相关临床试验
161
24 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
1901
进行中（未招募）
11
6.8%
已完成
78
48.5%
No Longer Available
1
0.6%
尚未招募
13
8.1%
招募中
37
23.0%
暂停
1
0.6%
终止
8
5.0%
Unknown
6
3.7%
撤回
6
3.7%
暂无批准数据
- Charles River Laboratories has announced two strategic collaborations to advance oncology research, including an alliance with the Parker Institute for Cancer Immunotherapy and support for Children's Hospital Los Angeles Phase I clinical trials. - The partnership with PICI will provide network members access to Charles River's comprehensive preclinical drug discovery and development services, from early discovery through manufacturing. - Children's Hospital Los Angeles received a $6 million award from the California Institute for Regenerative Medicine in 2024 to develop innovative stem cell approaches for treating recurrent solid tumors in children and adolescents. - Charles River's cell and gene therapy CDMO capabilities will support streamlined manufacturing processes to accelerate the development of cancer treatments for pediatric patients.
- Children's Hospital Los Angeles researchers have become the first in the world to investigate using genetically engineered pig hearts as a "bridge" for infants awaiting heart transplants. - The groundbreaking preclinical study showed promising results with 8 of 14 young baboons surviving for months after receiving pig hearts, with one living for nearly 21 months. - This approach could particularly benefit infants with single-ventricle heart disease, who currently face only a 30% survival rate while waiting for transplants on mechanical support devices.
- The FDA has approved trofinetide (developed by ACADIA Pharmaceuticals) as the first-ever treatment for Rett syndrome, a rare genetic disorder primarily affecting females that causes severe developmental delays and neurological symptoms. - Trofinetide demonstrated efficacy in a Phase III trial involving 187 girls and young women aged 5-20 years, meeting both co-primary endpoints measuring core symptom improvement and global clinical assessment. - Researchers at six centers nationwide, including Children's Hospital Los Angeles, are now working to establish key EEG biomarkers for Rett syndrome to better evaluate future treatments, including potential gene therapies in development.
- Children's Hospital Los Angeles (CHLA) celebrates administering its 100th retinal gene therapy, Luxturna, since 2018 for Leber congenital amaurosis (LCA). - The gene therapy, Luxturna, targets mutations in the _RPE65_ gene, with CHLA utilizing in-house genomic support for precise patient screening. - Patients treated with Luxturna have shown significant vision improvements, sometimes within the first week, marking a transformative impact. - Future goals include early diagnosis and treatment of retinal diseases in childhood to ensure lifelong vision improvements through gene therapy.
- A new study at Children's Hospital Los Angeles reveals that magnetic resonance elastography (MRE) and transient elastography (TE) demonstrate insufficient accuracy in detecting high-grade liver fibrosis in pediatric MASLD patients. - Research findings show MRE performed marginally better than TE with AUROC values of 0.817 versus 0.750, though the difference was not statistically significant (P=0.4785). - The study, involving 77 pediatric patients with biopsy-proven MASLD, highlights the urgent need for more reliable non-invasive monitoring tools as MASLD affects up to 38% of children with obesity.
