Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
This research study, called IDUN, is looking into a new medicine, AGA2115, for adults aged 18 to 75 who have a genetic bone condition called osteogenesis imperfecta (OI) types I, III, or IV. OI makes bones fragile and prone to breaking. The study wants to find out if AGA2115 is safe and effective in improving bone health by comparing it to a placebo (a dummy drug with no active medicine). Participants will receive either AGA2115 or a placebo for the first year, and then everyone will receive AGA2115 for another year, followed by a three-month check-up period. The total study duration is 27 months.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
This study, called IDUN, is focused on adults who live with a condition called osteogenesis imperfecta, often shortened to OI. OI is a genetic condition meaning it's passed down through families. It makes your bones very fragile and can lead to them breaking easily, even from minor bumps or falls. In this study, we are specifically looking at people with Type I, Type III, or Type IV OI, and where genetic tests have confirmed changes in certain genes that are linked to the condition.
The main goal of this study is to test a new investigational medicine called AGA2115. We want to see if AGA2115 can help make bones stronger and reduce the risk of fractures in adults with OI. We'll also be carefully checking to make sure the medicine is safe and doesn't cause too many side effects. This research is important because it could potentially offer a new treatment option for people living with this challenging bone condition.
To understand if AGA2115 truly helps, some participants will receive the active medicine, while others will receive a 'placebo.' A placebo looks just like the real medicine but contains no active ingredients. This helps researchers compare the effects of AGA2115 fairly. After the first year, everyone in the study will then receive AGA2115, allowing us to gather more information on its longer-term effects.
النقاط الرئيسية
- This study is for adults (18-75) with specific types (I, III, IV) of osteogenesis imperfecta.
- It tests a new medicine, AGA2115, against a dummy treatment (placebo) to see if it makes bones stronger.
- The study lasts over two years, with regular clinic visits.
- You might receive the active medicine or a placebo for the first year; everyone gets the active medicine for the second year.
- Your health condition and other medicines you take will affect whether you can join.
- المشاركة طوعية، ويمكنك الانسحاب في أي وقت.
من قد يكون مؤهلاً؟
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.
هل يمكن أن تناسبك هذه الدراسة؟
أجب عن هذه الأسئلة السريعة لترى ما إذا كنت مؤهلاً. هذا دليل فقط - الفريق البحثي هو الذي يتخذ القرار النهائي.
- هل عمرك بين 18 و 75 سنة؟
- Do you have a confirmed diagnosis of OI Type I, III, or IV with specific gene changes?
- Have you recently used certain bone medications (like bisphosphonates, Denosumab, or Romosozumab)?
- Are you pregnant, breastfeeding, or planning to become pregnant?
- Do you have other serious health problems, like recent heart problems or certain hormone imbalances?
ماذا يتضمن المشاركة؟
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.
المخاطر والفوائد المحتملة
المواقع (26)
- Phoenix Children'sالرمز البريدي المؤكدفينيكس، الولايات المتحدة· التوظيف
- كلية الطب بجامعة ييلالرمز البريدي المؤكدنيو هيفن، الولايات المتحدة· التوظيف
- Nemours/Alfred I. duPont Hospital for Childrenالرمز البريدي المؤكدويلمنجتون، الولايات المتحدة· التوظيف
- Indiana University School of Medicine, Department of Medicine and Pediatrics Division of Endocrinologyالرمز البريدي المؤكدإنديانابوليس، الولايات المتحدة· التوظيف
- كلية الطب بجامعة واشنطن في سانت لويسالرمز البريدي المؤكدسانت لويس، الولايات المتحدة· لم يبدأ التوظيف بعد
- New Mexico Clinical Research & Osteoporosis Center, Inc. (NMCROC)الرمز البريدي المؤكدألبوكيرك، الولايات المتحدة· التوظيف
- The Ohio State University Wexner Medical Center (OSUWMC)الرمز البريدي المؤكدكولومبوس، الولايات المتحدة· لم يبدأ التوظيف بعد
- Oregon Health & Science University (OHSU) - The Harold Schnitzer Diabetes Health Center (HSDHC) - Endocrinology Clinicالرمز البريدي المؤكدبورتلاند، الولايات المتحدة· التوظيف
- Vanderbilt University Medical Center (VUMC) - Eskind Diabetes Clinicالرمز البريدي المؤكدناشفيل، الولايات المتحدة· لم يبدأ التوظيف بعد
- Instituto de Investigaciones Metabolicas Dr. Zanchetta - Sede Centroالرمز البريدي المؤكدبوينس آيرس، الأرجنتين· التوظيف
- Monash University-Monash Medical Centre (MMC)الرمز البريدي المؤكدملبورن، أستراليا· التوظيف
- مستشفى رويال ملبورنالرمز البريدي المؤكدباركفيل، أستراليا· التوظيف
أسئلة شائعة
What is osteogenesis imperfecta (OI)?
OI is a genetic bone condition that makes your bones very fragile and likely to break easily.
ما هو الدواء الوهمي؟
A placebo is a dummy treatment that looks like the real medicine but doesn't contain any active drug. It helps researchers compare the effects of the active medicine fairly.
كم ستستغرق مدة الدراسة؟
The study will last a total of 27 months, which includes 24 months of treatment and 3 months of follow-up.
هل سأعرف إذا كنت أحصل على الدواء الحقيقي أم الدواء الوهمي؟
For the first 12 months, neither you nor your study doctor will know if you're receiving the active medicine or the placebo. After that, everyone will receive the active medicine.
هل يمكنني مغادرة الدراسة إذا غيرت رأيي؟
Yes, you can leave the study at any time, for any reason, and it won't affect your medical care.
كيفية معرفة المزيد
Kimberly Brown
تحدث دائمًا إلى طبيبك العام أو الأخصائي قبل اتخاذ قرار بالمشاركة في دراسة.
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نقاش
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