Registry for pediatric spinal muscular atrophy in China
Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
This study is trying to learn more about how spinal muscular atrophy affects kids in China and how well different treatments work for them.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 600 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Biogen Industry-sponsored |
| Locations | 25 sites (Hefei, Anhui and 24 other locations) |
| Trial ID | NCT05042921 on ClinicalTrials.gov |
What this trial studies
This observational study aims to document the natural history and treatment outcomes of pediatric patients with spinal muscular atrophy (SMA) linked to chromosome 5q in China. It will collect data both prospectively and retrospectively to understand how disease-modifying therapies are utilized and their effectiveness in a real-world setting. The study will involve participants under 18 years of age who have a confirmed genetic diagnosis of 5q-SMA. By gathering this information, the study seeks to provide insights into the progression of SMA and the impact of available treatments.
Who should consider this trial
Good fit: Ideal candidates for this study are pediatric patients under 18 years old with genetically confirmed 5q-SMA.
Not a fit: Patients with other types of SMA or those unable to provide informed consent may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of SMA and improve treatment strategies for affected children.
How similar studies have performed: Other studies focusing on the natural history and treatment outcomes of SMA have shown promise, indicating that this approach is supported by previous research.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Ability of the participant and/or his/her legally authorized representative (e.g., parent or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations. * Genetically confirmed 5q-SMA. * Age \< 18 years at enrollment. Key Exclusion Criteria: * Unable or unwilling to provide informed consent. * Other types of SMA (non 5q-SMA). NOTE: Other protocol defined inclusion/ exclusion criteria may apply.
Where this trial is running
Hefei, Anhui and 24 other locations
- Anhui Children's Hospital — Hefei, Anhui, China (Recruiting)
- Beijing Children's Hospital, Capital Medical University — Beijing, Beijing, China (Recruiting)
- Children's Hospital of Chongqing Medical University — Chongqing, Chongqing, China (Recruiting)
- Fujian Medical University Union Hospital — Fuzhou, Fujian, China (Recruiting)
- Xiamen Maternal and Child Health Hospital — Xiamen, Fujian, China (Recruiting)
- The First Affiliated Hospital, Sun Yat-sen University — Guangzhou, Guangdong, China (Recruiting)
- Shenzhen Children's Hospital — Shenzhen, Guangdong, China (Recruiting)
- Maternity and Child Health Care of Guangxi Zhuang Autonomous Region — Nanning, Guangxi, China (Recruiting)
- The Second Hospital of Hebei Medical University — Shijiazhuang, Hebei, China (Recruiting)
- The Third Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (Recruiting)
- Wuhan Children's Hospital, Tongji Medical College Huazhong University of Science & Technology — Wuhan, Hubei, China (Recruiting)
- Hunan Children's Hospital — Changsha, Hunan, China (Recruiting)
- Xiangya Hospital, Central South University — Changsha, Hunan, China (Recruiting)
- The Affiliated Hospital of Inner Mongolia Medical University — Hohhot, Inner Mongolia, China (Recruiting)
- Xuzhou Central Hospital — Xuzhou, Jiangsu, China (Recruiting)
- Jiangxi Provincial Children's Hospital — Nanchang, Jiangxi, China (Recruiting)
- The First Hospital of Jilin University — Changchun, Jilin, China (Recruiting)
- Children's Hospital Affiliated to Shandong University — Jinan, Jinan Shandong, China (Recruiting)
- Dalian Women and Children's Medical Group — Dalian, Liaoning, China (Recruiting)
- Shengjing Hospital of China Medical University — Shenyang, Liaoning, China (Recruiting)
- People's Hospital of Ningxia Hui Autonomous Region — YInchuan, Ningxia, China (Recruiting)
- Children's Hospital of Fudan University — Shanghai, Shanghai, China (Recruiting)
- The Second Affiliated Hospital of Xi'An Jiaotong University — Xi'an, Shanxi, China (Recruiting)
- West China Second University Hospital, Sichuan University — Chengdu, Sichuan, China (Recruiting)
- The Children's Hospital, Zhejiang University School of Medicine — Hangzhou, Zhejiang, China (Recruiting)
Study contacts
- Study coordinator: US Biogen Clinical Trial Center
- Email: clinicaltrials@biogen.com
- Phone: 866-633-4636
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.