This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors.
The primary outcome is the change in muscle strength (isometric knee extension) from baseline to 16 weeks. Key secondary outcomes are the change in muscle strength (ankle plantarflexion) from baseline to 16 weeks, the change in grip strength from baseline to 16 weeks, the change in lower extremity muscle mass from baseline to 16 weeks, the change in muscle OXPHOS capacity from baseline to 16 weeks, and the change in aerobic capacity (VO2 max) from baseline to 16 weeks.
Males and females, ages 10-30 years at enrollment
Able to understand and speak English
Diagnosis of leukemia (myeloid, lymphoid), aplastic anemia, bone marrow failure syndrome, or myelodysplastic syndrome requiring allogeneic HCT
6-48 months from allogeneic HCT
Known sensitivity to NR
Concurrent use of any medications, including statins, likely to increase risk of NR toxicity
Active malignancy, investigational agent(s) within 4 wks, or supraphysiological glucocorticoids at the baseline visit
Currently meeting public health exercise guidelines
Children's Hospital of Philadelphia
Lead sponsor
National Institutes of Health (NIH)
Collaborator
City of Hope Medical Center
Collaborator
St. Jude Children's Research Hospital
Collaborator
National Cancer Institute (NCI)
Collaborator
University of Pennsylvania
Collaborator