The aim of the study is to determine the safety and tolerability of different increasing doses of MIB-626 (a microcrystalline form of β nicotinamide mononucleotide; NMN) given daily for 28 consecutive days to adults with Down syndrome (DS). The study will also explore how the drug moves through the body over time (pharmacokinetics or PK) and how the drug affects the body in different ways (pharmacodynamics or PD). The study participants will be monitored for adverse events and measurements to detect safety events will take place including laboratory tests of blood counts, chemistries and coagulation profile, and electrocardiogram (ECG). The trial will enroll a total of 24 adults with DS who are 18 years or older and are medically stable. People enrolled in the study will be randomly assigned to receive either the active intervention (MIB-626) tablets, or placebo tablets for 28 days. Study participants will be followed for an additional 28 days (total 56 days of follow-up). This study will also explore the effect of MIB-626 on different measures associated with aging and risk of Alzheimer's Disease (AD).
A diagnosis of full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21, confirmed by karyotype analysis or clinical documentation.
18 years or older
Participant has a caregiver/ informant who has direct contact with the participant >10 hours/ week and who can provide information about participant's health
Participant or Legal Authorized Representative is able to understand and willing to provide written informed consent and capable of completing study assessments.
Fasting morning UACR > 5,000 mg/ g creatinine
Has AST or ALT > 3 times the upper limit of normal
eGFR < 30 mL/ min / 1.73 m2
Hematocrit < 0.34 or > 0.50 L/L
Parallel
MIB-626
DrugMIB-626 tablets will be provided at dose strength of 500mg. Three doses will be studied in ascending order: 500 mg (one tablet) once daily; 1000 mg (two 500 mg tablets once daily) or 1000 mg (two 500 mg tablets) twice daily.
Placebo
DrugMatching placebo tablets (number and frequency of tablets will match the active intervention group at each of the three doses)
Treatment emergent adverse events
All treatment emergent adverse events
Treatment emergent serious adverse events
All treatment emergent serious adverse events
Pharmacokinetics of NMN
Circulating levels of NMN
Pharmacodynamics - steady state concentration of NAD+ in the brain
steady stage concentration of NAD+ in the brain measured in situ using ultra-high field 7T magnetic resonance spectroscopy (MRS)
Pharmacodynamics - Blood NAD+ level
Blood NAD+ level
Pharmacodynamics - Plasma concentrations of NAD+ metabolites
Plasma concentrations of major NAD+ metabolites (nicotinamide, 1-methylnicotinamide, N-Methyl-2-pyridone-5-carboxamide \[2-PY\])
Muscle strength and endurance
muscle strength measured by the number of repetitions to failure with leg press exercise
Physical Function
Six-minute walk test, distance walked in 6 minutes
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Brigham and Women's Hospital
Lead sponsor
National Institutes of Health (NIH)
Collaborator
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