Comparison of weekly Somatrogon to daily Genotropin in children born small for gestational age or with idiopathic short stature.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Schneider Children's Medical Center Of Israel

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2026-06-24

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2025-523832-38-00

ClinicalTrials.gov

NCT07226089

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

To demonstrate that treatment effect of weekly somatrogon is non-inferior to daily Genotropin

Conditions and MedDRA coding

Small for gestational age (SGA) and idiopathic short stature (ISS)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. The participant has a diagnosis of SGA, defined as born with a birth weight and/or length <-2 SDS below the mean for gestational age (according to national standards).
2. The participant has a diagnosis of ISS, defined as: height < -2 SDS for age and gender without evidence of GHD (has a peak GH level >10 ng/mL, performed up to 24 months prior to screening).
3. For participants with SGA: • Pre-pubertal (Tanner stage 1 for breasts) females aged ≥4 years (EU) or ≥3 years (non-EU) and <9 years (8 years and 364 days) on the date of ICD signature. A bone age of not more than chronological age, recorded in previous 8 weeks. • Pre-pubertal (Tanner stage 1 for testes) males aged ≥4 years (EU) or ≥3 years (non-EU) and <11 years (10 years and 364 days) on the date of ICD signature. A bone age of not more than chronological age recorded in previous 8 weeks.
4. For participants with ISS: • Pre-pubertal (Tanner stage 1 for breasts) females aged ≥4 years (EU) or ≥3 years (non-EU) and <9 years (8 years and 364 days) on the date of ICD signature. A bone age of not more than chronological age and not greater than 2 years under chronological age, recorded in previous 8 weeks. • Pre-pubertal (Tanner stage 1 for testes) males aged ≥4 years (EU) or ≥3 years (non-EU) and <11 years (10 years and 364 days) on the date of ICD signature. A bone age of not more than chronological age and not greater than 2 years under chronological age, recorded in previous 8 weeks.
5. Capable of giving signed informed consent/assent, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.

Exclusion criteria 17

1. History of cancer, radiation therapy or chemotherapy.
2. IGF-1 >2 SDS
3. Any disorder or condition which, in the opinion of the investigator, might jeopardize participant’s safety or compliance with the protocol
4. History of GHD.
5. 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.
6. History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
7. Microcephaly (Head Circumference < -2 SDS)
8. 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.
9. Known or suspected skeletal dysplasias, (including significant spinal abnormalities, but not limited to scoliosis, kyphosis and spina bifida variants).
10. Psychosocial short stature
11. Known or suspected chromosomal abnormalities including, Turner Syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, short stature homeobox (SHOX) mutations/deletions.
12. Children with closed epiphyses
13. History or known presence of any active intracranial tumours or intracranial cysts.
14. Patients with acute critical illness suffering complications following open heart surgery, abdominal surgery, multiple accidental trauma, acute respiratory failure or similar conditions
15. Benign intracranial hypertension with papilledema
16. Untreated hormonal deficiencies
17. Type 2 diabetes, malignancies, chronic infections and other known causes for stunted growth

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Annualized HV after 12 months of treatment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Schneider Children's Medical Center Of Israel

Sponsor organisation

Schneider Children's Medical Center Of Israel

Address

4, Kaplan

City

Petakh Tikva

Postcode

4920230

Country

Israel

Scientific contact point

Organisation

Schneider Children's Medical Center Of Israel

Contact name

Alona Hamou

Public contact point

Organisation

Schneider Children's Medical Center Of Israel

Contact name

Alona Hamou

Third parties 8

Locations

2 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 37 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications