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Rare Disease CNS Study Delivers On-Time Results During Pandemic

Rare Disease CNS Study Delivers On-Time Results During Pandemic

Conducting rare disease clinical trials is always a challenge, particularly when it comes to recruitment.

Researchers look for patients who meet strict inclusion and exclusion criteria in addition to (of course) having the appropriate disease or condition. Participants are often very ill, very young, and immobile, requiring special considerations. Through contract research partners, sponsors can provide the right support to these populations, making studies more accessible to patients, parents, and families.

Precision for Medicine is an active rare disease and CNS research partner. The most recent example being a pivotal double-blind Phase III rare genetic neurological disorder program. It included multiple studies—beginning with an initial protocol—and was appended by long-term exposure studies and compassionate care.

Case Study: Success with a Rare Neurological Disease

For this study, the challenges of the indication were compounded by the initial spread of COVID-19 during recruitment. Despite this challenge, the team kept the trial on schedule to meet milestones, including database lock 10 business days after Last Patient Out.

Pivoting for the pandemic

A protocol amendment allowing remote assessments and virtual visits for patients were integral in countering COVID-19 lockdowns. Despite a three-month enrollment freeze at the onset of the pandemic, not a single target date was missed. Other adaptions included expanded use of Certified Mobile Research Nurses for each delivery to ensure IP integrity, white-glove courier service, and a secured SharePoint portal allowed sites to upload documentation to support remote monitoring – a program that still in place today.

Patient Recruitment Tools

To reach the right patients to meet enrollment goals, Precision collaborated with a patient advocacy group to raise awareness and ensure appropriate site selection. Indication-specialized pediatric neurologists served as principal investigators, providing valuable expertise and access to their own patient databases. Another 100+ interested patients were enrolled after expressing interest via the study website.

Patient and Caregiver Support

After consulting with the sponsor, participants and caregivers received full-service concierge services to retain and support the young patients and distressed families fighting this challenging neurological disease. Services included third-party transportation booking, as well as travel-related expense reimbursements for additional, unexpected costs, such as covering over-the-counter products to help manage adverse events (AEs).

  • Case Study

    Case Study: An Advanced Approach to Integrated Summary of Safety Studies



Moving Research Forward

The availability of neurological treatments is predicated on the success of clinical trials. Through a targeted and collaborative approach, Precision accelerated the development of a novel therapy to help combat a rare genetic condition.

Choosing a CRO with end-to-end service capabilities and the agility to pivot as the healthcare landscape evolves is critical to success when working to bring life-changing therapeutics to market.

Explore Precision’s ‘Rarefied Thinking’ approach to rare research >