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A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 (REAL 9)

Primary Purpose

SGA, Turner Syndrome, Noonan Syndrome

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Somapacitan
Sponsored by
Novo Nordisk A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for SGA

Eligibility Criteria

10 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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). Age: - Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age less than (<) 14 years for females and bone age < 16 years for males. For Growth Hormone (GH) treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. Applicable to children with TS: • Diagnosis of TS according to local clinical practice. Age: - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Impaired height defined as at least 2.0 standard deviation below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. For GH treatment naïve participants: Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis or confirmation of TS and TS mosaicism using comparative genomic hybridization (CGH)-array. Applicable to children with NS: Diagnosis of NS according to local clinical practice. Age: Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Clinical diagnosis of NS according to van der Burgt score list and genetic test result or confirmed mutation in any of the genes associated with NS before allocation. Applicable to children with ISS: Age: - Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening For GH treatment naïve participants: Normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performed within 18 months prior to screening. For GH treatment naïve participants: Bone age not delayed more than 2 years compared to chronological age at screening. Exclusion Criteria: Children with suspected or confirmed growth hormone deficiency according to local practice. Children diagnosed with diabetes mellitus or screening values from the central laboratory. Fasting plasma glucose above or equal to 126 milligrams per deciliter (mg/dL) [7.0 millimoles per litre (mmol/L)] or Glycated hemoglobin (HbA1c) above or equal to 6.5%. 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 greater than 400 micrograms per day (µg/day) of inhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening. History or known presence of malignancy including intracranial tumours. Applicable to children with SGA: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Poorly controlled or uncontrolled hormonal deficiencies. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal short stature homeobox (SHOX) gene analysis or absence of GH receptors. Applicable to children with TS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Mosaicism below 10%. TS with Y-chromosome mosaicism where gonadectomy has not been performed. New York Heart Association (NYHA) class II or above or requiring medication for any heart condition. Applicable to children with NS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Noonan-related disorders including but not limited to: Noonan syndrome with multiple lentigines (formerly called 'LEOPARD' syndrome), Noonan syndrome with loose anagen hair, cardiofaciocutaneous syndrome (CFC), Costello syndrome, neurofibromatosis type 1 (NF1) and Legius syndrome. Applicable to children with ISS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Poorly controlled or uncontrolled hormonal deficiencies. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis or absence of GH receptors.

Sites / Locations

  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting
  • Novo Nordisk Investigational SiteRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Somapacitan

Arm Description

Participants will receive Somapacitan for 26-week main phase followed by 130-week extension phase.

Outcomes

Primary Outcome Measures

Number of adverse events (AEs) reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)
Measured as number of events.

Secondary Outcome Measures

Number of adverse events (AEs) possibly or probably related to somapacitan reported separately for SGA, TS, NS and ISS
Measured as number of events.
Number of adverse events (AEs) reported separately for SGA, TS, NS and ISS
Measured as number of events.
Height Velocity reported separately for SGA, TS, NS and ISS
Measured in centimeters per year (cm/year).
Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NS and ISS
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Change in insulin-like growth factor binding protein-3 (IGFBP-3) SDS reported separately for SGA, TS, NS and ISS
Measured as score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Weekly average somapacitan concentration (Cavg) based on population pharmacokinetic (PK) analysis
Measured in nanograms per milliliter (ng/mL).

Full Information

First Posted
January 29, 2023
Last Updated
September 21, 2023
Sponsor
Novo Nordisk A/S
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1. Study Identification

Unique Protocol Identification Number
NCT05723835
Brief Title
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
Acronym
REAL 9
Official Title
A Study Evaluating the Safety and Efficacy of Once-weekly Dosing of Somapacitan in a Basket Study Design in Paediatric Participants With Short Stature Either Born Small for Gestational Age or With Turner Syndrome, Noonan Syndrome or Idiopathic Short Stature
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 1, 2023 (Actual)
Primary Completion Date
August 20, 2024 (Anticipated)
Study Completion Date
March 18, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novo Nordisk A/S

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature. Somapacitan is a new growth hormone medicine for treatment of low level of growth hormone. The study will last for about 3 years. During the study, the participants will be treated with somapacitan once a week. Somapacitan can be injected anytime during the day. The study doctor or nurse will show how to inject somapacitan, so that the participant knows how to do it at home.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
SGA, Turner Syndrome, Noonan Syndrome, ISS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
48 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Somapacitan
Arm Type
Experimental
Arm Description
Participants will receive Somapacitan for 26-week main phase followed by 130-week extension phase.
Intervention Type
Drug
Intervention Name(s)
Somapacitan
Intervention Description
Somapacitan 0.24 milligrams per kilograms per week (mg/kg/week) will be administered subcutaneously (s.c.) using PDS290 pen-injector.
Primary Outcome Measure Information:
Title
Number of adverse events (AEs) reported separately for small for gestational age (SGA), Turner syndrome (TS), Noonan syndrome (NS) and idiopathic short stature (ISS)
Description
Measured as number of events.
Time Frame
From baseline (week 0) to week 26
Secondary Outcome Measure Information:
Title
Number of adverse events (AEs) possibly or probably related to somapacitan reported separately for SGA, TS, NS and ISS
Description
Measured as number of events.
Time Frame
From baseline (week 0) to week 26
Title
Number of adverse events (AEs) reported separately for SGA, TS, NS and ISS
Description
Measured as number of events.
Time Frame
From baseline (week 0) to week 156
Title
Height Velocity reported separately for SGA, TS, NS and ISS
Description
Measured in centimeters per year (cm/year).
Time Frame
From baseline (week 0) to week 26
Title
Change in Height standard deviation scores (SDS) reported separately for SGA, TS, NS and ISS
Description
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Time Frame
From baseline (week 0) to week 26
Title
Change in Height Velocity SDS reported separately for SGA, TS, NS and ISS
Description
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Time Frame
From baseline (week 0) to week 26
Title
Change in insulin-like growth factor 1 (IGF-1) SDS reported separately for SGA, TS, NS and ISS
Description
Measured in score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Time Frame
From baseline (week 0) to week 26
Title
Change in insulin-like growth factor binding protein-3 (IGFBP-3) SDS reported separately for SGA, TS, NS and ISS
Description
Measured as score. Positive score indicates that the value is closer to or above the reference population compared to baseline.
Time Frame
From baseline (week 0) to week 26
Title
Weekly average somapacitan concentration (Cavg) based on population pharmacokinetic (PK) analysis
Description
Measured in nanograms per milliliter (ng/mL).
Time Frame
From baseline (week 0) to week 26

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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). Age: - Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age less than (<) 14 years for females and bone age < 16 years for males. For Growth Hormone (GH) treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. Applicable to children with TS: • Diagnosis of TS according to local clinical practice. Age: - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Impaired height defined as at least 2.0 standard deviation below the mean height for chronological age and sex at screening according to the standards of Centers for Disease Control and Prevention. For GH treatment naïve participants: Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis or confirmation of TS and TS mosaicism using comparative genomic hybridization (CGH)-array. Applicable to children with NS: Diagnosis of NS according to local clinical practice. Age: Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Clinical diagnosis of NS according to van der Burgt score list and genetic test result or confirmed mutation in any of the genes associated with NS before allocation. Applicable to children with ISS: Age: - Male participants: Age equal to or above 11.0 years and below 18.0 years at screening. - Female participants: Age equal to or above 10.0 years and below 18.0 years at screening. Open epiphyses; defined as bone age < 14 years for females and bone age < 16 years for males. For GH treatment naïve participants: Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and sex at screening For GH treatment naïve participants: Normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performed within 18 months prior to screening. For GH treatment naïve participants: Bone age not delayed more than 2 years compared to chronological age at screening. Exclusion Criteria: Children with suspected or confirmed growth hormone deficiency according to local practice. Children diagnosed with diabetes mellitus or screening values from the central laboratory. Fasting plasma glucose above or equal to 126 milligrams per deciliter (mg/dL) [7.0 millimoles per litre (mmol/L)] or Glycated hemoglobin (HbA1c) above or equal to 6.5%. 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 greater than 400 micrograms per day (µg/day) of inhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) for longer than 4 consecutive weeks within the last 12 months prior to screening. History or known presence of malignancy including intracranial tumours. Applicable to children with SGA: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Poorly controlled or uncontrolled hormonal deficiencies. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal short stature homeobox (SHOX) gene analysis or absence of GH receptors. Applicable to children with TS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Mosaicism below 10%. TS with Y-chromosome mosaicism where gonadectomy has not been performed. New York Heart Association (NYHA) class II or above or requiring medication for any heart condition. Applicable to children with NS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Noonan-related disorders including but not limited to: Noonan syndrome with multiple lentigines (formerly called 'LEOPARD' syndrome), Noonan syndrome with loose anagen hair, cardiofaciocutaneous syndrome (CFC), Costello syndrome, neurofibromatosis type 1 (NF1) and Legius syndrome. Applicable to children with ISS: • Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with height, such as, but not limited to: Known family history of skeletal dysplasia. Significant spinal abnormalities including but not limited to scoliosis, kyphosis and spina bifida variants. Any other disorder that can cause short stature such as, but not limited to nutritional disorders, chronic systemic illness and chronic renal disease. Poorly controlled or uncontrolled hormonal deficiencies. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes' with short stature, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis or absence of GH receptors.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Novo Nordisk
Phone
(+1) 866-867-7178
Email
clinicaltrials@novonordisk.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Transparency (dept. 2834)
Organizational Affiliation
Novo Nordisk A/S
Official's Role
Study Director
Facility Information:
Facility Name
Novo Nordisk Investigational Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95821
Country
United States
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Centennial
State/Province
Colorado
ZIP/Postal Code
80112
Country
United States
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010-2978
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Novo Nordisk Investigational Site
City
Idaho Falls
State/Province
Idaho
ZIP/Postal Code
83404-7596
Country
United States
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Individual Site Status
Not yet recruiting
Facility Name
Novo Nordisk Investigational Site
City
Yangsan
ZIP/Postal Code
50612
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Yangsan
ZIP/Postal Code
50612
Country
Korea, Republic of
Individual Site Status
Not yet recruiting
Facility Name
Novo Nordisk Investigational Site
City
Bandar Puncak Alam Selangor Darul Ehsan
ZIP/Postal Code
42300
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Kuala Lumpur
ZIP/Postal Code
59100
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands
Individual Site Status
Not yet recruiting
Facility Name
Novo Nordisk Investigational Site
City
Rzeszow
State/Province
Podkarpackie Voivodeship
ZIP/Postal Code
35-301
Country
Poland
Individual Site Status
Not yet recruiting
Facility Name
Novo Nordisk Investigational Site
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Lodz
ZIP/Postal Code
93-338
Country
Poland
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Zabrze
ZIP/Postal Code
41-800
Country
Poland
Individual Site Status
Recruiting
Facility Name
Novo Nordisk Investigational Site
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
IPD Sharing URL
http://novonordisk-trials.com

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A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9

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