Theme: Clinical Trial: Basics
Very few families are prepared for the news that a child has a rare and/or life-threatening condition. You likely feel that you have been plunged into a bewildering, overwhelming new world. Fortunately, the evolving landscape of potential new therapies and associated clinical trials offers great hope. But, like all of the other topics that may require your attention, the prospect of a clinical trial comes along with the need to learn many new terms and concepts, some complicated decision-making, and the potential for disappointment and grief.
After reading this Guide, you will be able to:
- Approach the prospect of clinical trial with a better sense of what is involved
- Reflect on what is important to you and your family as you consider enrolling in a trial
Theme: Clinical Trial: Basics
INTRODUCCIÓN
El ensayo clínico es un término médico para un experimento controlado con el fin de investigar cuestiones acerca de una condición o una enfermedad. Es una investigación que explora si una estrategia médica, un tratamiento, o algún equipo es seguro y efectivo (tiene beneficios) para los seres humanos. El ensayo a veces se llama una investigación clínica o un estudio clínico. Para ver otra definición de un estudio o ensayo visite:
El vocabulario utilizado en los ensayos clínicos es básicamente consistente en los diferentes estudios, pero debería siempre hablar con sus proveedores médicos o los coordinadores
de investigación acerca de como cada término se relaciona con el estudio que están considerando para su hijo.
Es posible que usted encuentre términos que no se han incluido en esta lista. Para listas adicionales y definiciones visita:
Theme: Clinical Trial: Basics
Theme: Clinical Trial: Basics
Theme: Clinical Trial: Basics
In this video, a mother of two sons, Austin and Max with Duchenne Muscular Dystrophy and a son with a Primary Immune Deficiency talks about why we cannot ignore the economics of drug development and how individuals advocating to insurance companies can help ensure that they have accurate knowledge about your disease.
Theme: Clinical Trial: Basics
In this video, a Principal Investigator explains inclusion criteria and exclusion criteria. He explains who determines the criteria, defining both the role of the investigator and the sponsor. He acknowledges that sometimes individuals/families feel frustration with criteria and left out when they do not qualify for a trial.
Theme: Clinical Trial: Basics
Theme: Clinical Trial: Basics
This video is about Clinical Trials. A Study Coordinator explains that not all patients in a trial are treated at the same time; enrollment is staggered for a reason. She explains what the term cohort means and the process of dose escalation trials. She also talks about trials as a learning process.
Theme: Clinical Trial: Basics
In this video a patient advocate in biotechnology discusses a placebo arm in a clinical trial. She talks about questions to ask when considering a trial that includes a placebo arm including if the trial includes a rescue clause.
Theme: Clinical Trial: Basics
In this video, a Patient Advocate explains her role in the biotechnology industry.
Theme: Clinical Trial: Basics
In this video, a patient advocate in biotechnology talks about how the families who participate in research are pioneers and medical heroes. Patients’ willingness to take the risk and contribute is the only way forward research. “We (biotech) can build a rocket ship to take us to space, but we can’t fly it. We need a volunteer.”
Theme: Clinical Trial: Basics
In this video a Principal Investigator explains pathways outside of the standard clinical trial pathways; expansion studies, expanded access and compassionate use. He stresses that most often these are not considered until a Phase I trial is complete and safety is known. Compassionate Use is not a substitute for the clinical trial pathway, however he acknowledges circumstances when it might be employed.