相关临床试验
60
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
N/A
进行中（未招募）
10
16.7%
已完成
20
33.3%
尚未招募
14
23.3%
招募中
15
25.0%
撤回
1
1.7%
暂无批准数据
- Australian researchers developed an AAV9-based gene therapy delivering a healthy ALPK3 gene that fully reversed established cardiomyopathy in adult mice and restored function in human cardiac organoids. - The therapy not only halted disease progression in newborn mice but also reversed existing heart damage, restoring contractile function and structure to near-normal levels. - In human stem cell-derived models, the treatment restored contraction strength and rhythm, and also improved muscle contractility in tissue carrying TTN mutations, the most common genetic cause of dilated cardiomyopathy. - The findings suggest ALPK3 gene therapy may treat a broader spectrum of inherited cardiomyopathies, potentially helping children avoid organ transplants.
- In Solomon Islands, two rounds of ivermectin-based mass drug administration reduced national scabies prevalence from 23.2% to 3.9%, an 83.3% relative risk reduction. - In Fiji, a single round of MDA nearly halved scabies prevalence from 6.2% to 3.4%, while impetigo rates dropped from 4.3% to 1.2% within 12 months. - The World Scabies Program, in partnership with ministries of health, delivered over 2.5 million ivermectin doses across both nations, demonstrating real-world feasibility of national scabies control. - Scabies prevalence remained above 10% among children under five in both countries, indicating a need for additional strategies to address residual disease burden.
- A study led by MCRI and the Luxembourg Centre for Systems Biomedicine identified nine new NAXD deficiency cases, broadening the known clinical spectrum to include cardiac and prenatal presentations. - Four children treated with high-dose niacin survived subsequent illnesses and infections that might otherwise have triggered severe deterioration or death. - Researchers conclude NAXD deficiency should now be considered a treatable disease, though optimal dosing, duration, long-term effectiveness, and safety remain unestablished. - Findings suggest genetic variant location may influence disease presentation, aiding earlier diagnosis across neurological, cardiac, and fetal anomaly contexts.
- A global analysis of over 106,000 pediatric infection samples across 82 countries found antibiotic resistance increased in every region between 2004 and 2022. - Gram-negative bacteria Acinetobacter baumannii and Klebsiella are driving resistance, with carbapenem resistance projected to reach 82% and 35% respectively by 2035. - Babies, children in intensive care, and those in low-resource countries are most affected, with resistance rising fastest to WHO-classified 'Watch' and 'Reserve' antibiotics. - Researchers launched the AMR in Kids platform providing country, pathogen, and antibiotic-class forecasts to 2035 to guide treatment decisions and public health planning.
- BridgeBio's PROPEL 3 trial met its primary endpoint, showing oral infigratinib significantly improved annualized height velocity by 1.74 cm/year compared to placebo in children with achondroplasia. - The study achieved the first statistically significant improvement in body proportionality for any achondroplasia therapy, with a meaningful reduction in upper-to-lower body ratio in children under 8 years. - Infigratinib demonstrated excellent safety with no drug-related discontinuations or serious adverse events, positioning it as the first oral FGFR3-targeted therapy for this genetic condition. - BridgeBio plans regulatory submissions in the second half of 2026 and will accelerate development for hypochondroplasia based on these breakthrough results.
- Researchers developed IgE-K, an experimental vaccine that provides year-long protection against anaphylaxis in mice by training the immune system to produce antibodies that block IgE, the key driver of allergic reactions. - The vaccine demonstrated remarkable efficacy in preventing deadly allergic reactions, with eight of nine unvaccinated control mice dying within 30 minutes of allergen exposure while all vaccinated mice survived with only mild symptoms. - Unlike current treatments like omalizumab that require injections every two weeks, the vaccine maintained high levels of protective antibodies for up to 52 weeks after immunization. - The vaccine showed no adverse effects and preserved the immune system's ability to fight parasites, addressing key safety concerns for potential human applications.
- PRT120 peanut oral immunotherapy achieved sustained remission in over 90% of children at 3-year follow-up, with patients safely consuming peanuts without daily dosing or moderate-to-severe reactions. - Children who achieved remission showed superior outcomes compared to those only desensitized, with no moderate or severe reactions requiring epinephrine among 53 remission patients versus 16.7% and 11.1% rates in desensitized participants. - The PPOIT-003 long-term study findings earned an EAACI Prize for outstanding research, highlighting remission as the optimal treatment goal over desensitization. - Prota Therapeutics plans to initiate a Phase 3 trial in the US next year, building on PRT120's differentiated short-term dosing regimen that delivered nearly 80% efficacy in the Phase 2b trial.
- Australian researchers at Murdoch Children's Research Institute and Doherty Clinical Trials Limited have launched a human challenge trial that deliberately infects healthy volunteers with Strep A bacteria to accelerate vaccine development. - The trial follows successful development of a safe human challenge model and aims to enable faster testing of vaccine candidates against a pathogen that kills over 500,000 people globally each year. - Separately, University of Queensland researchers in partnership with Moderna have demonstrated promising results using mRNA vaccine technology against Strep A in mouse models, showing strong immune responses. - The combined research efforts represent significant progress toward developing the first vaccine against Strep A, which could prevent devastating complications like rheumatic heart disease and invasive infections.
- Four out of five Americans are projected to be overweight or obese by 2050, with obesity rates increasing faster than overweight, according to a comprehensive study published in The Lancet. - The obesity epidemic is affecting younger populations at alarming rates, with teenage obesity more than doubling between 1990 and 2021, and people becoming obese at earlier ages than previous generations. - Researchers warn of a looming public health crisis as obesity triggers serious conditions including diabetes, heart disease, stroke, and cancer, with the highest rates concentrated in Southern states.
- Prota Therapeutics' PRT120 and PRT100 peanut oral immunotherapies demonstrate sustained benefits two years post-treatment, including improved quality of life. - A study showed participants achieving remission experienced fewer allergic reactions and no moderate or severe reactions, or need for epinephrine. - Remission, compared to desensitization alone, leads to substantial improvements in health-related quality of life for peanut allergy sufferers. - The PPOIT-003LT study validates remission as a superior outcome, reducing reaction severity and enhancing overall well-being for treated children.
