Theme: Clinical Trial: Qualifying

Patient advocacy organizations are united in the common goal of improving the lives of those living with the rare diseases they represent. For some in the community, the science has advanced and treatments are available. For others, a treatment is still off on the horizon. In this episode, CPN’s Jennifer Siedman talks with Leslie Urdanta, Family Support Coordinator for the National MPS Society, a community for individuals and families living with MPS and ML about how the Society supports its families through clinical trials – managing expectations, tempering frustrations and keeping the community united.

Theme: Clinical Trial: Qualifying

In this video, the mother of two boys with Duchenne Muscular Dystrophy talks about putting a child in a dose escalating study. She explains the difficult decision of participating in a Phase 1 gene therapy trial at the lowest dose because currently you cannot be re-dosed. She briefly discusses placebo-controlled trials.

Theme: Clinical Trial: Qualifying

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks honestly about how often in rare disease it feels like there are “haves” and “have-nots” in the community. She talks about both the division trials can create in a community but also the support you can gain.

Theme: Clinical Trial: Qualifying

In this video, a mother of two sons with Duchenne Muscular Dystrophy talks about finding out about a trial on Facebook, screening, and the whys of trial design.  She speaks about why it is important to enroll the right candidates in trial. She also discusses the misconception that you can “train” your child to meet the inclusion criteria, in this case a walk test.

Theme: Clinical Trial: Qualifying

In this video, a mom talks about prioritizing her children’s physical and speech therapies in the hopes that it would increase their performance in the screening process so they would meet the eligibility criteria. She talks about the importance of accurately representing your child so as not to risk jeopardizing the trial.

Theme: Clinical Trial: Qualifying

In this video, a mother discusses having two children with a metabolic disorder. Her youngest screened for a gene therapy trial but it was found he possesses the antibodies to the vector and could not participate. Her older daughter was able to participate. She never had to choose a child because the antibodies made the decision for her.

Theme: Clinical Trial: Qualifying

In this video, a mother of two children with Sanfilippo Syndrome discusses the screening process, explains that her younger child, a son, met the inclusion criteria but then screened positive for antibodies to the vector and was excluded. She discusses isolating her other child so as not to risk her exposure to the virus and developing the antibodies during her screening.

Theme: Clinical Trial: Qualifying

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: Qualifying

This video is about Clinical Trials: Recruitment and Pre-Screening. A Study Coordinator explains the process of recruiting participants for a clinical trial and having an informal pre-screening or informational call with a Study Coordinator to understand the inclusion criteria, commitments necessary for participation, and the medical history of the potential participant.

Theme: Clinical Trial: Qualifying

Theme: Clinical Trial: Qualifying

In this video, a patient advocate in biotechnology discusses inclusion criteria and why finding appropriate candidates for a trial is important to a safe, successful trial and how it often results in a more rapid acceleration of approved treatments. She talks about the heartbreaking reality that in choosing candidates the focus has to be on the greater good of the entire community over the individual.

Theme: Clinical Trial: Qualifying

In this video, a patient advocate in biotechnology discusses how patient advocates can help families feel excited and hopeful about clinical research while at the same time balancing realistic expectations. Clinical research is a process, and even if the trial is considered unsuccessful or the outcome is not what was hoped for, something is always learned.