Clinical Trial Considerations

What is the APBD community thinking about clinical trials?

The Foundation is entering into a clinical trial phase. That is because our esteemed team of scientific advisers and researchers have successfully done the basic research and brought many of them to the drug testing stage. But there are other factors that go into a successful clinical trial that are not entirely under the purview of the scientists.

Note to the community: A member of the Foundation, a statistician, has written papers on various aspects of clinical trials. Other members may have other expertise to apply or simply have experiences/concerns worth sharing. With the FDA focused on patient-centric clinical trials, a dialogue between the APBD community and the researchers is in everyone’s interest.

 

Table of contents

(1) Endpoints or outcome measures

(2) Design/protocol

(3) Registry of patients (CAP and FAN)

(4) Statisticians (role in trials)

(5) Heterogeneity of the APBD condition

(6) Grant Support

 

Endpoints or outcome measures

 

Design/protocol

 

Registry of patients (CAP and FAN)

 

Statisticians

 

Heterogeneity of the APBD condition

 

(6) Grant Support