Kick off a study of a drug to treat sickle cell disease and you’re facing a tough climb. Experimental drugs for sickle cell are few and far between, so there is little in the way of clinical trial infrastructure. The patient population is demographically challenging, too, made up largely of socioeconomically disadvantaged people who find it difficult to comply with the rigors of clinical drug trials.
That’s what made it so remarkable when we enrolled the first patient three weeks early in this Phase 1 open-label, dose-escalation trial of a drug to make hemoglobin more efficiently transport oxygen in sickle cell patients.
Recruiting for sickle cell trials is difficult not just because of demographics, but because to participate, patients must suspend participation in the only approved treatment regimen. So the sponsor was asking a lot when it imposed a tight timeline: less than four months from delivery of the final protocol to first patient in. Recognizing that subjects would be hard to locate, the sponsor budgeted 15 sites to achieve its target of 30 patients.