A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day

Novo Nordisk

Status and phase: Active, not recruiting; Phase 2

Conditions: Short Stature Children Born Small for Gestational Age (SGA)

Treatments: Drug: Norditropin®; Drug: Somapacitan

Study type: Interventional

Funder types: Industry

Identifiers

NCT03878446

U1111-1207-9741 (Other Identifier)

NN8640-4245

2018-000232-10 (EudraCT Number)

Details and patient eligibility

About

The study compares 2 medicines used for the treatment of children who are born small and who stayed small: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe).

Participants will either get somapacitan or Norditropin® - which treatment is decided by chance. Both participants and the study doctor will know which treatment the participants get. The study will last for 5 years. Participants will take either an injection once every week or once every day.

Enrollment: 62 patients

Sex: All

Ages: 2 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Pre-pubertal children, boys:

  1. age between 2.5 and 11.0 years at screening.
  2. testes volume below 4 ml.

• Pre-pubertal children, girls:

  1. age between 2.5 and 10.0 years at screening.
  2. Tanner stage 1 for breast development (no palpable glandular breast tissue).

• Born small for gestational age (birth length and/or weight below -2 standard deviation scores) (according to national standards).

• Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention at screening.

• Impaired height velocity defined as annualized height velocity below the 50th percentile for chronological age and gender according to the standards of Prader calculated over a time span of minimum 6 months and maximum 18 months prior to screening.

• No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment.

Exclusion criteria

• Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements.
• Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise.
• Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening.
• Children requiring inhaled glucocorticoid therapy at a dose of greater than 400 μg/day of inhaled budesonide or equivalents for longer than 4 consecutive weeks within the last 12 months prior to screening.
• 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.
• Diagnosis of attention deficit hyperactivity disorder.
• Prior history or presence of malignancy including intracranial tumours.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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62 participants in 5 patient groups

Somapacitan 0.24 mg/kg/week

Experimental group

Description:

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Treatment:

Drug: Somapacitan

Somapacitan 0.20 mg/kg/week

Experimental group

Description:

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Treatment:

Drug: Somapacitan

Somapacitan 0.16 mg/kg/week

Experimental group

Description:

Same treatment in main period (26 weeks) and extension period I (26 weeks). Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Treatment:

Drug: Somapacitan

Norditropin® 0.035 mg/kg/day

Active Comparator group

Description:

Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Treatment:

Drug: Norditropin®

Norditropin® 0.067 mg/kg/day

Active Comparator group

Description:

Same treatment in main period (26 weeks) and extension period I (26 weeks). Participants will switch to Somapacitan during extension period II. Dosage of Somapacitan during extension period II will be determined based on the data from main phase.

Treatment:

Drug: Norditropin®