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) Overview of APBD Statistical Research

(2) Endpoints or outcome measures

(3) Design/protocol

(4) Registry of patients (CAP and FAN)

(5) Statisticians (role in trials)

(6) Heterogeneity of the APBD condition

(7) APBD Prevalence

(8) Cures Act

(9) What did we learn from the APBD Phase 1 trial using Triheptanoin oil

 

Overview of APBD Statistical Research

Endpoints or outcome measures

Design/protocol

Registry of patients (CAP and FAN)

Statisticians

Heterogeneity of the APBD condition

Grant Support

Cures Act

What did we learn from the APBD Phase 1 trial using Triheptanoin oil

This was an APBD Phase 1 trial with the Triheptanoin oil.  It tested the use of the oil and a low-sugar diet on five APBD patients for a full year. It showed the oil was safe to use and provided meaningful functional improvement for the first six months. However, the trial patients reported a leveling off of functional improvement and an eventual decline. Some patients were advised to add over the counter PhosChol and Sam-e, and it seemed to slow their decline.