A Research Study in Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day (REAL4)

Novo Nordisk

Status and phase: Active, not recruiting; Phase 3

Conditions: Growth Hormone Deficiency in Children

Treatments: Drug: Norditropin®; Drug: Somapacitan

Study type: Interventional

Funder types: Industry

Identifiers

NCT03811535

U1111-1207-9691 (Other Identifier)

2018-000231-27 (Other Identifier)

JapicCTI-194773 (Registry Identifier)

NN8640-4263

Details and patient eligibility

About

The study compares 2 medicines for children who do not have enough hormone to grow: somapacitan given once a week (a new medicine) and Norditropin® given once a day (the medicine doctors can already prescribe). Researchers will test to see how well somapacitan works. The study will also test if somapacitan is safe. Participants will either get somapacitan or Norditropin® - which treatment participants get, is decided by chance. Both participants and the study doctor will know which treatment participants get. The study will last for 4 years. Participants will attend 19 clinic visits and have 1 phone call with the study doctor.

Enrollment: 200 patients

Sex: All

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Prepubertal children: a) Boys: Age more than or equal to 2 years and 26 weeks and less than 11.0 years at screening. Testis volume less than 4 ml. b) Girls: Age more than or equal to 2 years and 26 weeks and less than 10.0 years at screening. Tanner stage 1 for breast development (no palpable glandular breast tissue)
• Confirmed diagnosis of growth hormone deficiency determined by two different growth hormone stimulation tests performed within 12 months prior to randomisation, defined as a peak growth hormone level of less than or equal to 10.0 ng/ml using the World Health Organisation (WHO) International Somatropin 98/574 standard
• Impaired height defined as at least 2.0 standard deviations below the mean height for chronological age and gender at screening according to the standards of Center for Disease Control and Prevention
• Impaired height velocity, defined as annualised height velocity below the 25th 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
• Insulin-like Growth Factor-I (IGF-I) less than -1.0 SDS at screening, compared to age and gender normalized range measured at central laboratory
• No prior exposure to growth hormone therapy or 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
• 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
• Diagnosis of attention deficit hyperactivity disorder
• 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
• Prior history or presence of malignancy including intracranial tumours

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

200 participants in 2 patient groups

Somapacitan weekly

Experimental group

Description:

Participants will receive somapacitan weekly for 52 weeks (main trial period). Participants completing the main trial period in both the treatment arms ('Somapacitan weekly' and 'Norditropin® daily') will receive somapacitan weekly for 3 years (extension trial period).

Treatment:

Drug: Somapacitan

Norditropin® daily

Active Comparator group

Description:

Participants will receive Norditropin® daily for 52 weeks (main trial period).

Treatment:

Drug: Norditropin®