Sheridan Clinical Research is participating in a multicentered, randomized, double-blind clinical trial using an Investigational probiotic for the prevention of necrotizing enterocolitis (NEC) in premature infants. The research is sponsored by Sigma-Tau Pharmaceuticals, Inc. Sheridan’s NICU Medical Director Mitchell Stern, MD, is the Principal Investigator for the Phase Ib/IIa trial being conducted at Plantation General Hospital in Plantation, Florida, to study the safety and efficacy of once-daily dosing of STP206 in premature very low birth weight (VLBW) and extremely low birth weight (ELBW) neonates to decrease the incidence of NEC.
NEC is the most common serious acquired disease of the gastrointestinal tract in preterm infants. The majority of NEC occurs in infants weighing less than 1500 g at birth. It affects the bowel of premature infants in the first few weeks of life and has been reported to occur in approximately 10 percent of VLBW infants, although the incidence varies among countries and neonatal centers. The mortality rate of VLBW infants with NEC is approximately 20 percent. In addition, infants with NEC often require surgical intervention, have an increased rate of complications related to total parenteral nutrition (TPN) and require extended hospitalizations. In a study by National Institute of Child Health and Human Development Network (NICHD) published in JAMA in 2004, researchers also found an increase in neurodevelopmental impairment rates among infants with NEC and sepsis.
A 2010 analysis of previous clinical trials, published in Evidence-Based Child Health, found that the use of probiotics, dietary supplements containing potentially beneficial bacteria or yeast, reduces the occurrence of NEC and death in VLBW premature infants, but that there was insufficient data on the benefits and potential adverse effects in ELBW neonates.
The four-year Phase Ib/IIa clinical trial is intended primarily to assess the safety and tolerability of once-daily dosing of two dose levels of an investigational probiotic versus control in four different birth weight strata in premature neonates. Plantation General Hospital’s NICU has historically been involved in multiple trials with Dr. Stern as the Principal Investigator. For the current probiotic trial, Dr. Stern has had the highest enrollment for cohort 3b, which closed in May 2016, and was one of the highest enrollers for newly closed cohort 4a.
As Jessy Tharakan, NNP, knows very well, obtaining informed consent for clinical trials in the NICU setting is particularly challenging, because the patient population is both delicate and vulnerable. As the authors of the 2014 Pediatrics Perspectives article “Honesty, Trust and Respect During Consent Discussions in Neonatal Clinical Trials” wrote, “Participation in neonatal clinical trials is often viewed as risky, ethically challenging, burdensome for parents, and a favor that altruistic families are performing for future generations of babies.” But they pointed out that the consent process also can empower and reassure families, as long as certain conditions are met. For example, the investigators and clinicians must believe that the research is both safe and important and must agree that participation in the proposed research would be meaningful and safe for each prospective participant. And the research team should strive for an open, trusting relationship with the parents and enable them to make an informed decision about what is best for their baby, rather than focusing on getting the consent form signed.
Kochuthresia (Jessy) Tharakan, NNP with Mitchell Stern, MD