دراسة لتقييم كيفية عمل Apitegromab في الأشخاص الذين تقل أعمارهم عن سنتين ويعانون من ضمور العضلات الشوكي
This research study is investigating a new medication called apitegromab for very young children, specifically those under two years old, who have a condition called spinal muscular atrophy (SMA). SMA affects muscles and can make it difficult for children to move and meet developmental milestones. In this study, doctors want to understand several things: how the body absorbs and uses apitegromab, whether it helps to improve or maintain muscle function, and if it's safe to use. All children taking part will already be receiving other approved SMA treatments. This study is in an early stage (Phase 2), meaning it's still gathering important information about this new potential treatment.
نظرة سريعة
ما هو موضوع هذه الدراسة؟
This study is all about a new medicine called apitegromab for very young children, specifically those under two years old, who have a condition called spinal muscular atrophy (SMA). SMA is a genetic condition that makes muscles weaker over time, affecting a child's ability to move, sit up, and sometimes breathe. There are different types of SMA, and this study focuses on children with specific types (known as 5q autosomal recessive SMA).
The main goal of this research is to see if apitegromab could be a helpful new treatment. Because it's a new medicine, the researchers want to carefully check a few things. They'll be looking at how the body deals with the medicine (this is called PK/PD), how well it works to help with muscle strength and movement (its efficacy), and most importantly, whether it's safe and well-tolerated by the children receiving it.
It's important to know that all children taking part in this study will already be receiving another approved treatment for SMA, such as Nusinersen or Risdiplam. This study is testing apitegromab in addition to, or alongside, these existing treatments to see if it can offer further benefits. This is a "Phase 2" study, which means it's an important step in understanding a new medicine, but it's not the final stage before a medicine becomes widely available.
النقاط الرئيسية
- This study is for children under 2 with specific types of SMA.
- It's testing a new medicine called apitegromab alongside existing SMA treatments.
- The aim is to check the medicine's safety, how it works, and if it helps with movement.
- Regular health checks and movement assessments will be part of the study.
- Families can decide to stop participating at any time.
من قد يكون مؤهلاً؟
To join this study, a child must be under two years old and have a confirmed diagnosis of 5q autosomal recessive SMA. They also need to have received, or still be receiving, an approved treatment for SMA. The child's overall health and growth will be checked to make sure they are well enough to take part, and they must show some delays in their movement skills related to SMA.
There are also some reasons why a child might not be able to join. For instance, if they have major bone problems like severe curvature of the spine (scoliosis) or stiff joints that would make it hard to measure their movement, or if they've had recent major surgery related to these issues. Children who need a permanent feeding tube for most of their meals, or have other physical limitations that would stop them from doing movement tests, also can't 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 طفلك أقل من سنتين؟
- Does your child have a confirmed diagnosis of 5q SMA?
- Is your child currently receiving, or has received, an approved SMA treatment?
- Does your child have some delays in their movement skills related to SMA?
- Does your child not have severe bone problems or recent major surgery that would prevent movement tests?
ماذا يتضمن المشاركة؟
If your child takes part, they will receive doses of the study medicine, apitegromab. This study is "double-blind," meaning neither you nor the study doctors will know if your child is receiving the active medicine or a placebo (a dummy medicine) – this helps ensure fair results. Your child will have regular visits to the clinic where doctors will check their health, take blood samples to see how the medicine is working in their body, and perform tests to assess their muscle function and development, such as movement tests. The total duration of the study will involve several visits over a period of time, with ongoing follow-up to monitor your child's progress and safety.
المخاطر والفوائد المحتملة
المواقع (23)
- مستشفى فينيكس للأطفالالرمز البريدي المؤكدفينيكس، الولايات المتحدة· التوظيف
- Children's Hospital of Orange County (CHOC)الرمز البريدي المؤكدأورانج، الولايات المتحدة· التوظيف
- Stanford Neuroscience Health Center (SNHC)الرمز البريدي المؤكدبالو ألتو، الولايات المتحدة· التوظيف
- مستشفى الأطفال كولورادوالرمز البريدي المؤكدأورورا، الولايات المتحدة· التوظيف
- الرعاية الصحية للأطفال في أتلانتاالرمز البريدي المؤكدأتلانتا، الولايات المتحدة· التوظيف
- جامعة آيواالرمز البريدي المؤكدمدينة آيوا، الولايات المتحدة· التوظيف
- Helen DeVos Children's Hospital at Spectrum Healthالرمز البريدي المؤكدغراند رابيدز، الولايات المتحدة· التوظيف
- مستشفى الأطفال في فيلادلفياالرمز البريدي المؤكدفيلادلفيا، الولايات المتحدة· التوظيف
- مركز جامعة تكساس ساوثويسترن الطبيالرمز البريدي المؤكددالاس، الولايات المتحدة· التوظيف
- مركز أمراض الأعصاب النادرةالرمز البريدي المؤكدFlower Mound, United States· التوظيف
- مركز كوك الطبي للأطفالالرمز البريدي المؤكدفورت وورث، الولايات المتحدة· التوظيف
- Texas Children's Hospital (TCH)-Clinical Care Center (CCC)الرمز البريدي المؤكدهيوستن، الولايات المتحدة· التوظيف
أسئلة شائعة
What is SMA?
Spinal Muscular Atrophy (SMA) is a genetic condition that causes muscles to weaken and waste away over time, affecting movement and sometimes breathing.
What is apitegromab?
Apitegromab is a new medicine being studied to see if it can help children with SMA improve their muscle function.
Will my child still receive their current SMA treatment?
Yes, children in this study will continue to receive their approved SMA treatments like Nusinersen or Risdiplam alongside the study medicine.
What does "double-blind" mean?
It means neither you nor the doctors will know if your child is getting the actual study medicine or a dummy medicine, to ensure fair and unbiased results.
ما هي دراسة المرحلة الثانية؟
A Phase 2 study is an early stage of clinical research where the main goals are to gather more information about a medicine's safety, how it works in the body, and its effectiveness.
كيفية معرفة المزيد
Scholar Rock
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نقاش
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