ISS, SGA
Conditions
Keywords
Growth hormone, Somatrogon
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.
Interventions
DRUGSomatrogon
Once weekly Growth Hormone
DRUGGenotropin
Daily Growth Hormone
Sponsors
Rabin Medical Center
Pfizer
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 11 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
France, India, Israel, Japan, United States
Contacts
CONTACTMoshe Phillip, Professor
CONTACTAlona Hamou, MSc
STUDY_DIRECTORMoshe Phillip, Professor
Schneider Children's Medical Center
Outcome results
None listed