Comparison of Weekly Somatrogon to Daily Genotropin in Children Born Small for Gestational Age or With Idiopathic Short Stature.

Not Applicable

Not yet recruiting

• Conditions: ISS; SGA
• Interventions: Drug: Somatrogon; Drug: Genotropin

• Registration Number: NCT07226089
• Lead Sponsor: Rabin Medical Center

Brief Summary

This study is a randomized, open-label, active controlled, parallel group study comparing the efficacy and safety of once weekly Somatrogon to daily Growth Hormone (Genotropin) in pre-pubertal children with short stature either born Small for Gestational Age (SGA) or with Idiopathic Short Stature (ISS). The planned study duration is 12 months with a screening period of up to 30 days. The study will consist of two groups: 140 children with SGA who are naïve to GH treatment will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months. A second group will include 114 children with ISS who are naïve to GH treatment who will be randomized 1:1 to receive either Somatrogon or Genotropin for 12 months.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-10
• Study First Submitted: 2025-11-06
• Study First Submitted QC: 2025-11-06
• Last Update Submitted: 2025-11-06
• Study First Posted: 2025-11-10
• Last Update Posted: 2025-11-10
• Study Start: 2026-02-01
• Primary Completion: 2027-12
• Study Completion: 2028-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 254

Inclusion Criteria

1. Diagnosis of SGA or ISS. SGA, defined as born with a birth weight and/or length <-2 SDS below the mean for gestational age. ISS, defined as height < -2 SDS for age and gender without evidence of GHD
2. Females aged ≥3 years and <9 years. Males aged ≥3 years and <11 years
3. Pre-pubertal- Tanner stage 1 for breasts and testes.
4. A bone age of not more than chronological age recorded in previous 8 weeks.
5. Current height < -2 SDS for age and gender.
6. Participants using hormonal replacement therapy(s) must be on an optimized and stable treatment regimen (hormone levels within normal ranges on screening) for at least three months prior to screening
7. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.

Exclusion Criteria

1. History of cancer, radiation therapy or chemotherapy.
2. History of GHD.
3. Children who are small due to malnutrition, defined as a Z score of weight for height and/or BMI below -2 for age, according to national standards.
4. History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
5. Microcephaly (Head Circumference < -2 SDS)
6. Any chronic disease or diagnosis, likely to affect growth, including but not limited to gastrointestinal disorder, celiac disease, untreated thyroid disease, diabetes mellitus and metabolic disorders.
7. Known or suspected skeletal dysplasias
8. Known or suspected chromosomal abnormalities
9. IGF-1 >2 SDS
10. Any disorder or condition which, in the opinion of the investigator, might jeopardize participant's safety or compliance with the protocol
11. Prior exposure to growth promoting therapy
12. Current use of any prohibited concomitant medication(s): Any rhGH or growth-promoting therapy, Any therapy that affects appetite or weight, Psychiatric medications associated with weight changes and/or diabetes, excluding medications used to treat ADHD, Any androgen or estrogen therapy including over the counter supplements, Systemic corticosteroids (inhaled or oral) exceeding the doses: Inhaled: > 400 μg/day of inhaled budesonide or equivalent. Oral: > 8 mg/m2/day of oral hydrocortisone or equivalent.
13. Previous administration with an investigational drug within 90 days.
14. Fasting blood glucose >126 mg/dL
15. Renal impairment
16. Hepatic dysfunction.
17. Pregnancy
18. Known hypersensitivity to the components of the study intervention

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Somatrogon	Somatrogon	Somatrogon will be administered subcutaneously (s.c) once weekly using a multi dose disposable prefilled pen for single patient use intended for SC self injection.
Genotropin	Genotropin	Genotropin will be administrated subcutaneously (s.c) daily using Genotropin Pen growth hormone delivery devices or Genotropin two chamber cartridges (will be supplied in their primary commercial packaging)

Primary Outcome Measures

Name	Time	Method
Annual Height Velocity	• Annualized HV after 12 months of treatment	Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.

Secondary Outcome Measures

Name	Time	Method
Height Velocity over time	• HV over 3, 6 and 9 months
height SDS	3, 6, 9 and 12 months	Changes in height Standard Deviation Score (SDS)
Bone maturation	at screening and after 12 months	Annual change in bone age measurements as per Gruelich-Pyle method
Insulin-like Growth Factor-1 (IGF-1)	Screening and after 3, 6, 9 and 12 months	Via central lab analysis
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)	Baseline and at 12 months	Via central lab analysis
change in health-related quality of life	baseline and after 12 months	Assessed by QoLISSY questionnaire

Trial Locations

Locations (6)

Shaare Zedek Medical Center; 🇮🇱 Jerusalem, Israe, Israel

Soroka hospital; 🇮🇱 Bear Sheva, Israel

Schneider Children Medical Center- the institute of Endocrinology and Diabetes; 🇮🇱 Petah Tikva, Israel

Sheba Medical Center; 🇮🇱 Ramat Gan, Israel

Assaf Harofe Medical Center; 🇮🇱 Rishon LeZiyyon, Israel

Dana-Duek children's hospital; 🇮🇱 Tel Aviv, Israel