How to Nail Your Phase 1 Clinical Trial: Strategies for Success

By Jenna Levenson, PhD, RN, MS

How to Nail Your Phase 1 Clinical Trial: Strategies for Success
Because First-in-Human Should Not Mean First-in-Trouble

Phase 1 trials are where it all begins—and where so much can go wrong. From dosing errors to missed enrollment targets, a shaky start can derail years of preclinical effort. But with smart planning and strategic execution, Phase 1 can be your launchpad, rather than a landmine.

Here are proven strategies to set your Phase 1 trial up for success:

1. Start with Scalable Protocol Design

Avoid over-engineering your Phase 1. Remember, Phase 1 is about safety, tolerability, and pharmacokinetics. Build a protocol that is lean but flexible enough to accommodate real-world findings. Overly complex criteria or endpoints slow down enrollment and inflate costs. Consider using modeling and simulation from preclinical data to inform dose escalation design.

2. Choose the Right Sites—Not Just the Famous Ones (read that one again)

Well-known academic sites often have long queues. For Phase 1, speed is critical. Consider hybrid models with nimble early-phase units, or experienced community centers that have access to healthy volunteers and quick IRB turnaround.

3. Build in Real-Time Data Monitoring

Do not wait for end-of-cohort reports. I repeat, do not wait for end-of-cohort reports. Use eSource, eCRFs, and integrated data platforms to monitor trends in safety, lab values, and AE patterns in real-time.

Early detection = early intervention = trial stays on track.

4. Align with Regulatory Early and Often

A successful pre-IND meeting (if applicable) can save you six months of confusion. Engage with FDA or other authorities to confirm your toxicology package, CMC readiness, and starting dose rationale. Document everything and update your protocol based on feedback.

5. Invest in Participant Experience

Even in healthy volunteer studies, participant satisfaction matters. Clear communication, thoughtful scheduling, and quick reimbursement build trust and help retention in multiple ascending dose (MAD) cohorts.

6. Plan for What Comes Next

Keep in mind that a Phase 1 is not the finish line. Teams need to start mapping out your Phase 2 strategy now. Consider:

• Biomarker development

• Adaptive design options

• Early go/no-go signals based on PK/PD

This foresight helps you pivot fast—and keeps investors engaged.

A Phase 1 trial should be strategic, not just procedural. Design with foresight, staff with experience, and execute with precision. The success of your entire program depends on what happens in those first 30–60 days.

Are you gearing up for a Phase 1 trial? Let’s Talk. I have helped dozens of team’s de-risk early development and move faster with fewer surprises.