Welcome to our Clinical Trials Information Hub
Here, we aim to demystify the process of clinical trials and explain how they can offer hope, especially for those with rare conditions like pediatric brain tumors.
Why Clinical Trials?
Pediatric brain tumors are often very rare, and for some of these tumors, there may be no effective standard treatments available. If initial treatments don’t work, options can be limited. Clinical trials offer a way to explore new and experimental therapies that might provide hope where traditional treatments fall short. They test cutting-edge treatments that are not yet widely available but may offer new hope. Participating in these trials can give patients access to innovative therapies and contribute to advancing medical knowledge for future patients.
Clinical trials are accessible to everyone, but patients and families cannot always rely on their doctors to be aware of all available options. To help navigate this, resources like ClinicalTrials.gov provide information on ongoing trials, and nurse navigators like Colleen Sanders at Weill Cornell Medicine, or those at the Brain Tumor Network, offer free assistance to help families explore and understand their options.
Accessible to All
Clinical trials are open to everyone who meets the criteria for participation. Trials are designed with the highest ethical standards, and participant safety is always a top priority. Diverse patient populations are encouraged to participate to ensure that new therapies are offered to all those in need of treatment options.
For individuals with rare diagnoses, such as pediatric brain tumors, clinical trials can be a beacon of hope. These conditions often have limited treatment options, and clinical trials offer access to cutting-edge therapies that may not yet be available through standard treatment routes. Participation in these trials not only contributes to the advancement of medical science but can also provide new opportunities for managing or potentially curing challenging diseases.
If you or a loved one is considering participating in a clinical trial, we are here to support you every step of the way. Feel free to reach out with any questions or concerns. Together, we can advance the frontiers of medicine and improve outcomes for patients with rare conditions.
PHASE ONE – SAFETY
In Phase 1 trials, the primary goal is to assess the safety of a new treatment. Researchers evaluate how the treatment is absorbed, metabolized, and excreted by the body, and identify any potential side effects. Phase 1 trials help determine the appropriate dosage and treatment schedule, laying the groundwork for further testing.
PHASE TWO – EFFICACY
Phase 2 trials expand on the findings from Phase 1 by focusing on the treatment’s effectiveness. Researchers continue to monitor safety but are primarily interested in determining whether the new treatment works as intended for the specific condition it is designed to treat. Phase 2 trials provide critical data on how well the treatment performs.
PHASE THREE – VALIDATION
Phase 3 trials involve a much larger group of participants. The primary aim is to confirm the treatment’s effectiveness and monitor any long-term side effects. These trials compare the new treatment against the current standard treatment or a placebo to determine if it offers significant benefits.
“When my son was in treatment, I was told his diagnosis was terminal and that there were no treatment options available. No one told me that I could explore clinical trials. I would have done anything just for the opportunity to try something, anything that may have helped.”