A research study in children with low level of hormone to grow. Treatment is somapacitan once a week compared to Norditropin® once a day

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novo Nordisk A/S

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

29 May 2019 → 1 Oct 2025

Decision date (initial)

2024-06-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-506829-11-00

EudraCT number

2018-000231-27

WHO UTN

U1111-1207-9691

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To compare the effect of somapacitan vs Norditropin® on longitudinal growth in children with growth hormone deficiency

Secondary objectives 1

1. To compare the safety of somapacitan vs Norditropin® in children with growth hormone deficiency

Conditions and MedDRA coding

Growth Hormone Deficiency in children

Regulatory references

Scientific advice from competent authorities

Pharmaceuticals And Medical Devices Agency, Federal Institute For Drugs And Medical Devices, European Medicines Agency, Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Prepubertal children: a) Boys: o Age ≥ 2 years and 26 weeks and < 11.0 years at screening o Testis volume < 4 ml b)Girls: o Age ≥ 2 years and 26 weeks and <10.0 years at screening o Tanner stage 1 for breast development (no palpable glandular breast tissue)
2. 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 ≤ 10.0 ng/ml using the WHO International Somatropin 98/574 standard
3. 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
4. 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
5. Insulin-like Growth Factor-I < -1.0 Standard Deviation Score at screening, compared to age and gender normalized range measured at central laboratory
6. No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment

Exclusion criteria 6

1. Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements
2. Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening
3. 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
4. Diagnosis of attention deficit hyperactivity disorder
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
6. Prior history or presence of malignancy including intracranial tumours

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Endpoint title: Height velocity Time frame: From baseline (week 0) to visit 7 (week 52) Unit: cm/year

Secondary endpoints 8

1. Effect Endpoint title: Change in bone age Time frame: From screening (visit 1) to visit 7 (week 52) Unit: Years
2. Effect Endpoint title: Change in Height Standard Deviation Score Time frame: From baseline (week 0) to visit 7 (week 52) Unit: -10 to +10
3. Effect Endpoint title: Change in Height Velocity SDS Time frame: From baseline (week 0) to visit 7 (week 52) Unit: -10 to +10
4. Safety Endpoint title: Change in fasting plasma glucose Time frame: From screening (visit 1) to visit 7 (week 52) From screening (visit 1) to visit 11 (week 104) From screening (visit 1) to visit 15 (week 156) From screening (visit 1) to visit 19 (week 208) Unit: mmol/L
5. Safety Endpoint title: Change in homeostatic model assessment (HOMA) Time frame: From screening (visit 1) to visit 7 (week 52) From screening (visit 1) to visit 11 (week 104) From screening (visit 1) to visit 15 (week 156) From screening (visit 1) to visit 19 (week 208) Unit: %
6. Safety Endpoint title: Change in Glycated haemoglobin (HbA1c) Time frame: From screening (visit 1) to visit 7 (week 52) From screening (visit 1) to visit 11 (week 104) From screening (visit 1) to visit 15 (week 156) From screening (visit 1) to visit 19 (week 208) Unit: % point
7. Pharmacodynamics Endpoint title: Change in IGF-I SDS Time frame: From baseline (week 0) to visit 7 (week 52) From baseline (week 0) to visit 11 (week 104) From baseline (week 0) to visit 15 (week 156) From baseline (week 0) to visit 19 (week 208) Unit: -10 to +10
8. Pharmacodynamics Endpoint title: Change in IGFBP-3 SDS Time frame: From baseline (week 0) to visit 7 (week 52) From baseline (week 0) to visit 11 (week 104) From baseline (week 0) to visit 15 (week 156) From baseline (week 0) to visit 19 (week 208) Unit: -10 to +10

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novo Nordisk A/S

Sponsor organisation

Novo Nordisk A/S

Address

Novo Alle 1

City

Bagsvaerd

Postcode

2880

Country

Denmark

Scientific contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Public contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Third parties 7

Locations

8 EU/EEA countries · 16 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 10 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications