 2 diabetes in GH-treated patients, this risk is primarily associated with pre-existing conditions, and patients with risk factors for malignancy should be monitored closely during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety of growth hormone-A combined registry analysis. Stochholm, K., Kiess, W.[2019]

Recombinant human growth hormone (rGH) is effective for treating various growth disorders, providing a reliable treatment option for conditions like Turner syndrome and idiopathic short stature.

Recent studies, including the National Cooperative Growth Study and the EU SAGhE study, indicate that rGH has a favorable safety profile, which is important for physicians when considering treatment options for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Growth Hormone and Treatment Controversy; Long Term Safety of rGH. Divall, SA., Radovick, S.[2021]

In a study of 67 Italian patients with growth hormone deficiency, Omnitrope® was found to be effective in improving health markers like insulin-like growth factor-1 levels and body composition over an average treatment duration of 45.4 months.

While 55.2% of patients experienced adverse events, none were deemed related to Omnitrope®, indicating that it is generally safe for long-term use in adults with growth hormone deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of Omnitrope&#174; in adults with growth hormone deficiency: Italian interim analysis of the PATRO Adults study. Ferone, D., Profka, E., Gasco, V., et al.[2018]

References

1. Sweden pubmed.ncbi.nlm.nih.gov

Clinical experience with Genotropin in growth hormone deficient children. [2019]

2. England pubmed.ncbi.nlm.nih.gov

TransCon human growth hormone for children with growth hormone deficiency: a technology evaluation. [2022]

3. Sweden pubmed.ncbi.nlm.nih.gov

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

4. England pubmed.ncbi.nlm.nih.gov

Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin. [2023]

5. Germany pubmed.ncbi.nlm.nih.gov

An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment. [2023]

6. Israel pubmed.ncbi.nlm.nih.gov

Monitoring rhGH Safety: rhGH Registries, SAGhE and Future Needs. [2019]

7. England pubmed.ncbi.nlm.nih.gov

Long-term safety of growth hormone-A combined registry analysis. [2019]

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

Growth Hormone and Treatment Controversy; Long Term Safety of rGH. [2021]

9. Italy pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of Omnitrope&#174; in adults with growth hormone deficiency: Italian interim analysis of the PATRO Adults study. [2018]

10. Switzerland pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Collaborative study of the effects of human growth hormone in growth hormone deficiency. V. Treatment with growth hormone administered once a week. [2006]