Nicotinamide Adenine Dinucleotide Augmentation to Prevent or Reverse the Progression of Alzheimer's Disease in People With Down Syndrome
Brigham and Women's Hospital
Summary
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).
Description
Specific Aims / Objectives Primary Aim To determine the safety and tolerability of multiple ascending doses of MIB-626 administered daily for 28 consecutive days in adults with DS by structured monitoring of adverse events and the measurement of safety laboratory tests including blood counts and chemistries, coagulation profile, and ECG. Secondary Aims 1. To study the PK of oral, multiple ascending doses of MIB-626 in adults with DS starting with the 500 mg daily dose and escalating the dose in ascending order to the maximum dose of 1000 mg twice daily or a maximal tolerated dose if that is…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. A diagnosis of full trisomy for chromosome 21 or complete unbalanced translocation of chromosome 21, confirmed by karyotype analysis or clinical documentation. 2. 18 years or older 3. Participant has a caregiver/ informant who has direct contact with the participant \>10 hours/ week and who can provide information about participant's health 4. Participant or Legal Authorized Representative is able to understand and willing to provide written informed consent and capable of completing study assessments. 5. In addition, female participants must Not be pregnant and not pla…
Interventions
- DrugMIB-626
MIB-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.
- DrugPlacebo
Matching placebo tablets (number and frequency of tablets will match the active intervention group at each of the three doses)
Locations (2)
- Massachusetts General HospitalBoston, Massachusetts
- Brigham and Women's HospitalBoston, Massachusetts