Loading
Down Syndrome (DS) / MIB-626 · Phase 1 Trial
ClinicalTrials.gov · August 19, 2026
Early or partial results. Treat as a signal, not a conclusion.
This is a Phase 1 registry record for a planned randomized, placebo-controlled study of MIB-626 (a nicotinamide mononucleotide formulation) in 24 adults with Down syndrome. The trial is designed to evaluate safety, tolerability, and pharmacokinetics over 56 days of follow-up, with exploratory measures of aging and Alzheimer's disease biomarkers; no results are yet available.
Phase 1, Interventional, Randomized, Parallel, Quadruple masking, Treatment purpose. Down Syndrome (DS); age from 18 Years. Intervention: Safety and pharmacokinetic ascending dose intervention group. Compared with: Placebo tablets — Placebo Comparator. n = 24. 2 sites: United States.
Planned enrollment: 24 adults with Down syndrome, age ≥18 years Primary outcomes: treatment-emergent adverse events and serious adverse events from baseline to 56 days Intervention: MIB-626 tablets at escalating doses daily for 28 days versus placebo
No prior human safety or efficacy data on MIB-626 reported in this record. Primary outcomes: treatment-emergent adverse events and serious adverse events from baseline to 56 days
This early-phase trial is not yet recruiting and has not reported results. Clinicians should not base practice decisions on this registry entry. The study may inform future trials if safety and PK data support progression to efficacy evaluation in this vulnerable population.
Phase 1 safety and tolerability study in a small population (n=24) with surrogate mechanistic endpoints; no efficacy data reported and recruitment not yet started.
As stated by the source record.
Quoted from the source exactly as published.
This early-phase trial is not yet recruiting and has not reported results. Clinicians should not base practice decisions on this registry entry. The study may inform future trials if safety and PK data support progression to efficacy evaluation in this vulnerable population.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Registry record from ClinicalTrials.gov (NCT07278492). This is a study registration, not published results. Lead sponsor: Brigham and Women's Hospital. Recruitment status: NOT_YET_RECRUITING. Phase: PHASE1. Study type: INTERVENTIONAL. Enrollment: 24 participants (ESTIMATED). Conditions: Down Syndrome (DS). Interventions: DRUG: MIB-626; DRUG: Placebo. Primary outcome measures: Treatment emergent adverse events , Baseline to 56 days after drug administration; Treatment emergent serious adverse events , Baseline to 56 days after drug administration. Brief 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).
Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.