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

Официальное название

Multicenter Interventional Study: Somatrogon Impact on Outcomes in Naive Small for Gestational Age or Idiopathic Short Stature Pediatric Patients Compared With Daily Growth Hormone

Обзор

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.

Вмешательства

• Препарат Somatrogon
  Once weekly Growth Hormone
• Препарат Genotropin
  Daily Growth Hormone

Первичные конечные точки

• Annual Height Velocity [Срок оценки: • Annualized HV after 12 months of treatment]

Вторичные конечные точки (6)

• Height Velocity over time [Срок оценки: HV over 3, 6 and 9 months]
• height SDS [Срок оценки: 3, 6, 9 and 12 months]
• Bone maturation [Срок оценки: at screening and after 12 months]
• Insulin-like Growth Factor-1 (IGF-1) [Срок оценки: Screening and after 3, 6, 9 and 12 months]
• Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) [Срок оценки: Baseline and at 12 months]
• change in health-related quality of life [Срок оценки: baseline and after 12 months]

Критерии участия

Критерии включения

• 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
• Females aged ≥3 years and <9 years. Males aged ≥3 years and <11 years
• Pre-pubertal- Tanner stage 1 for breasts and testes.
• A bone age of not more than chronological age recorded in previous 8 weeks.
• Current height < -2 SDS for age and gender.
• 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
• Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.

Критерии исключения

• History of cancer, radiation therapy or chemotherapy.
• History of GHD.
• 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.
• History of HIV-positive, acquired immune deficiency syndrome (AIDS), hepatitis B, hepatitis C, or tuberculosis.
• Microcephaly (Head Circumference < -2 SDS)
• 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.
• Known or suspected skeletal dysplasias
• Known or suspected chromosomal abnormalities
• IGF-1 >2 SDS
• Any disorder or condition which, in the opinion of the investigator, might jeopardize participant's safety or compliance with the protocol
• Prior exposure to growth promoting therapy
• 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.
• Previous administration with an investigational drug within 90 days.
• Fasting blood glucose >126 mg/dL
• Renal impairment
• Hepatic dysfunction.
• Pregnancy
• Known hypersensitivity to the components of the study intervention

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 12 центров

• University of California Los Angeles (UCLA) - Mattel Children's Hospital — Los Angeles
• Rocky Mountain Pediatric Endocrinology — Centennial
• Nemours Children's Clinic - Jacksonville — Jacksonville
• Children's Minnesota - Minneapolis — Minneapolis
• Hackensack University Medical Center — Hackensack
• Atlantic Health System — Morristown
• Rutgers Robert Wood Johnson Medical School - The Child Heath Institute of New Jersey — New Brunswick
• The State University of New York (SUNY) School of Medicine and Biomedical Sciences — Buffalo
• … и ещё 4 центра

Франция · 6 центров

• Centre Hospitalier Universitaire d'Angers — Angers
• Hôpital Bicêtre — Le Kremlin-Bicêtre
• Chu de Lyon - Hopital Femme Mere Enfant — Lyon
• CHU Hôpital de la Timone — Marseille
• Hôpital Armand-Trousseau — Paris
• Hôpital Necker - Enfants Malades — Paris

Израиль · 6 центров

• Schneider Children Medical Center- the institute of Endocrinology and Diabetes — Petah Tikva
• Shaare Zedek Medical Center — Jerusalem
• Soroka hospital — Bear Sheva
• Sheba Medical Center — Ramat Gan
• Assaf Harofe Medical Center — Rishon LeZiyyon
• Dana-Duek children's hospital — Tel Aviv

Индия · 4 центра

• Jehangir Hospital — Maheshra
• Apollo Hospitals Enterprise Limited — Navi Mumbai
• All India Institute of Medical Sciences (AIIMS) - New Delhi — New Delhi
• Sir Ganga Ram Hospital — New Delhi

Япония · 4 центра

• Osaka Women's and Children's Hospital — Izumi-Shi
• Nara Prefecture General Medical Center — Nara
• Osaka City General Hospital — Osaka
• National Center for Child Health and Development — Setagaya-Ku

Идентификаторы

NCT: NCT07226089 · 0081-24-RMC

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗