Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
This study is called the OraGrowtH Phase 3 Trial and is looking at an investigational new medicine called LUM-201. It's a pill being tested for children with Growth Hormone Deficiency (GHD) who haven't had treatment before. The main goals are to find out if LUM-201 helps children with GHD grow better and to see if a specific test, called the LUM-201 PEM (predictive enrichment marker) strategy, can accurately identify which children are most likely to respond well to this daily oral treatment. This is a large study taking place in different countries, focusing on ensuring the treatment is safe and effective for young people.
At a glance
What is this study about?
This study is a research trial exploring a new medication called LUM-201. It's a pill designed for children who have a condition called Growth Hormone Deficiency (GHD). This means their body doesn't produce enough growth hormone, which is important for them to grow and develop properly. Currently, many children with GHD need daily injections, so having a pill could make treatment much easier.
The main aim of this trial is to see if LUM-201 helps children with GHD grow better. Researchers will carefully measure the children's growth to check if the medicine is effective. Another important part of the study is to test a special method, called the LUM-201 PEM strategy. This method aims to predict which children are most likely to respond well to the LUM-201 treatment. If successful, this could help doctors in the future to choose the best treatment for each child right from the start.
This trial is in its third phase (Phase 3), meaning it's a large study involving many children in different countries. This stage is crucial because it helps confirm if the treatment is safe and works consistently across a broad group of people before it can potentially become more widely available. It's all about making sure that any new treatment is as good and safe as possible for children who need it.
Key takeaways
- This study is testing a new oral medication (pill) for children with Growth Hormone Deficiency (GHD).
- It aims to see if the medicine helps children grow and if a special test can predict who will respond best.
- Children will either receive the active medicine or a 'dummy' pill (placebo).
- Regular clinic visits and health checks will be part of taking part.
- This is a large, international study (Phase 3) to ensure the medicine's safety and effectiveness.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, children need to meet certain requirements. They must be between 3 and 10 years old if they are a girl, or between 3 and 11 years old if they are a boy. They should not have received any previous treatment for Growth Hormone Deficiency (GHD) and must not have started puberty yet. Their height should be quite a bit shorter than what's typical for their age and sex, and their bones should show a delay in development compared to their real age. We also need to see that their body isn't producing enough growth hormone, based on special tests.
There are also some reasons why a child might not be able to join. For example, if they have any other serious medical conditions or genetic issues that could affect their growth or make the study medicine risky. If they are taking certain medications that could interfere with growth or the study drug, they might also not be eligible. We also need to make sure their heart is healthy based on an ECG test.
Girls will need specific genetic tests to rule out a condition called Turner syndrome, and any other genetic tests related to short stature should be negative. If a child has an underactive thyroid, it needs to be successfully treated for at least 3 months before starting the study. These rules are in place to ensure the safety of the children taking part and to make sure the study results are as clear and accurate as possible.
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 your child a girl aged 3 to 10, or a boy aged 3 to 11?
- Has your child never been treated for Growth Hormone Deficiency before?
- Is your child shorter than expected for their age and sex?
- Has your child's doctor confirmed they have Growth Hormone Deficiency with specific tests?
- Does your child NOT have other serious medical or genetic conditions that could cause short stature?
- Is your child NOT taking medications that interfere with growth or growth hormone treatment?
What does participation involve?
If your child takes part in this study, they will receive either the study medication LUM-201 or a matched placebo (a dummy pill that looks the same but contains no active medicine) in capsule form. You won't know which one your child is receiving. They will need to take this medicine daily. Participation will involve regular visits to the study clinic where various assessments will be conducted. These assessments will include measuring height and weight, taking blood samples, and having bone age X-rays. You'll also need to keep track of your child's growth and any health changes at home. The total duration of participation in the study, including follow-up, will be determined by the study design, but generally, Phase 3 trials run for a significant period to monitor long-term effects.
Potential risks and benefits
Locations (29)
- Lumos Pharma Investigational SiteVerified postcodeBirmingham, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeMadera, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeSacramento, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeCentennial, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeGreenwood Village, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeWashington D.C., United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeHollywood, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeMiami, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeOrlando, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeTallahassee, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeIndianapolis, United States· Recruiting
- Lumos Pharma Investigational SiteVerified postcodeIowa City, United States· Recruiting
Common questions
What is Growth Hormone Deficiency (GHD)?
GHD is a medical condition where a child's body doesn't produce enough growth hormone, which is essential for normal growth and development.
What is a 'placebo'?
A placebo is a 'dummy' pill that looks just like the study medicine but contains no active drug. It helps researchers compare the effects of the active medicine.
Will my child definitely get the new medicine?
No, children in this study will either receive the new medicine (LUM-201) or a placebo. Neither you nor the study team will know which until the study is over.
How long will the study last?
Phase 3 trials typically last for a significant period, often many months or even a few years, to carefully study the effects of the treatment. The exact duration will be discussed by the study team.
What if we change our mind about participating?
You are free to withdraw your child from the study at any time, for any reason, without it affecting your child's regular medical care.
How to find out more
Lumos Pharma
Always speak to your GP or specialist before deciding to take part in a study.
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