A study comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin as well as evaluating long-term safety of somapacitan in a basket study design in children with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome, or idiopathic short stature

JRCT ID: jRCT2031220263

Registered date:10/08/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: SGA, Turner syndrome, Noonan syndrome or idiopathic short stature (ISS)
• Date of first enrollment: 10/08/2022
• Target sample size: 45
• Countries of recruitment: Austria,Japan,Belgium,Japan,Brazil,Japan,Bulgaria,Japan,Canada,Japan,China,Japan,Croatia,Japan,Finland,Japan,France,Japan,Germany,Japan,Greece,Japan,India,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Latvia,Japan,Lithuania,Japan,Malaysia,Japan,Mexico,Japan,Netherlands,Japan,Poland,Japan,Portugal,Japan,Russia,Japan,Saudi Arabia,Japan,Serbia,Japan,Slovenia,Japan,South Africa,Japan,South Korea,Japan,Spain,Japan,Switzerland,Japan
• Study type: Interventional
• Intervention(s): 0.24mg/kg/week s.c. treatment of somapacitan or 0.035mg/kg/day, 0.05mg/kg/day, 0.067mg/kg/day s.c. growth hormone (GH) (Norditropin) treatment

Outcome(s)

• Primary Outcome: Height velocity

Key inclusion & exclusion criteria

• Age minimum: >= 2age 6month old
• Age maximum: < 11age old
• Gender: Both

Include criteria

1. No prior exposure to growth promoting therapy, including but not limited to growth hormone, IGF-I and ghrelin analogues.
2. Boys:Age above or equal to 2 years and 26 weeks and below 11.0 years at screening.
Girls:Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
small for gestational age(SGA)
3. SGA(according to national standards).
4. Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention
Turner syndrome(TS)
5. Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis.
6. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention
Noonan syndrome(NS)
7. Clinical diagnosis of NS
8. Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention
idiopathic short stature(ISS)
9. Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention
10. Normal GH secretion during GH stimulation test performed within 18 months prior to screening

Exclude criteria

1. Children with suspected or confirmed growth hormone deficiency according to local practice.
2. Children diagnosed with diabetes mellitus or screening values from the central laboratory of
a. fasting plasma glucose above or equal to 126 mg/dL (7.0 mmol/L) or
b. HbA1c above or equal to 6.5%.
3. Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening.
4. Children requiring inhaled glucocorticoid therapy at a dose greater than 400 ug/day of inhaled budesonide or equivalent (i.e., 250 ug/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening.
5. Concomitant administration of other treatments that may have an effect on growth, e.g., but not limited to methylphenidate for treatment of attention deficit hyperactivity disorder (ADHD).
6. Diagnosis of attention deficit hyperactivity disorder (ADHD).
7. History or known presence of malignancy including intracranial tumours.

Related Information

• Primary Sponsor: Fujioka Koichi
• Secondary ID(s): NCT05330325