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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the effectiveness and safety of a new weekly growth hormone treatment, GenSci004, compared to the daily treatment, Genotropin. It focuses on children with untreated growth hormone deficiency (GHD) who have experienced growth issues. The trial includes two groups: one receiving the experimental weekly treatment and the other receiving the standard daily treatment. Ideal participants are prepubertal children diagnosed with GHD and exhibiting noticeable growth delays. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that GenSci004, a once-a-week growth hormone treatment, is safe in studies with children who have growth hormone deficiency (GHD). These studies report that side effects are usually mild and similar to those of other growth hormone treatments. Genotropin, a daily growth hormone treatment, has FDA approval for GHD and is known to be well-tolerated, with low rates of side effects like antibody development, meaning patients' bodies usually don't react badly to it.

Since GenSci004 is in the final stages of clinical trials, earlier tests have already demonstrated some safety. For those considering joining a trial, it's important to know that both treatments being compared have been studied for safety, with Genotropin already having a history of low side effects in children with GHD. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about GenSci004 for treating growth hormone deficiency because it offers a new weekly dosing schedule. Unlike the standard daily injections, this treatment could significantly reduce the treatment burden on patients, making it easier to stick to the regimen. This longer-acting formulation can enhance convenience without compromising effectiveness, potentially improving adherence and outcomes for those with growth hormone deficiency.

What evidence suggests that this trial's treatments could be effective for growth hormone deficiency?

This trial will compare GenSci004, a weekly growth hormone treatment, with Genotropin, a daily hormone shot, for children who need help growing. Research has shown that GenSci004 appears promising, with studies finding it works as well as daily hormone shots like Genotropin. One study found that a similar weekly treatment, somatrogon, was as effective and safe as daily treatments, offering a more convenient option. Early results suggest that weekly injections could help children grow significantly taller, possibly adding 2-3 cm more per year compared to less frequent treatments. This suggests that GenSci004 might be just as effective in promoting growth in children. ^{3 4 6 7 8}

Who Is on the Research Team?

Bradley Miller

Principal Investigator

University of Minnesota

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. 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.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06007417

NCT06007417 | A Study to Investigate Efficacy and Safety ... 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 ...

3. withpower.com withpower.com/trial/phase-3-ghd-11-2023-6f59f

Weekly Growth Hormone for Growth Hormone Deficiency ... 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 ...

4. go.drugbank.com go.drugbank.com/conditions/DBCOND0051106

Human Growth Hormone Deficiency (DBCOND0051106) ... (GenSci004) in Children With Growth Hormone Deficiency. PEG-somatropin ... Outcomes Of Genotropin Treatment In Growth Hormone Deficiency (GHD) Patients ...

5. delta.larvol.com delta.larvol.com/Products/?ProductId=b4df41cc-77a6-4b06-8b2c-0cfede6516c2

pegylated somatropin (Gensci 004) / GeneScience September 06, 2023. ESCALATE: A Study to Investigate the Safety and Efficacy of Once-weekly PEG-somatropin (GenSci004) in Children With Growth Hormone ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06024967

NCT06024967 | A Study to Investigate the Safety and ... The purpose of this Phase 3 study is to evaluate the safety and efficacy of once-weekly GenSci004 over 108 weeks in children with GHD aged from 6 months to 17 ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06007417?cond=Isolated%20congenital%20growth%20hormone%20deficiency%20OR%20%22congenital%20IGHD%22%20OR%20%22%20congenital%20isolated%20GH%20deficiency%22%20OR%20%22%20congenital%20isolated%20growth%20hormone%20deficiency%22%20OR%20%22%20familial%20isolated%20growth%20hormone%20deficiency%22%20OR%20%22%20ICGHD%22%20OR%20%22%20IGHD%22%20OR%20%22%20isolated%20growth%20hormone%20deficiency%22&aggFilters=status:not%20rec&viewType=Table&rank=8

A Study to Investigate Efficacy and Safety of Weekly PEG ... 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 ...

8. medthority.com medthority.com/clinical-trials/growth-hormone-deficiency/a-study-to-investigate-the-safety-and-efficacy-of-once-weekly-peg-somatropin-gensci004-in-children-with-growth-hormone-deficiency-escalate/

GenSci004 safety & efficacy in paediatric GHD A study to investigate the safety and efficacy of once-weekly PEG-somatropin (GenSci004) in children with growth hormone deficiency (ESCALATE).