Phase 2
N=1
Nicotinamide Riboside on Mitochondrial Function in Li-Fraumeni Syndrome
Cancer · Skin Fibroblasts · Muscle Weakness
Bottom Line
View on ClinicalTrials.gov: NCT03789175 ↗Enrolled (actual)
1
Serious AEs
0.0%
Results posted
Dec 2020
Primary outcome: Primary: Change in PCr Recovery Tc Measurement From Baseline to 12 Week NR Supplementation Using the 31P-MRS Skeletal Muscle Submaximal Exercise. — 60; 73 Seconds
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Nicotinamide Riboside (NR) (Dietary_supplement)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Primary completion
- Nov 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in PCr Recovery Tc Measurement From Baseline to 12 Week NR Supplementation Using the 31P-MRS Skeletal Muscle Submaximal Exercise. |
60; 73 | — |
| SECONDARY Change in Time of CPET Time as a Measure of Exercise Tolerance From Baseline to 12 Weeks of NR Supplementation. |
12.1; 11.68 | — |
Summary
Background:
Nicotinamide riboside (NR) is a vitamin B3 dietary supplement. It may help improve muscle function, that may in turn may improve a person s exercise capacity. Researchers want to study how skeletal muscle responds to NR in an individual who has Li-Fraumeni Syndrome and slow muscle energy recovery after exercise.
Objective:
To study how nicotinamide riboside affects skeletal muscle after exercise.
Eligibility:
One person at least 18 years old with Li-Fraumeni syndrome and a certain gene mutation
Design:
The participant will be screened with a medical history, physical exam, and blood and urine tests.
The participant may also have a heart test.
The participant will maintain their regular diet and supplements during the study.
The participant will take the study drug as 1-4 tablets twice a day for 12 weeks. The participant may be contacted with reminders and questions about side effects.
The participant will have 4-5 visits over 18-30 weeks. At visits, the participant will repeat screening tests. At some visits they will also have:
* Ultrasound of the heart with a wand placed on the chest.
* Test of oxygen used at rest and exercise, while wearing a face mask.
* Exercise test on a treadmill or bicycle with electrodes on the skin.
* Magnetic resonance spectroscopy. The participant will have no caffeine for 12 hours. Then they will lie in a machine for about 2 hours. Sometimes they will lie still. Sometimes they will be asked to move.
Health questionnaire
The participant may have a skin sample taken by needle.
The participant will be withdrawn from the study if they become pregnant.
Eligibility Criteria
- INCLUSION CRITERIA:
- At least 18-years of age and able to give informed consent
- Have delayed phosphocreatine (PCr) recovery time constant >45 sec by 31P-magnetic resonance spectroscopy (MRS) testing and a history of fatigue symptoms
- Ability to undergo study procedures, including scheduled visits, blood draws and skeletal muscle exercise Nuclear Magnetic Resonance (NMR)
- Have Li-Fraumeni syndrome and confirmed TP53 mutation by genetic testing
- Committed to using reliable contraception which may include abstinence during study participation
- Female participants of child-bearing ability and potential willing to commit to reliable contraception while participating in the study
EXCLUSION CRITERIA
- Current systemic treatment for cancer
- Unable to perform required study visits or procedures
- Magnetic resonance imaging (MRI) incompatible hardware
- Pregnant or breastfeeding women
- History of intolerance to NR precursor compounds, including niacin or nicotinamide
Data sourced from ClinicalTrials.gov (NCT03789175). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.