SOMAPACITAN for Short stature in children born small for gestational age (SGA)

Inclusion criteria

• {"criterion_text":"- Prepubertal children: a)\tBoys: o\tAge ≥ 2 years and 26 weeks and < 11.0 years at screening o Testis volume < 4 ml b)\tGirls: o\tAge ≥ 2 years and 26 weeks and <10.0 years at screening o\tTanner stage 1 for breast development (no palpable glandular breast tissue)"}
• {"criterion_text":"- Born small for gestational age (birth length and/or weight < -2 standard deviation scores) (according to national standards)."}
• {"criterion_text":"- Impaired height defined as at least 2.5 standard deviations below the mean height for chronological age and gender at screening according to the standards of Centers for Disease Control and Prevention at screening."}
• {"criterion_text":"- Impaired height velocity defined as annualised height velocity below the 50th 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."}
• {"criterion_text":"- No prior exposure to growth hormone therapy or Insulin-like Growth Factor-I (IGF-I) treatment."}

Exclusion criteria

• {"criterion_text":"- Any known or suspected clinically significant abnormality likely to affect growth or the ability to evaluate growth with standing height measurements."}
• {"criterion_text":"- Children with hormonal deficiencies including suspected or confirmed growth hormone deficiency according to local practise."}
• {"criterion_text":"- Current inflammatory diseases requiring systemic corticosteroid treatment for longer than 2 consecutive weeks within the last 3 months prior to screening."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Diagnosis of attention deficit hyperactivity disorder."}
• {"criterion_text":"- Prior history or presence of malignancy including intracranial tumours."}

Primary endpoints

• {"endpoint_text":"- Endpoint title: Height velocity (HV) Time frame: From baseline (week 0) to visit 6 (week 26) Unit: cm/year","definition_or_measurement_approach":"Height velocity measured in cm/year from baseline (week 0) to visit 6 (week 26)."}

Secondary endpoints

• {"endpoint_text":"- Effect Endpoint title: Change in bone age Time frame: From baseline (week 0) to visit 8 (week 52) Unit: Years","definition_or_measurement_approach":"Change in bone age (years) from baseline to week 52."}
• {"endpoint_text":"- Effect Endpoint title: Change in height SDS Time frame: From baseline (week 0) to visit 6 (week 26) Unit: -10 to +10","definition_or_measurement_approach":"Change in height standard deviation score (SDS) from baseline to week 26; unit scale reported as -10 to +10."}
• {"endpoint_text":"- Effect Endpoint title: Change in Height velocity SDS Time frame: From baseline (week 0) to visit 6 (week 26) Unit: -10 to +10","definition_or_measurement_approach":"Change in height velocity SDS from baseline to week 26; unit scale reported as -10 to +10."}
• {"endpoint_text":"- Safety Endpoint title: Change in fasting plasma glucose Time frame: From screening (visit 1) to visit 6 (week 26) Unit: mmol/l","definition_or_measurement_approach":"Change in fasting plasma glucose (mmol/l) from screening to week 26."}
• {"endpoint_text":"- Safety Endpoint title: Change in homeostatic model assessment (HOMA) Time frame: From screening (visit 1) to visit 6 (week 26) Unit: %","definition_or_measurement_approach":"Change in HOMA index (%) from screening to week 26."}
• {"endpoint_text":"- Safety Endpoint title: Change in Glycated haemoglobin (HbA1c) Time frame: From screening (visit 1) to visit 6 (week 26) Unit: % point","definition_or_measurement_approach":"Change in HbA1c (% point) from screening to week 26."}
• {"endpoint_text":"- Pharmacodynamics Endpoint title: Change in IGF-I SDS Time frame: From baseline (week 0) to visit 6 (week 26) Unit: -10 to +10","definition_or_measurement_approach":"Change in IGF-I SDS from baseline to week 26; unit scale reported as -10 to +10."}
• {"endpoint_text":"- Pharmacodynamics Endpoint title: Change in IGFBP-3 SDS Time frame: From baseline (week 0) to visit 6 (week 26) Unit: -10 to +10","definition_or_measurement_approach":"Change in IGFBP-3 SDS from baseline to week 26; unit scale reported as -10 to +10."}

Austria

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

712

Number Of Sites

1

Number Of Participants

1

France

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

17-03-2026

Processing Time Days

706

Number Of Sites

2

Number Of Participants

3

Hungary

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

23-03-2026

Processing Time Days

712

Number Of Sites

1

Number Of Participants

1

Latvia

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

30-09-2024

Processing Time Days

173

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

10-04-2024

Latest Decision Or Authorization Date

20-03-2026

Processing Time Days

709

Number Of Sites

2

Number Of Participants

3

Primary sponsor

Full Name

Novo Nordisk A/S

Organisation Type

Pharmaceutical company

Country Of Registered Address

Denmark

Contract research organisations

Name

WCG Clinical Inc.

Responsibilities

sponsorDuties code: 10

Name

IQVIA Limited

Responsibilities

sponsorDuties code: 4

Name

Syneos Health Clinique Inc.

Responsibilities

sponsorDuties code: 4

Third parties

• {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"sponsorDuties code: 10","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"eDiary supplier (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"Imaging Laboratory (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Syneos Health Clinique Inc.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United Kingdom","full_name":"Perceptive Eclinical Limited","duties_or_roles":"IWRS Supplier (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Oracle America Inc.","duties_or_roles":"CRF supplier (sponsorDuties code: 15)","organisation_type":"Pharmaceutical company"}