Clinical Research: Phase 1 - Phase 4

Against the Odds: Achieving Fast Startup in a Sickle Cell Drug Trial

Even when things go pretty well, launching a clinical trial is a game of long odds. And when the deck is stacked against you the way it was when we set out to study a drug for sickle cell disease, the difficulties can add up fast.

So even we were surprised by the success of this Phase I open-label, dose-escalation trial of a drug to mitigate the disease’s severity and complications by making hemoglobin more efficiently transport oxygen in sickle cell patients. Key among the obstacles we faced:

  • There are few experimental drugs for sickle cell, and consequently, the clinical trial infrastructure is minimal.
  • Patients tend to be socioeconomically disadvantaged people who may have trouble even getting to the trial sites, much less complying with the rigorous requirements of clinical drug research.
  • To participate in this trial, subjects had to stop using the only approved treatment regimen and take a chance on the unknown.

Recognizing that recruiting would be tough, the sponsor budgeted 15 sites to achieve its target of 30 patients. But it also imposed an aggressive timeline — less than four months from delivery of the final protocol to first patient in. We targeted one fast-start site for the initial site initiation visit, and its staff worked with us to quickly prescreen potential subjects.

But even with that quick and promising start, we were surprised to enroll the first patient three weeks early.

Focus on quick start-up
To beat that ambitious goal, we applied an unusual level of discipline. First, we picked a site that we knew could accommodate our need for fast start-up with an accelerated contracting process and eagerness to prescreen potential patients. We held weekly meetings between the principal investigator and the project team. That’s an unusual step so early in a Phase I trial, and it was indispensable in helping us complete contract negotiations, implement regulatory processes, appoint an institutional review board, assemble lab kits, and put the investigational product in place.

Fifteen sites are now operating, and the trial is progressing rapidly on the strength of an enthusiastic sponsor and a highly engaged project team.