How YOU Can Speed Up the Clinical Trial Process!

CLINICAL TRIALS – The path to approval.

Typically we think about the Clinical Trial Process in the context of our kids.   We hear about Phase I trials.   Depending on the drug/biologic, many of these compounds will be required to be tested in healthy human subjects.   Some Phase I trials have inclusion criteria, suggesting they are looking for healthy adults of a certain age to take a single or multiple doses of a compound or biologic.   The purpose of the study is safety and is a first and critical step before moving to the relevant population.   Tests are done following dosing to understand pharmacokinetics and pharmacodynamics of the study drug.    Sometimes there is a hospital stay and in other circumstances, just an outpatient visit.

PARTICIPATION in clinical trials is not limited to our children! Often companies will talk about Phase I studies with healthy volunteers that are required before conducting a trial in Duchenne.  We sigh and think TIME.   TIME we do not have.   How much time for the Phase I?  How long will we have to wait?  And then we wait, imagining that ‘others’ such as medical students are readily available for these studies. I’m sure that is the case is some instances, but not all.

I decided to ask several companies about their Phase I trials (pending and in progress) and if we might help accelerate the process, thinking how important it might be for parents to participate, to take the first dose of a promising therapeutic, to learn about participation in clinical trials first hand, and to truly understand the burden we ask of our sons and daughters.

Are you willing to consider participation?  What would it involve? 


In some cases, the only way to get from Phase I (healthy volunteer) to Phase IIa, IIb, and III in Duchenne (or other diseases) is to recruit healthy adults.  If the sponsor is unable to recruit Phase I or if it recruitment is slow, we lose time.   Participation takes TIME – time off work, time away from our families.  On the other hand, participating in Phase I may save time for our sons.

Following a Phase I study, analysis is conducted to establish safety, to have baseline data about the administration, distribution, metabolism, and excretion of the drug, to be able to deliver analysis to the FDA/EMA in order to initiate Phase II studies.  The longer the Phase I takes to recruit, the longer to analyze and the longer it will take to test in Duchenne.   Healthy volunteers facilitate Phase I and accelerate the process for Phase II studies.


Sponsors pay for participation.   At minimum, they cover all expenses related to travel and cover all tests related to the study.  Some sponsors offer additional support for participation to incentivize and facilitate the recruitment process.

UNDERSTAND and APPRECIATE the burden of clinical trials.

Thankfully we have a robust pipeline of possibilities for our sons. We are anxious to see treatments and we have to consider that our participation as healthy volunteers is a critical piece, an opportunity, and may fill the gap, close the distance between Phase I trials and clinical trials for Duchenne.  Participation will likely increase our confidence about safety,  potentially add that one additional bit of confidence to steady your hand as you sign the informed consent for your son.  And perhaps even more importantly, you will walk into a clinical site with your son, eyes wide open about clinical trials and what it means to participate.

You will accelerate the possibility of treatments for your son and for every person with Duchenne.  YOU, as a healthy volunteer, have an opportunity to take that first BIG step.

For Phase I Studies visit .     


Examples of Current Phase I Trials, Duchenne Related

  • CAT- Catabasis 1004 COMPLETED
  • Pfizer PF-06252616   Currently  Recruiting




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