A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need He… (NCT05723835) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
United States, Malaysia, Netherlands47 participantsStarted 2023-02-01
Plain-language 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.
Who can participate
Age range
10 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Adverse Events (AEs) Reported in Children Born Small for Gestational Age- Weeks 0 to 26
Timeframe: From baseline (Week 0) to Week 26
2
Number of Adverse Events Reported for Turner Syndrome (TS)- Weeks 0 to 26
Timeframe: From baseline (Week 0) to Week 26
3
Number of Adverse Events Reported for Noonan Syndrome- Weeks 0 to 26
Timeframe: From baseline (Week 0) to Week 26
4
Number of Adverse Events Reported for Idiopathic Short Stature (ISS)- Weeks 0 to 26