How Top Clinical Trial Enrollment is Advancing Pediatric Cystic Fibrosis Care

Can high clinical trial participation rates accelerate access and drive innovation in pediatric cystic fibrosis care?

In 2024, the Pediatric Cystic Fibrosis (CF) Therapeutics Development Center at Children’s Hospital Colorado, in partnership with National Jewish Health, enrolled more participants in clinical trials than any other center within the Cystic Fibrosis Therapeutics Development Network (TDN) — a national network of 80 top-tier research centers devoted to cystic fibrosis research.

The TDN, founded in 1998 and primarily funded by the Cystic Fibrosis Foundation, is one of the most competitive and trusted networks in rare-disease research. Only CF Foundation-accredited centers with proven research infrastructure, qualified personnel and high trial performance are selected to be a part of the network, and they must renew through a rigorous review every two years.

By centralizing trial design, protocol review, safety monitoring and statistical support, the TDN speeds the development and delivery of life-altering therapies for CF. Ranking first in enrollment within this network is more than just a data point — it reflects a culture rooted in trust and collaboration between families, clinicians and researchers.

“We are very optimistic about the future for children with CF,” says Edith Zemanick, MD, pediatric pulmonologist and director of the Therapeutics Development Center at Children’s Colorado. “High enrollment reflects commitment and shared urgency to move science and care forward.”

Why participant enrollment in clinical trials matters

Clinical trials are the foundation of every treatment advancement in cystic fibrosis. Before a therapy can reach the bedside — or change a child’s daily life — it must be tested across different age groups and disease stages. Leading the nation in enrollment means Children’s Colorado has a role to play in speeding up this process and making it more inclusive for others across the country.

“We cannot get from a lab discovery to an approved therapy without children and their families,” Dr. Zemanick says. “There’s no shortcut. And that’s why participation matters so much.”

Children’s Colorado and National Jewish Health have a long-standing partnership that brings together nationally ranked pediatric and adult CF programs. This integrated model ensures that patients can access trials at every age and stage of life, with consistent support from familiar providers.

Research isn’t just something that happens in a lab — it’s introduced as a natural part of the clinical experience. Families often first learn about trial opportunities from the same providers they’ve built trusted relationships with over years of care. That continuity makes a difference.

“Because our research team is embedded in our care team, families aren’t being approached by strangers; they’re being approached by people they know and trust,” Dr. Zemanick says. “That model of care builds confidence in the process and helps families see research as an extension of their child’s treatment, not something separate.”

This is a proactive, collaborative approach designed to ensure that the right patients can access the right studies at the right time. It’s also one of the reasons why Children’s Colorado consistently ranks among the top TDN sites in trial activation and participant enrollment.

A transformed landscape in cystic fibrosis research

In 2024, Children’s Colorado enrolled participants in a wide variety of studies, including those for all age ranges, for common and rare CF gene variants, and for therapies focused on everything from early treatment to quality-of-life improvements. Across all of them, high enrollment was essential not only for reaching study goals, but also for ensuring that tomorrow’s treatments reflect the full diversity of patients today.

The results of CF clinical research have been dramatic. Dr. Zemanick recalls that when she first started working in CF care and clinical trials, routine hospitalizations were the norm for pediatric patients with CF because there were no cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies — groundbreaking drugs that improve lung function and reduce disease burden for patients.

“Now we often go weeks without admitting a single CF patient,” Dr. Zemanick says. “That’s the power of research, and it started with families saying yes to participation.”

Children’s Colorado has contributed to landmark research advancing CF care in meaningful ways. One study looked at prescribing patterns for a highly effective triple-combination CFTR modulator: elexacaftor/tezacaftor/ivacaftor (ETI/Trikafta). Researchers found that compared to non-Hispanic white individuals with CF, patients from racial and ethnic minoritized groups were less likely to have a prescription for ETI, even when eligible, and faced longer delays in therapy initiation. This study recognized the need to address equity in prescribing practices for CF therapies.

Another noteworthy study that Children’s Colorado participated in examined how CFTR modulator therapies impact sweat chloride, the diagnostic benchmark for CF. The research found that while all modulator treatments reduced sweat chloride levels, responses varied widely by genetic variant and modulator. Patients treated with ETI, the most effective CFTR modulator to date, showed the most significant reductions in sweat chloride.

While treatments have been game-changing, the work isn’t finished. Some children can’t benefit from modulators due to rare genetic variants or other medical complexities. Others still manage time-consuming care regimens and long-term complications. That’s why ongoing enrollment in clinical trials is so critical.

For Dr. Zemanick, the momentum is promising. “What excites me the most,” she says, “is that we’re continuing to develop therapies that are more targeted, more effective and able to help even those with rare or currently untreatable genetic variants. We’re also focusing on quality-of-life outcomes and how lives are affected in ways that aren’t just about lung function.”

The Therapeutics Development Center isn’t slowing down. With every new study, the future of cystic fibrosis care expands one family, one patient and one trial at a time.