Long-Term Follow-Up

What is long-term follow-up?

Long-term follow-up (LTFU) is a valuable part of a clinical trial where researchers continue to monitor participants after the main study period ends.

Why is long-term follow-up important?

Extended monitoring is important for tracking the safety of gene therapies over time, as well as their efficacy (how well the therapy works) and durability (how long the benefit lasts).

Types of long-term follow-up studies

Integrated

  • Protocol is part of the main clinical trial.
  • Participants build trust with the research team.
  • The informed consent process covers both the initial trial and LTFU.

 

 

Stand-alone Protocol

  • Separate protocol from the initial clinical trial and may have different study sites.
  • May include participants from multiple clinical trials.
  • Participants need to consent to the LTFU study in addition to completing the consent process for the initial trial.

 

How long is extended monitoring?

This period of time can be up to 15 years and depends on the type of gene therapy.

Can patients withdraw from LTFU?

Yes. LTFU is voluntary and participants can withdraw at any time. However, gene therapies make changes to a patient’s genome, so withdrawal from the intervention in the traditional sense is not possible.