Phase 3
N=200
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)
Growth Hormone Deficiency in Children
Bottom Line
View on ClinicalTrials.gov: NCT03811535 ↗Enrolled (actual)
200
Serious AEs
4.0%
Results posted
Aug 2023
Primary outcome: Primary: Height Velocity: In-trial Observation Period — 11.8; 11.2 centimeters per year (cm/year)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Somapacitan (Drug); Norditropin® (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Nov 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Height Velocity: In-trial Observation Period |
11.8; 11.2 | — |
| PRIMARY Height Velocity: On-treatment Observation Period |
11.8; 11.2 | — |
| SECONDARY Change in Bone Age |
1.28; 1.13 | — |
| SECONDARY Change in Height Standard Deviation Score (HSDS) |
1.37; 1.21 | — |
| SECONDARY Change in Height Velocity Standard Deviation Score (HV SDS) |
8.97; 7.97 | — |
| SECONDARY Change in Fasting Plasma Glucose (FPG) at Week 52 |
0.255; 0.083 | — |
| SECONDARY Change in FPG at Week 104 |
— | — |
| SECONDARY Change in FPG at Week 156 |
— | — |
| SECONDARY Change in FPG at Week 208 |
— | — |
| SECONDARY Change in Homeostatic Model Assessment Steady State Beta Cell Function (HOMA-B) at Week 52 |
69.24; 37.71 | — |
| SECONDARY Change in HOMA-B at Week 104 |
— | — |
| SECONDARY Change in HOMA-B at Week 156 |
— | — |
| SECONDARY Change in HOMA-B at Week 208 |
— | — |
| SECONDARY Change in Homeostatic Model Assessment Insulin Resistance (HOMA-IR) at Week 52 |
1.03; 0.60 | — |
| SECONDARY Change in HOMA-IR at Week 104 |
— | — |
| SECONDARY Change in HOMA-IR at Week 156 |
— | — |
| SECONDARY Change in HOMA-IR at Week 208 |
— | — |
| SECONDARY Change in Glycated Haemoglobin (HbA1c) at Week 52 |
0.11; 0.09 | — |
| SECONDARY Change in HbA1c at Week 104 |
— | — |
| SECONDARY Change in HbA1c at Week 156 |
— | — |
| SECONDARY Change in HbA1c at Week 208 |
— | — |
| SECONDARY Change in Insulin-like Growth Factor I (IGF-I) Standard Deviation Score (SDS) at Week 52 |
2.41; 2.32 | — |
| SECONDARY Change in IGF-I SDS at Week 104 |
— | — |
| SECONDARY Change in IGF-I SDS at Week 156 |
— | — |
| SECONDARY Change in IGF-I SDS at Week 208 |
— | — |
| SECONDARY Change in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) Standard Deviation Score (SDS) at Week 52 |
1.68; 1.58 | — |
| SECONDARY Change in IGFBP-3 SDS at Week 104 |
— | — |
| SECONDARY Change in IGFBP-3 SDS at Week 156 |
— | — |
| SECONDARY Change in IGFBP-3 SDS at Week 208 |
— | — |
Summary
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT03811535). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.