Project Summation
The purpose of the Down syndrome (DS) project is to enhance clinical trial support for medication usage in children with DS and to promote training for clinical trials in those with DS for PTN and DS clinicians. Specifically, the PTN DS will (1) Characterize the pharmacokinetics (PK), pharmacodynamics (PD), and pharmacogenomics (PGx) of understudied off-patent drugs administered to children and young adults with DS. (2) Develop a stand-alone protocol specifically for those with DS after convening thought leaders and experts. (3) Develop a training program for clinical researchers, both within the PTN and with external DS experts, to provide insight and guidance in trial design, recruitment, and engagement (including assent/consent) issues specifically in this population.
2020 November Meeting
This half-day virtual meeting focuses on presentations and discussions about therapeutic challenges in treating children with DS; community advocacy and partnering with communities to engage patients with DS in clinical trials; parent/family engagement in current and future research; lessons learned from current efforts to enroll patients with Down syndrome into an active PTN study; and input on a prospective randomize clinical trial the PTN is interested in conducting.
LMS Credit
All targeted audience members are eligible to receive Joint Accreditation credits for this activity. No attendance certificates are issued for this course. See the PTN Down Syndrome 2020 Virtual Meeting Enduring Syllabus pdf for CME credit statements.
Perspectives
The following testimonials and videos help illustrate the importance of the INCLUDE initiative, from a variety of perspectives:
PDFs and Publications
Enrolling Participants into Two Drugs of Interest in POP02
Assenting Children with Down syndrome - Best Practices
Recruitment Successes for the POP 02 Down syndrome Guanfacine Cohort
Dr. Maria Stanley: Connecting Parents with the Best Informaiton for Children with Down syndrome
Dr. Brian Skotko: Collaboration Leads to Better Outcomes for Children with Down syndrome
Michelle Pfeiffer: Researching Unanswered Questions for Children with Down syndrome
Conducting clinical trials in persons with Down syndrome: summary from the NIH INCLUDE Down syndrome clinical trials readiness working group (Journal of Neurodevelopmental Disorders)
Video
Enrolling Participants into Two Drugs of Interest in POP02
Assenting Children with Down syndrome - Best Practices
Recruitment Successes for the POP 02 Down syndrome Guanfacine Cohort
Building Evidence to Improve Care for People with Down syndrome
Collaboration Leads to Better Outcomes for Children with Down syndrome
Researching Unanswered Questions for Children with Down syndrome