Research clinicians at the Children’s Hospital of Philadelphia (CHOP) are enrolling 10 participants in a study to evaluate safety, tolerability, and effects on cardiac and skeletal muscle bioenergetics of compound MIB-626, which is an NAD+ precursor. This Phase 2 study will be enrolling 10 participants and is open label, meaning that all participants will be taking the compound.

NAD+, or nicotinamide adenine dinucleotide, has two general functions in the human body: helping to generate energy in mitochondria and working as a helper molecule for proteins that regulate other cellular functions.

The study investigators plan to complete enrollment by April 10, 2022.

Eligibility criteria include:

-  Genetically confirmed diagnosis of FA
-  Age 18-64 years old

-  Concurrent use of Vit B3 supplements
-  Cardiac ejection fraction less than 40%
-  Contraindication to MRI (e.g., spinal rods)
-  Involvement in another investigational drug study (must be >4 weeks out from another trial)

Participation is this study involves completion of two (2) in-person clinic visits, at the beginning and end of the 14-day study. At the clinic visits, you will be asked to complete blood testing and MRI scans.

If you are interested in learning more about this study, please contact Anna DeDio at Children’s Hospital of Philadelphia through email This email address is being protected from spambots. You need JavaScript enabled to view it. or by phone (267)-425-1998.

Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.