ELEVATE: A Study to Investigate Efficacy and Safety of Weekly PEG-somatropin (GenSci004) in Treatment Naive Children With Growth Hormone Deficiency
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Detailed Description
The purpose of this Phase 3 study is to evaluate the efficacy, safety, and tolerability of weekly GenSci004 compared to daily Genotropin over 52 weeks in prepubertal treatment-naïve children with growth failure due to GHD.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: GenSci004
|
Drug: GenSci004
GenSci004 is a pegylated rhGH (PEG rhGH) (i.e., PEG-somatropin)
|
| Active Comparator: Genotropin
|
Drug: Genotropin
Genotropin
|
Outcome Measures
Primary Outcome Measures
- Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups [52 weeks]
Annualized HV (height at two different visits) at 52 weeks of treatments with GenSci004 vs. Genotropin groups
Secondary Outcome Measures
- Annualized Height Velocity (AHV) for GenSci004 and Genotropin groups [104 weeks]
Annualized HV (height at two different visits) at 104 weeks treatment with GenSci004 vs. Genotropin groups
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Prepubertal children with GHD in Tanner Stage 1
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Baseline HT at least -2.0 SD below the mean HT for CA and sex (HT SDS ≤ 2.0).
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Body mass index (BMI) within ±2.0 SD of the mean BMI for BA and sex.
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Growth hormone stimulation tests:
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FOR ALL COUNTRIES EXCEPT JAPAN: ≤10 ng/mL.
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FOR JAPAN ONLY: ≤6 ng/mL.
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Baseline IGF 1 level of at least 1.0 SD below the mean IGF 1 level standardized for age and sex (IGF 1 SDS ≤-1.0)
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Normal 46 XX karyotype for girls.
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Normal fundoscopy at Screening.
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Children with multiple hormonal deficiencies must be on stable replacement therapy for other hypothalamo-pituitary axes for at least 3 months.
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Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant
Exclusion Criteria:
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BA≥CA
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Prior exposure to rhGH, long-acting growth hormones, or IGF 1 therapy.
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Major medical conditions or presence of contraindication to human growth hormone (hGH) treatment.
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Known hypersensitivity to the components of the study drug.
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Children born small for gestational age.
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Children with psychosocial dwarfism.
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Children with idiopathic short stature.
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Other causes of short stature or clinically significant abnormality likely to affect growth or the ability to evaluate growth.
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Has an unstable or severe hepatic, nephropathy, digestive tract, psychiatric, hematological, immune system disease, diabetes mellitus and impaired glucose tolerance, or abnormal laboratory test values, and investigator determines that participation in the study places the participant at unacceptable risk.
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Malignant diseases.
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Serum albumin level <LLN.
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Abnormal liver level >2xULN or renal function.
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Disagreement to refrain from sexual activity or utilize acceptable contraceptive measure during the study.
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Concomitant administration of other treatments that may influence growth.
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Children requiring glucocorticoid therapy.
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Participation in any other trial of an investigational agent within 3 months prior to Screening.
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Any reason per investigator's discretion
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Cook Childrens | Fort Worth | Texas | United States | 76104 |
Sponsors and Collaborators
- Changchun GeneScience Pharmaceuticals Co., Ltd.
Investigators
- Study Chair: Bradley Miller, University of Minnesota
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GenSci004-301