Active not recruitingPHASE3INTERVENTIONAL

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow

This research study is testing a new medicine called somapacitan, given once a week, to help children grow. It's being compared to Norditropin®, a medicine that doctors already use for growth, which is given once a day. The study is for children who were born small and stayed small, or those with specific conditions like Turner Syndrome, Noonan Syndrome, or idiopathic short stature (meaning the cause of short height isn't known). We want to see how well somapacitan works compared to Norditropin®. Children in the study will either receive somapacitan once a week for up to 5.5 years, or Norditropin® once a day for one year followed by somapacitan once a week for up to 4.5 years. Which treatment a child gets is decided randomly, like flipping a coin. The study will last for up to five and a half years.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Novo Nordisk A/S

Enrolment target

412

Start

10 Aug 2022

Estimated completion

29 Oct 2027

SGA, Turner Syndrome, Noonan Syndrome, ISS

What is this study about?

This study is designed to help children who aren't growing as expected. Sometimes, children are born smaller than average and continue to be short, or they might have conditions like Turner Syndrome or Noonan Syndrome, which can affect their growth. There are also children whose short height doesn't have a clear explanation, which doctors call idiopathic short stature.

The main goal of this study is to look at a new medicine called somapacitan. Currently, medicines like Norditropin® are available to help children grow. Somapacitan is designed to be given just once a week, which could be more convenient than daily injections. By comparing somapacitan to Norditropin®, researchers hope to understand if the new weekly medicine is just as good, or even better, at helping children grow taller.

Taking part in this study means your child would receive one of these medicines. Half the children would get the new weekly medicine, somapacitan, for the whole study period (up to 5.5 years). The other half would start with the daily medicine, Norditropin®, for one year, and then switch to the weekly somapacitan for the remaining time. The choice of which group your child would be in is made completely by chance, similar to drawing straws. This ensures the results are fair and reliable.

Key takeaways

The study compares a new weekly growth medicine (somapacitan) with a daily one (Norditropin®).
It's for children aged 2.5 to 10 years who need help with their growth.
Conditions include being born small and staying short, Turner Syndrome, Noonan Syndrome, or unexplained short stature.
Children will be randomly assigned to receive one of two treatment plans.
The study will involve regular clinic visits and take up to 5.5 years.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for children aged between 2.5 and 10 years old. If you're considering this study, your child must not have received any growth-boosting treatments before, such as growth hormone medicines.

For children who were born small for their age (SGA), their birth length or weight would have been significantly below average. They also need to be clearly shorter than most children their age and sex, and their growth rate needs to be slower than expected. For boys, they must be between 2 years, 6 months and 11 years old and show no signs of puberty. For girls, they must be between 2 years, 6 months and 10 years old and also show no signs of puberty. Their body mass index (BMI) should also be within a healthy range.

Girls with Turner Syndrome would need a confirmed diagnosis of this condition. Like the other groups, they must be between 2 years, 6 months and 10 years old and have no signs of puberty. They also need to be shorter than most girls their age.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child between 2 years, 6 months and 10 years old?
Has my child never had growth hormone treatment before?
Is my child significantly shorter than other children their age?
Does my child fit one of the categories: born small and staying short, Turner Syndrome, Noonan Syndrome, or unexplained short stature?
Are there no signs of puberty for my child (for boys under 11, for girls under 10)?

Answer every question to see your result.

What does participation involve?

If your child joins this study, they will have regular visits to the clinic over a long period, potentially up to five and a half years. These visits will involve various checks to monitor their health and growth, such as measuring their height and weight. They will also have blood tests and other assessments to see how they are responding to the medication. Your child will receive either somapacitan once a week, administered through an injection, or Norditropin® once a day, also through an injection, depending on which group they are randomly assigned to. After one year, some children might switch from Norditropin® to somapacitan. You'll be given clear instructions on how to administer the medicine at home. Throughout the study, the research team will be there to support you and your child, and answer any questions you may have. The exact number of visits and specific tests will be explained in detail if you decide to take part.

Potential risks and benefits

Taking part in a study like this could potentially benefit your child by providing access to new growth-promoting treatments that aren't yet widely available, or by offering a familiar treatment with close medical supervision. However, there are also potential risks, such as side effects from the medicines, discomfort from injections or blood tests, or the time commitment involved with clinic visits. The research team will carefully explain all known side effects. It's very important to remember that participating is entirely voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (199)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Univ of AL at Birmingham_BRM
Verified postcode
Birmingham, United States
Children's Hospital Los Angeles - Endocrinology
Verified postcode
Los Angeles, United States
Sutter Valley Med Fdt Ped Endo
Verified postcode
Sacramento, United States
Rady Childrens Hosp San Diego
Verified postcode
San Diego, United States
Children's Hospital Colorado
Verified postcode
Aurora, United States
Rocky Mt Ped and Endo
Verified postcode
Centennial, United States
Ped Endo Assoc PC-G.V
Verified postcode
Greenwood Village, United States
Nemours/AI duPont Hosp-Chld
Verified postcode
Wilmington, United States
Childrens National Medical Ctr
Verified postcode
Washington D.C., United States
Nemours Chld Clnc Jacksonville
Verified postcode
Jacksonville, United States
Atlanta Diabetes Associates
Verified postcode
Atlanta, United States
St. Luke's Children's Endo
Verified postcode
Boise, United States

Common questions

What is 'idiopathic short stature'?

This simply means that a child is significantly shorter than average, and doctors can't find a clear medical reason for it.

How is the medicine given?

Both medicines are given as injections, either once a day or once a week, depending on which one your child receives.

How long does the study last?

The study could last for up to five and a half years to see the long-term effects of the treatment.

Will my child definitely get the new medicine?

It's decided by chance, like a lottery. Your child could get the new weekly medicine or the daily medicine already in use.

What if we change our minds about participating?

You can withdraw your child from the study at any time, for any reason, and it won't affect their regular medical care.