A research study looking at how safe somapacitan is and how well it works in children who need help to grow – REAL 9

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

18 May 2023 → ongoing

Decision date (initial)

2023-03-27

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novo Nordisk A/S

External identifiers

EU CT number

2022-501055-87-01

WHO UTN

U1111-1277-9765

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy

To evaluate the safety of once-weekly somapacitan in children, either naïve or non-naïve to GH treatment, within each of the four indications: SGA, TS, NS or ISS.

Secondary objectives 3

1. To evaluate the long-term safety of once-weekly somapacitan in children, either naïve or non-naïve to GH treatment, within each of the four indications: SGA, TS, NS or ISS.
2. To evaluate the efficacy of once-weekly somapacitan in children, either naïve or non-naïve to GH treatment, within each of the four indications: SGA, TS, NS or ISS.
3. To evaluate the steady state pharmacokinetics of once-weekly somapacitan in children, either naïve or non-naïve to GH treatment, within each of the four indications: SGA, TS, NS or ISS.

Conditions and MedDRA coding

Noonan syndrome

Regulatory references

Scientific advice from competent authorities

National Medical Products Administration, European Medicines Agency, Pharmaceuticals And Medical Devices Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-001469-PIP01-13

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Applicable to children with SGA - Born small for gestational age (according to national standards).
2. Applicable to children with NS - Diagnosis of NS according to local clinical practice.
3. Applicable to children with NS - Male participants: Age equal to or above xx years and below xx years at screening. Female participants: Age equal to or above xx years and below xx years at screening.
4. Applicable to children with NS - Open epiphyses; defined as bone age xx years for females and bone age xx years for males.
5. Applicable to children with ISS - Male participants: Age equal to or above xx years and below xx years at screening. Female participants: Age equal to or above xx years and below xx years at screening.
6. Applicable to children with ISS - Open epiphyses; defined as bone age xx years for females and bone age xx years for males.
7. Applicable to children with SGA - Male participants: Age equal to or above xx years and below xx years at screening. Female participants: Age equal to or above xx years and below xx years at screening.
8. Applicable to children with SGA - Open epiphyses; defined as bone age xx years for females and bone age xx years for males.
9. Applicable to children with TS - Diagnosis of TS according to local clinical practice.
10. Applicable to children with TS - Female participants: Age equal to or above xx years and below xx years at screening.
11. Applicable to children with TS - Open epiphyses; defined as bone age xx years for females and bone age xx years for males.

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 fasting plasma glucose above or equal to xx
3. Current inflammatory diseases requiring xx treatment for longer than xx

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of adverse events (AEs) from week 0 to week 26

Secondary endpoints 8

1. Number of adverse events (AEs) possibly or probably related to somapacitan from week 0 to week 26
2. Number of adverse events (AEs) from week 0 to week 156
3. Height Velocity from week 0 to week 26
4. Change in Height SDS from week 0 to week 26
5. Change in Height Velocity SDS from week 0 to week 26
6. Change in IGF-1 SDS from week 0 to week 26
7. Change in IGFBP-3 SDS from week 0 to week 26
8. Weekly average somapacitan concentration (C[avg]) based on population PK analysis from week 0 to week 26

Investigational products

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

Test 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

3 EU/EEA countries · 6 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 34 files

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

Application history

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