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NAD Augmentation to Prevent or Reverse Alzheimer's Disease in People With Down Syndrome

Nicotinamide Adenine Dinucleotide Augmentation to Prevent or Reverse the Progression of Alzheimer's Disease in People With Down Syndrome

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 28 days, 12 weeks
  • Procedures mentioned: Heart tracing (ECG), Pills, MRI scans

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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)."
  • "People enrolled in the study will be randomly assigned to receive either the active intervention (MIB-626) tablets, or placebo tablets for 28 days."
  • "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."
  • "The investigators will determine whether oral MIB-626 increases the abundance of NAD+ in the brain, measured by ultra-high field 7T magnetic resonance spectroscopy (MRS)."
  • "Participants will be assigned to up to three dose cohorts and will receive MIB-626 or placebo for 28 days."
  • "Estimated Study Duration The study duration for each subject is anticipated to be up to about 12 weeks, which includes the screening, 4 weeks of intervention, and 28-day post-intervention follow-up visit after investigational product administration."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the safety of different doses of MIB-626 in medically stable adults with Down syndrome who are 18 or older. Participants take MIB-626 or placebo tablets (tablets with no study drug) daily for 28 days, then are followed for 28 more days. Researchers also study how the drug works in the body and its effects on signs linked to aging and Alzheimer's disease risk.

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).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have a confirmed diagnosis of Down syndrome from full trisomy 21 or a complete unbalanced translocation of chromosome 21—specific changes in chromosome 21.
  • Be 18 years or older.
  • Have a caregiver who spends more than 10 hours a week with you and can report on your health.
  • You or your legal representative must understand the study, agree in writing to join, and be able to complete study assessments.
  • Female participants must not be pregnant or plan to become pregnant in the next 6 months.

Who can't join

  • Certain abnormal tests: urine albumin-to-creatinine ratio (a urine protein test) above 5,000 mg/g; AST or ALT liver tests above 3 times normal; eGFR (kidney function) below 30; or hematocrit (the share of blood made up of red blood cells) below 34% or above 50%.
  • A major heart or blood vessel event in the past 3 months.
  • Taking part in another experimental drug or biologic treatment trial within the past 3 months, or within 5 drug half-lives, whichever is shorter. A half-life is the time for half a drug to leave the body.
  • A current alcohol or drug use disorder or dependence.
  • Major depression, bipolar disorder, schizophrenia, or current psychotic symptoms or behavior problems that could interfere with study tasks.
  • A short-term illness, including COVID-19, in the past month, or one that required a hospital stay in the past 3 months.
  • A past severe allergic reaction called anaphylaxis to a form of vitamin B3.
  • A body mass index (BMI, a measure based on height and weight) above 42.5, or being unable to walk.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 planning to become pregnant over the next 6 months. Exclusion Criteria 1. Fasting morning UACR \> 5,000 mg/ g creatinine 2. Other laboratory abnormalities: 1. Has AST or ALT \> 3 times the upper limit of normal 2. eGFR \< 30 mL/ min / 1.73 m2 3. Hematocrit \< 0.34 or \> 0.50 L/L 3. A major adverse cardiovascular event in preceding 3 months 4. Participation in an investigational trial to evaluate pharmaceuticals or biologics within the past 3 months or 5 half-lives, whichever is shorter 5. Current alcohol or substance use disorder or dependence (DSM 5 criteria). 6. Major depressive disorder, bipolar disorder, schizophrenia, or current psychotic symptoms or behavioral problems that could interfere with study procedures. 7. An acute illness, including COVID-19, requiring hospitalization within the past 3 months or any acute illness, including COVID-19, within the past month. 8. Has a history of anaphylaxis from vitamin B3 derivatives 9. BMI \> 42.5 kg/ m2 10. Non-ambulatory

Study sites (2)

MA