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

Children's Hospital of Philadelphia (CHOP) logo

Children's Hospital of Philadelphia (CHOP)

Status and phase

Enrolling
Phase 2

Conditions

Lymphoblastic Leukemia
Cancer Survivors
Myeloid Leukemia
Aplastic Anemia
Myelodysplastic Syndromes

Treatments

Other: Exercise Intervention
Dietary Supplement: Placebo
Dietary Supplement: Nicotinamide Riboside

Study type

Interventional

Funder types

Other
NIH

Identifiers

NCT05194397
1R01CA254955-01A1 (U.S. NIH Grant/Contract)
20-017320

Details and patient eligibility

About

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.

Full description

Adolescents and young adults (AYAs) who undergo hematopoietic cell transplantation (HCT) are at an especially high risk of developing sarcopenia (loss of skeletal muscle mass), which occurs earlier than would be expected from advancing age alone. This is important as not only it doubles their risk for non-relapse mortality, but it also leads to premature cardiovascular disease. The investigators propose a mechanistic randomized controlled trial of home-based remotely supervised exercise (aerobic and resistance) training and supplementation with nicotinamide riboside, a precursor of NAD+, a metabolite that is critical for muscle metabolism, in AYA survivors of HCT. The findings will lay the critical groundwork for future trials to prevent or mitigate the myriad downstream adverse effects of low muscle mass in AYA HCT survivors.

Enrollment

80 estimated patients

Sex

All

Ages

10 to 30 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  1. Males and females, ages 10-30 years at enrollment
  2. Able to understand and speak English
  3. Diagnosis of leukemia (myeloid, lymphoid), aplastic anemia, or myelodysplastic syndrome requiring allogeneic HCT
  4. 6-48 months from allogeneic HCT
  5. Females must have a negative urine/serum pregnancy test and must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.
  6. Subject's informed consent or parental/guardian permission (informed consent) and if appropriate, child assent
  7. Minimum weight of 24 kg

Exclusion criteria

  1. Known sensitivity to NR
  2. Concurrent use of any medications, including statins, likely to increase risk of NR toxicity
  3. Active malignancy, investigational agent(s) within 4 wks, or supraphysiological glucocorticoids at the baseline visit
  4. Currently meeting public health exercise guidelines
  5. Use of NAD+ precursors (supra-physiologic) within 4 weeks
  6. Hemoglobin < 10 g/dL
  7. Platelets < 50K
  8. Diabetes Mellitus requiring insulin or insulin secretagogue
  9. HbA1C ≥ 8% or fasting glucose > 125 mg/dL; However if the participant is diabetic, both fasting glucose level and HbA1C must meet the criteria to be deemed eligible.
  10. Kidney disease (eGFR < 60 ml/min/1.73 m2)
  11. Liver disease Alanine aminotransferase (ALT)/Aspartate aminotransferase (AST) (ALT/AST > 3 x ULN)
  12. Limitations in physical function preventing exercise testing/training
  13. Unstable angina or history of acute myocardial infarction (<5 days of planned study procedures)
  14. Recurrent syncope
  15. Symptomatic severe aortic stenosis
  16. Uncontrolled arrhythmia causing symptoms
  17. Pulmonary embolus <3 months of study procedures
  18. Thrombosis of lower extremities
  19. Symptomatic moderate or severe persistent asthma based on Forced Expiratory Volume (FEV) from pre-HCT pulmonary function testing
  20. Room air desaturation at rest ≤85%
  21. Females: Pregnant or planning pregnancy
  22. Non-cardiopulmonary disorders that may affect exercise performance or be aggravated by exercise (e.g. infection, renal failure, thyrotoxicosis, >moderate Graft versus host disease (GVHD) resulting in physical or functional impairment)
  23. Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.
  24. For MRI procedure: Contraindications to MRI. Individuals will be screened to ensure they can safely complete the MRI. *participants who cannot complete the MRI will not be excluded from participation in the remainder of the study procedures if they meet those inclusion and exclusion criteria.
  25. Current weight precludes safe completion of study procedures

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Factorial Assignment

Masking

Quadruple Blind

80 participants in 4 patient groups, including a placebo group

Nicotinamide Riboside (NR)
Experimental group
Description:
Investigators will use Good Manufacturing Process (GMP)-grade 300 mg capsules of the dietary supplement nicotinamide riboside (ChromaDex, Irvine CA). Investigators dose based on body weight, and monitor for adverse effects (AEs). For individuals with weight \> 72 kg: 900 mg po qd x 16 wks. For individuals with weight \> 48 kg and ≤ 72 kg: 600 mg po qd x 16 wks. For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 16 wks.
Treatment:
Dietary Supplement: Nicotinamide Riboside
Placebo
Placebo Comparator group
Description:
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). Doses of placebo will match the body weight schema for dosing of NR. Investigators dose based on body weight, and monitor for adverse effects (AEs). For individuals with weight \> 72 kg: 900 mg po qd x 16 wks. For individuals with weight \> 48 kg and ≤ 72 kg: 600 mg po qd x 16 wks. For individuals with weight 24 ≤ 48 kg: 300 mg po qd x 16 wks.
Treatment:
Dietary Supplement: Placebo
Exercise Intervention and NR
Experimental group
Description:
The exercise program consists of at-home training sessions including: aerobic and strengthening components designed to progress persons gradually to 150-300 minutes of the equivalent of moderate aerobic activity, and twice weekly strength training exercises. Participants in this arm will receive both the Exercise Intervention and the NR.
Treatment:
Dietary Supplement: Nicotinamide Riboside
Other: Exercise Intervention
Exercise Intervention and Placebo
Experimental group
Description:
The exercise program consists of at-home training sessions including: aerobic and strengthening components designed to progress persons gradually to 150-300 minutes of the equivalent of moderate aerobic activity, and twice weekly strength training exercises. Participants in this arm will receive both the Exercise Intervention and the Placebo
Treatment:
Other: Exercise Intervention
Dietary Supplement: Placebo

Trial contacts and locations

3

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Central trial contact

Katherine Cambareri, MPH; Kristin Wade

Data sourced from clinicaltrials.gov

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