MIB-626 for Alzheimer's Disease in Down Syndrome

Not yet recruiting at 1 trial location

Overseen By Nancy Latham, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Brigham and Women's Hospital

Disqualifiers: Cardiovascular event, Substance use, Major depression, others

Compensation: Varies

Number of Visits: 2

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety of a new drug, MIB-626, for adults with Down syndrome to assess its potential benefits for Alzheimer's disease. Researchers aim to determine if MIB-626 is safe and how it affects the body when taken daily for 28 days. Participants will receive either MIB-626 pills or placebo pills without active ingredients. Adults with Down syndrome who are medically stable and have a caregiver to assist with the trial may be suitable candidates for this study. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Is there any evidence suggesting that MIB-626 is likely to be safe for humans?

Research has shown that MIB-626 is safe for humans. In one study, taking 1 gram of MIB-626 twice a day was safe for healthy adults and significantly increased their blood levels of NAD, a molecule important for energy and cell repair. Another study found that MIB-626 was safe and effective in raising blood NAD+ levels in adults with COVID-19.

While these studies indicate that MIB-626 is generally well-tolerated, the current trial is in its early stages and focuses on safety in adults with Down syndrome. Although the treatment is likely safe, the main goal is to confirm its safety for this specific group. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike standard treatments for Alzheimer's in Down syndrome, which often focus on managing symptoms, MIB-626 is unique because it targets cellular energy production. Researchers are excited about MIB-626 because it enhances mitochondria, the powerhouses of cells, potentially slowing disease progression rather than just alleviating symptoms. By improving mitochondrial function, MIB-626 aims to address the underlying causes of cognitive decline, offering a new approach that could change the way Alzheimer's is treated in individuals with Down syndrome.

What evidence suggests that MIB-626 might be an effective treatment for Alzheimer's Disease in Down Syndrome?

Research shows that MIB-626 can increase levels of NAD, a molecule important for energy and cell repair, in the blood. Studies have found that taking MIB-626 significantly boosts NAD levels in just a few weeks. This increase in NAD may improve brain function and could slow aging effects, including conditions like Alzheimer's Disease. In this trial, participants will receive either MIB-626 or a placebo to evaluate its effectiveness in treating Alzheimer's in people with Down syndrome. Although earlier trials showed MIB-626 to be safe, its effectiveness in this specific population is still under investigation. ^{1 2 3 4 6}

Who Is on the Research Team?

Shalender K Bhasin, MB, BS

Principal Investigator

Brigham and Women's Hosptial

Are You a Good Fit for This Trial?

Adults with Down syndrome who are 18 years or older and medically stable can participate in this trial. They will be tested for their ability to tolerate a new drug, MIB-626, over 28 days. People with certain health conditions that could interfere with the study or those taking conflicting medications may not qualify.

Inclusion Criteria

My condition involves an extra chromosome 21 or a related genetic change.

I am 18 years old or older.

I have someone who helps me for more than 10 hours a week and can talk about my health.

Exclusion Criteria

Fasting morning UACR > 5,000 mg/g creatinine

AST or ALT > 3 times the upper limit of normal

Hematocrit < 0.34 or > 0.50 L/L

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MIB-626 or placebo tablets daily for 28 consecutive days to assess safety, tolerability, pharmacokinetics, and pharmacodynamics.

4 weeks

Weekly visits for safety assessments and dose adjustments

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of adverse events and pharmacodynamics.

4 weeks

2 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• MIB-626

Trial Overview The trial is testing MIB-626, which is believed to potentially prevent or reverse Alzheimer's Disease in people with Down Syndrome. Participants will either receive MIB-626 or a placebo daily for 28 days and will be monitored for an additional month.

How Is the Trial Designed?

Group I: Safety and pharmacokinetic ascending dose intervention group Experimental Treatment 1 Intervention

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.

Group II: Placebo tablets Placebo Group 1 Intervention

Matching placebo tablets (number and frequency of tablets will match the active intervention group at each of the three doses)

Who Is Running the Clinical Trial?

Brigham and Women's Hospital

Lead Sponsor

Trials

1,694

Recruited

14,790,000+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07278492

NAD Augmentation to Prevent or Reverse Alzheimer's ... The aim of the study is to determine the safety and tolerability of different increasing doses of MIB-626 given daily for 28 consecutive ...

2. alzheimers.gov alzheimers.gov/clinical-trials/mib-626-alzheimers-disease

MIB-626 for Alzheimer's Disease This Phase 1/2 clinical trial will test the safety and effectiveness of the experimental drug MIB-626 to improve cognitive and daily functioning in people with ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05040321

NCT05040321 | Sirtuin-NAD Activator in Alzheimer's Disease These phase 1 studies have shown that a regimen of 1 g MIB-626 twice daily is safe and effective in substantially raising circulating NAD levels in healthy ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12312518/

Oral MIB‐626 (β Nicotinamide Mononucleotide) Safely ... MIB‐626 treatment significantly but gradually raised blood NAD+ levels to a peak between 5 to 14 days (16.0 ± 6.9, 25.5 ± 12.6, and 42.6 ± 25.6 μg/mL at ...

5. researchgate.net researchgate.net/publication/366568023_MIB-626_an_Oral_Formulation_of_a_Microcrystalline_Unique_Polymorph_of_b-Nicotinamide_Mononucleotide_Increases_Circulating_Nicotinamide_Adenine_Dinucleotide_and_its_Metabolome_in_Middle-aged_and_Older_

MIB-626, an Oral Formulation of a Microcrystalline Unique ... MIB-626 treatment was associated with substantial dose-related increases in blood NAD levels. Blood levels of NAD metabolites were higher in NMN-treated ...

6. rally.massgeneralbrigham.org rally.massgeneralbrigham.org/study/nmn_ad_study

Have you or a loved one been diagnosed with Alzheimer's ... We are conducting this study to determine how MIB-626 affects individuals with Alzheimer's disease. ... and our data indicates that this dose is safe.