Jessi: Coordinating Research on New Treatment for Alagille Syndrome, a Rare Childhood Liver Disorder

Jessi served as a coordinator for NIDDK-supported studies of the
Childhood Liver Disease Research Network; photo courtesy of
Children’s Hospital of Philadelphia

Clinical research studies are truly a team effort, relying on the contributions of study participants, study coordinators, doctors, nurses, medical facilities, patient support organizations, and sponsors like NIDDK, other NIH collaborators, and partners in the pharmaceutical industry. Some of the essential, unsung heroes of this team effort are the study coordinators, also called research coordinators, who facilitate the participation of children and their families while ensuring the highest-quality science.

Jessi, 43, a native and current resident of Philadelphia with a master’s degree in public health, has been involved in clinical research at the Children’s Hospital of Philadelphia, or CHOP, throughout her entire career. When not busy with her work, she enjoys staying active—biking, running, doing yoga, and spending time with her husband and two daughters, including having picnics in the city’s many parks and taking trips outside the area for fruit picking.

Currently, Jessi serves as a Senior Director of Research, providing strategic day-to-day support to CHOP’s Chief Scientific Officer. But a large part of her formative career development was spent as a study coordinator, and later coordinator supervisor, working with her site from the ground up to build their participation in the Childhood Liver Disease Research Network (ChiLDReN), and its ITCH and IMAGINE II studies of Alagille syndrome treatment, testing a new drug to relieve the pruritus (severe itching) associated with the disease.

Jessi’s motivation for being involved in clinical research studies is clear: “We’re really concerned about the lives of these children and ways to improve them—that’s such a gratifying mission to stand behind.”

“When I started my career at CHOP 20 years ago, it was as a research coordinator,” recalls Jessi. She began work at the CHOP’s Fred and Suzanne Biesecker Pediatric Liver Center on a study collecting information on children with cholestatic liver diseases, where she had her first interactions with children with Alagille syndrome and their families. The Center attracted many of these families due to the renown of its investigators who helped to identify some of the key genetic variants associated with Alagille syndrome. Jessi observes, “At CHOP, it doesn’t feel like it’s such a rare disease. Our site has been a key center for patients with Alagille syndrome all around the world.”

Based on her experience, Jessi was tapped to be the first study coordinator for her site’s team within NIDDK’s multi-center Biliary Atresia Research Consortium or “BARC.” The predecessor of the modern-day ChiLDReN Network, BARC later merged with another NIDDK consortium called the Cholestatic Liver Disease Consortium or “CLiC.” “Everyone likes to joke, ‘BARC and CLiC got married and then they had ChiLDReN,’” quips Jessi.

As part of the research team, Jessi worked on a number of NIDDK-sponsored BARC, CLiC, and ChiLDReN studies of children with liver diseases due to cholestasis (or limited bile flow from the liver), including Alagille syndrome. An early, ongoing study in which CHOP participated was on the progression of liver diseases in children over time, including rare cholestatic liver diseases such as Alagille syndrome. Later, Jessi served as the study coordinator for the ITCH interventional trial testing the new drug maralixibat as a treatment for pruritus in children with Alagille syndrome. ITCH presented some new challenges through its design as an NIDDKsupported partnership with the pharmaceutical industry. “Although this was an NIDDK-funded study, it had a lot of the complexity and organization that a traditionally industry-sponsored study may have in terms of the interaction and coordination with multiple units and parts,” notes Jessi.

From early on, families with children affected by Alagille syndrome expressed interest in joining the study. “We had families who were very eager when they heard about this as a possible study because the pruritus that these families experience can really be so debilitating and compromise quality of life and day-to-day activities,” says Jessi. She describes the struggles these families go through, trying one drug after another that only provides limited or waning relief. “For many of them, they’ve gone through all that we have available to offer, so there was a lot of excitement in the patient community to know that there were potentially new treatments available to treat the itching.”

In addition to coordinating study conduct and data collection among the CHOP team, NIDDK, data coordinating center, and industry partner, Jessi and the other study coordinators also helped facilitate other day-to-day tasks in running the study. These included screening potential participants and attending each of their study visits—about 10 visits per year over the multiple years of the study for each of the two families who participated at the CHOP site in ITCH, and later in IMAGINE II. In between study visits, she assisted with collecting data entered by parents into electronic diaries and scheduling the families’ upcoming visits. Coordinators also attended meetings of the Steering Committees overseeing the studies and helped address practical concerns. “One of the interesting things about the ChiLDReN Network is the way it involves everyone in effect, not only the participants and our PI [principal investigator] leaders, but that there is a real emphasis on involving the coordinators,” she observes. As Jessi grew more experienced, she transitioned from her role as a coordinator for ITCH to one managing a group of coordinators for the follow-on study of IMAGINE II.

“The research coordinator for ITCH and IMAGINE is maybe like the conductor of an orchestra,” says Jessi, who has served as a coordinator for studies of the Childhood Liver Disease Research Network.

“The research coordinator for ITCH and IMAGINE is maybe like the conductor of an orchestra,” says Jessi. The work of the “conductor” is multi-dimensional, seamlessly blending the efforts of three instrumental functions—interactions with patients and their families, fulfilling regulatory requirements at the institutional level, and performing administrative duties that interact with the data coordinating center and industry partner. “The research coordinator is really at the nexus of the family participation in the study, coordinating everything on their behalf, while at the same time, adhering to the protocol.… It’s a fine dance,” she says.

The coordinator provides a high level of “concierge-type service,” says Jessi, to reduce participants’ burden, while still allowing the study to collect the highest-quality data. For example, CHOP study coordinators accommodated travel plans and provided translation services to overcome language barriers for one participating family, and helped another participant family who had a newborn child by being flexible with appointment scheduling and mailing treatments. “We’re really grateful for the participation of families in these studies,” Jessi says. “That is one of the high-value components of serving in this role … the relationship that is established with these research participants.… that’s probably one of the things that those of us in clinical research at the coordinator level can find so valuable and rewarding is that we are directly having an impact on care.”

“The relationship that is established with these research participants … that’s probably one of the things that those of us in clinical research at the coordinator level can find so valuable and rewarding,” remarks Jessi, a coordinator for groundbreaking studies testing a new treatment for the rare pediatric liver disease of Alagille syndrome.

When asked about whether others should consider getting involved in clinical research, her answer is a “resounding yes.” She adds that “there’s a real value in facilitating or participating in research … from a participant perspective, from a physician perspective, and also from the perspective of the non-physician research team members.” Jessi’s motivation for being involved in clinical research is clear: “We’re really concerned about the lives of these children and ways to improve them—that’s such a gratifying mission to stand behind.”

Jessi’s commitment to caring for children and families affected by disease is also evident in her choice of activities outside of CHOP. She has been involved with the local chapter of the American Liver Foundation and with the Alagille Syndrome Alliance, serving on committees and planning patient education events.

In her new role supporting CHOP’s Chief Scientific Officer, she has the opportunity to make an even broader impact on a wide range of childhood diseases. But Jessi has not forgotten her roots as an experienced study coordinator and draws upon them often to inform her current work. “There are many days that I do miss that opportunity to have that interface at the patient or clinic level,” Jessi says. Still, she feels that her 20 years worth of experience with the practical aspects of clinical research at CHOP, largely as part of the CHiLDReN Network studies, is an integral part of the value she brings to her current role.

Looking back on Jessi’s impressive performance as an “orchestra conductor,” coordinating this groundbreaking research while optimizing participants’ study experiences, one can only say “Bravo!”