A research study to compare somapacitan once a week with Norditropin® once a day in children who need help to grow

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 Aug 2022 → ongoing

Decision date (initial)

2024-07-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-506927-27-00

EudraCT number

2021-005607-13

WHO UTN

U1111-1270-0862

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To confirm non-inferiority of once-weekly somapacitan compared with once-daily Norditropin® in terms of longitudinal growth measured by height velocity at week 52 in children with each of the four indications: SGA, TS, NS or ISS.

Secondary objectives 4

1. To evaluate once-weekly somapacitan compared with once-daily Norditropin® in terms of other aspects of longitudinal growth in children with each of the four indications: SGA, TS, NS or ISS.
2. To evaluate safety of once-weekly somapacitan compared with once-daily Norditropin® in terms of safety parameters measured by glucose metabolism in children with each of the four indications: SGA, TS, NS or ISS.
3. To evaluate the steady state pharmacokinetics of once-weekly somapacitan in children with each of the four indications: SGA, TS, NS or ISS.
4. To evaluate long-term safety of once-weekly somapacitan in terms of safety parameters measured by glucose metabolism in children with each of the four indications: SGA, TS, NS or ISS.

Conditions and MedDRA coding

Children with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome, or idiopathic short stature

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-001469-PIP01-13, EMEA-001469-PIP02-17

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. No prior exposure to growth promoting therapy, including but not limited to growth hormone, IGF-I and ghrelin analogues.
2. Applicable to children with SGA: Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDS OR both) (according to national standards). Japan: Please see local requirements in Appendix 11 (Section ‎10.11).
3. Applicable to children with SGA: Prebubertal Children: a) Boys: - Age above er equal to 2 years and 26 weeks and below 11.0 years at screening. - Testis volume below 4 mL b) Girls: - Age above or equal to 2 years and 26 weeks and below 10.0 years at screening. Tanner stage 1 for breast development: No palpable glandular breast tissue
4. Applicable to girls with TS: Confirmed diagnosis of TS: CCI United Kingdom: Please see local requirements in Appendix 11 (Section ‎10.11).
5. Applicable to girls with TS: Prepubertal girls: - Age above or equal to 2 years and 26 weeks and below 10.0 years at screening. - Tanner stage 1 for breast development: No palpable glandular breast tissue)
6. Applicable to children with NS: Clinical diagnosis of NS according to van der Burgt score list (See Table 7) Japan: Please see local requirements in Appendix 11 (Section ‎10.11).
7. Applicable to children with NS: Prepubertal children: a) Boys: - Age above or equal to 2 years and 26 weeks and below 11.0 years at screening. - Testis volume below 4mL b) Girls: - Age above or equal to 2 years and 26 weeks and below 10.0 years at screening. -Tanner stage 1 for breast development: No palpable glandular breast tissue
8. Applicable to children with ISS: Prepubertal children: a) Boys: - Age above or equal to 2 years and 26 weeks and below 11.0 years at screening. - Testis volume below 4mL b) Girls: - Age above or equal to 2 years and 26 weeks and below 10.0 years at screening. -Tanner stage 1 for breast development: No palpable glandular breast tissue
9. Applicable to children with ISS: Bone age: a) Boys: - Bone age below or equal to 12 years. - Bone age not delayed or advanced mor than 2 years compared to chronological age. b) Girls: - Bone age below or equal to 11 years - Bone age not delayed or advanced more than 2 years compared to chronological age.

Exclusion criteria 3

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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Height velocity

Secondary endpoints 10

1. Change in Height SDS
2. Change in Height Velocity SDS
3. Change in bone age
4. Change in IGF-I SDS
5. Change in IGFBP-3 SDS
6. Change in fasting plasma glucose
7. Change in homeostatic model assessment-B (HOMA-B)
8. Change in homeostatic model assessment-IR (HOMA-IR)
9. Change in glycated haemoglobin (HbA1c)
10. CCI

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 15

Locations

16 EU/EEA countries · 46 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

Documents 227 files

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

Application history

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