Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors

Summary

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.

Conditions

• Lymphoblastic Leukemia
• Cancer Survivors
• Aplastic Anemia
• Myelodysplastic Syndromes
• Myeloid Leukemia

Interventions

DIETARY_SUPPLEMENT

Nicotinamide Riboside

Investigators will use (Good Manufacturing Process) GMP-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA). NR is distributed by ChromaDex, Inc., Irvine, CA. NR is available as 300 mg capsules. The dietary supplement will be re-labeled by the Hospital of the University of Pennsylvania (HUP) Investigational Drug Service according to FDA regulations, including subject and physician name and NR or Placebo 300 mg capsules.

DIETARY_SUPPLEMENT

Placebo

The matched placebo will contain the same excipients without the active supplement and is generally recognized as safe. The placebo will be covered in an identical capsule (NR will be covered in the same capsule)

OTHER

Exercise Intervention

The exercise program consists of at-home training sessions and will include aerobic and strengthening components designed to progress persons gradually to 150-300 minutes of the equivalent of moderate aerobic activity, and twice weekly strengthening over 16 weeks. For aerobic training, the eventual intensity and duration goal (adapted for each person as necessary) will be for persons to train at 70-80% of peak 2MSPT heart rate 20-45 minutes 3-5 days/week. For resistance exercises the goal will be a load/weight that results in fatigue after 3 sets of 10-12 repetitions on 8-10 exercises 2 days/week.

Sponsors & Collaborators

• National Institutes of Health (NIH)

  collaborator NIH

• City of Hope Medical Center

  collaborator OTHER

• St. Jude Children's Research Hospital

  collaborator OTHER

• National Cancer Institute (NCI)

  collaborator NIH

• University of Pennsylvania

  collaborator OTHER

• Children's Hospital of Philadelphia

  lead OTHER

Principal Investigators

• Sogol Mostoufi-Moab, MD, MSCE · Children's Hospital of Philadelphia

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

FACTORIAL

Eligibility

Min Age

10 Years

Max Age

30 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-02-23

Primary Completion

2026-06-30

Completion

2026-06-30

Countries

• United States

Study Locations