Clinical Trials
232
25 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1892
Active, not recruiting
10
4.3%
Completed
129
55.6%
Enrolling By Invitation
8
3.5%
Not yet recruiting
7
3.0%
Recruiting
29
12.5%
Terminated
24
10.3%
Unknown
5
2.2%
Withdrawn
20
8.6%
No approval data available
- The FDA granted standard full approval to Ultragenyx's Fayuvi (rebisufligene etisparvovec-hopf) for pediatric mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome Type A. - Fayuvi is a one-time intravenous AAV9 gene therapy delivering a functional SGSH gene to restore the missing sulfamidase enzyme and reduce heparan sulfate accumulation. - Approval was supported by the pivotal Transpher A trial, in which treated patients scored 23.5 points higher on Bayley-III cognitive measures than an external natural history cohort (p<0.0001). - Ultragenyx expects commercial product to ship to Qualified Treatment Centers within 30-60 days, supported by its UltraCare program, and received a Priority Review Voucher.
- Oregon Health & Science University secured over $9 million in NIH funding to develop advanced microphysiologic models that recreate how cancers grow, spread and respond to treatment within bone and bone-associated tissues. - Two new projects focus on osteosarcoma, a rare bone cancer affecting children with unchanged survival rates for four decades, and prostate cancer bone metastasis affecting over 80% of advanced patients. - The research utilizes organs-on-chips technology, transparent USB-sized devices containing living human cells that allow real-time observation of cancer behavior at single-cell resolution. - Davies' team will test MCL-1 protein blocking combined with cyclophosphamide chemotherapy, which completely eliminated lung tumors in early lab studies, while Bertassoni's group examines how blood vessel forces and nerve signals influence cancer cell survival in bone.
- St. Jude researchers analyzed data from nearly 900 pediatric medulloblastoma patients across three clinical trials to develop new treatment risk groups that could safely reduce therapy intensity. - The study found that 40% of medulloblastoma patients can receive lower doses of craniospinal radiation therapy and almost all can receive less chemotherapy while maintaining equivalent survival outcomes. - Scientists created the Medulloblastoma Meta-Analysis Portal, a web-based tool that allows physicians to predict patient outcomes using demographic, clinical and molecular features through a point-and-click interface. - The research identified four actionable treatment recommendation groups based on molecular features including chromosome alterations, methylation subgroups and MYC oncogene amplifications in G3 and G4 tumor types.
- Researchers at St. Jude Children's Research Hospital have developed a breakthrough combination therapy using indisulam, a molecular glue drug, with anti-GD2 immunotherapy that achieved complete tumor eradication in preclinical neuroblastoma models. - The study, published in Nature Communications, reveals that neuroblastoma cells exhibit more complex plasticity than previously understood, with multidirectional state transitions that enable resistance to single-targeted therapies. - The combination approach exploits indisulam's dual mechanism of disrupting RNA splicing through RBM39 degradation while simultaneously activating natural killer cells and enhancing GD2 expression for improved immunotherapy effectiveness. - This therapeutic strategy addresses a critical unmet need in high-risk neuroblastoma, where nearly half of patients face aggressive treatments with substantial toxicity and relapse rates approaching 50%.
- UC Davis Health scientists led by Sanchita Bhatnagar have developed a novel gene therapy that reactivates silenced healthy genes in Rett syndrome by targeting microRNA-106a. - The therapy uses a DNA-based "sponge" molecule delivered via gene therapy vector to block miR-106a, allowing dormant healthy MECP2 genes to become active again. - In female mouse models of Rett syndrome, treated animals showed longer survival, improved movement and cognition, and significant improvements in breathing irregularities. - The approach could potentially treat other X-linked genetic conditions and represents a promising therapeutic strategy for a disorder that currently has no cure.
- New research reveals mothers who use cannabis during pregnancy are 3.5 times more likely to have children with disruptive behavioral disorders, including hostility and aggression. - Cannabis compounds can cross the placenta and enter breast milk, potentially affecting fetal brain development and leading to long-term cognitive and behavioral consequences. - The findings align with previous studies, including a 2024 report linking prenatal cannabis exposure to poorer thinking skills, impulse control issues, and attention problems in early childhood.
- A breakthrough gene therapy for spinal muscular atrophy (SMA), initially funded by $2 million in charity and government grants, became the world's most expensive drug at $2.125 million per dose after corporate acquisition. - The Gaynor family's charity Sophia's Cure provided crucial early funding for Zolgensma's development, only to be sidelined once pharmaceutical companies recognized its commercial potential. - Novartis has generated over $6.4 billion in revenue from Zolgensma sales, while access remains limited in many countries and families face insurance battles despite the therapy's life-saving potential.
