Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)

To join this study, you need to be an adult between 18 and 75 years old and have a confirmed diagnosis of osteogenesis imperfecta (OI) Type I, III, or IV. This diagnosis must be supported by genetic test results showing specific changes in your COL1A1 or COL1A2 genes. Additionally, your bone density at your lower back (lumbar spine), hip, or the top of your thigh bone (femoral neck) needs to be lower than a certain level (a T-score of -1.0 or less).

There are also some reasons why you might not be able to join. For instance, if you have a vitamin D deficiency or other conditions that seriously affect your bone health, like certain thyroid problems, you likely wouldn't be eligible. Also, if you've recently had a heart attack or stroke, or if you've been treated with certain other bone medications within the past few months or years, you wouldn't qualify. Women who are pregnant, breastfeeding, or planning to get pregnant during the study also cannot take part.

If you decide to join this study, it will last for a total of 27 months (about two and a quarter years). For the first 12 months, you will either receive the study medicine, AGA2115, or a placebo (a dummy drug), which will be decided randomly, like flipping a coin. You won't know whether you're getting the active medicine or the placebo, and neither will your study doctor – this is called 'double-blind'.

During this time and for the next 12 months (making it 24 months of treatment), you'll need to attend regular visits to the clinic. At these visits, doctors and nurses will check how you're feeling, perform various assessments, and take measurements to see how the treatment is affecting you and to ensure your safety. For the final 3 months, you'll have a follow-up period without further treatment, where your health will be checked one last time. You will be given the study medication to take as instructed by the study team.