NCT05194397 Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
| NCT ID | NCT05194397 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Children's Hospital of Philadelphia |
| Condition | Lymphoblastic Leukemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 80 participants |
| Start Date | 2023-02-23 |
| Primary Completion | 2026-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 80 participants in total. It began in 2023-02-23 with a primary completion date of 2026-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
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, bone marrow failure syndrome, 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
Contact & Investigator
Sogol Mostoufi-Moab, MD, MSCE
PRINCIPAL INVESTIGATOR
Children's Hospital of Philadelphia
Frequently Asked Questions
Who can join the NCT05194397 clinical trial?
This trial is open to participants of all sexes, aged 10 Years or older, up to 30 Years, studying Lymphoblastic Leukemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05194397 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05194397 currently recruiting?
Yes, NCT05194397 is actively recruiting participants. Contact the research team at cambarerik@chop.edu for enrollment information.
Where is the NCT05194397 trial being conducted?
This trial is being conducted at Duarte, United States, Philadelphia, United States, Memphis, United States.
Who is sponsoring the NCT05194397 clinical trial?
NCT05194397 is sponsored by Children's Hospital of Philadelphia. The principal investigator is Sogol Mostoufi-Moab, MD, MSCE at Children's Hospital of Philadelphia. The trial plans to enroll 80 participants.
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