A Phase 2, Multi-center, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety and Efficacy of AGA2115 in Adults with Type I, III, or IV Osteogenesis Imperfecta (OI)
Osteogenesis imperfecta (OI) is often known as brittle bone disease. People living with OI have fragile bones that can break easily. This clinical trial is testing an experimental medicine called AGA2115 to find out if it can help improve bone strength and reduce fractures in adults with Type I, III, or IV OI. Participants will be placed into groups by chance. Some will receive different doses of AGA2115, while others will receive a placebo (a dummy treatment that contains no active medicine). Neither the participants nor the research doctors will know who gets which treatment during the study. Over 12 months, doctors will track changes in bone density in the lower spine using painless bone scans. They will also keep a close eye on safety and overall health throughout the trial.
At a glance
What is this study about?
Osteogenesis imperfecta (OI), commonly called brittle bone disease, is a genetic condition where the body does not make collagen properly or makes too little of it. Collagen is a key protein needed to build strong bones. Because of this, people with OI often face frequent broken bones, pain, and other bone-related complications throughout their lives. Current treatments can help manage symptoms, but doctors are looking for new therapies that can directly strengthen the skeleton.
This trial is looking at a new drug called AGA2115. The main aim is to find out if AGA2115 can increase bone mineral density—a measure of how strong and solid bones are—specifically in the lower back (lumbar spine). Researchers also want to find the safest and most effective dose for adults with three common types of the condition: Type I, Type III, and Type IV.
Finding new ways to boost bone density could make a meaningful difference to everyday life for adults with OI. Stronger bones may lead to fewer painful fractures, less hospital care, and better mobility. Because this is an early-stage study, it will provide crucial clues on whether AGA2115 should be developed into an approved NHS treatment in the future.
Key takeaways
- Tests an experimental drug, AGA2115, for adults with brittle bone disease (OI).
- Aimed at people aged 18 and over with Type I, III, or IV osteogenesis imperfecta.
- Measures bone density changes in the lower spine over a 12-month period.
- Participants will receive either the study medicine or a dummy treatment (placebo).
- Taking part is voluntary, and you can withdraw at any time.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with osteogenesis imperfecta Type I, III, or IV. These types range from mild to more severe forms of the condition. Men and women of any background can take part.
To join, participants will need to be in generally stable health, aside from their OI. The study team will review medical history, previous bone treatments, and current medications to make sure the trial is safe for each person.
People who are pregnant, planning to become pregnant, or who have certain other serious health conditions may not be able to participate. The trial team will carry out standard health checks before anyone officially enrols.
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.
- I am aged 18 or older.
- I have been diagnosed with Osteogenesis Imperfecta Type I, III, or IV.
- I am able to attend regular hospital clinic visits over a 12-month period.
- I am willing to undergo routine bone scans and blood tests.
- I am comfortable receiving either the trial medicine or a placebo.
What does participation involve?
Taking part in this study lasts for at least 12 months. During this time, participants will be randomly chosen to receive either the active medicine (at one of several doses) or a placebo. You will need to take the study treatment as directed by the clinic team. You will have regular appointments at a specialist hospital clinic so doctors can monitor your health. These visits will include health check-ups, blood and urine tests, and DXA scans (low-dose X-ray scans that measure bone density). You will also be asked about any fractures, aches, or side effects you experience. Travel expenses are often covered by trial programmes.
Potential risks and benefits
Locations (3)
- —UnverifiedNetherlands
- —UnverifiedFrance
- —UnverifiedDenmark
Common questions
What is a placebo?
A placebo is a harmless dummy treatment that looks just like the real medicine but contains no active drug. It helps doctors see whether changes are truly caused by AGA2115.
Can I choose to have the real medicine instead of the placebo?
No. A computer assigns participants to either AGA2115 or the dummy medicine by chance. Neither you nor your study doctor can choose your group.
Does this trial offer a cure for brittle bone disease?
No. The trial is testing whether the medicine can strengthen bones and reduce fractures, but it is not a cure for the genetic cause of OI.
Are the bone density scans painful?
No. Bone density scans (DXA scans) are completely painless, quick, and use very low amounts of radiation.
What happens if I want to stop taking part?
You are free to leave the trial at any point. You do not need to give a reason, and deciding to leave will not affect your normal NHS treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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