Memantine for Down Syndrome

MR
AC
Overseen ByAlberto C Costa, MD, PhD
Age: < 65
Sex: Any
Trial Phase: Phase 1
Sponsor: University Hospitals Cleveland Medical Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

Down syndrome (DS) is typically caused by an extra chromosome 21 in the cell nucleus (trisomy 21, or T21). T21 is both the most common cause of genetically defined intellectual disability and the earliest documented cause of Alzheimer's disease (AD)-type pathology. Currently, all presymptomatic individuals with DS are classified as having 'Stage 0' DS-associated AD (DSAD). DSAD pathology evolves inexorably, with virtually all individuals with DS developing AD pathology by age 40, and approximately 50% meeting clinical dementia diagnosis criteria at 55 years of age. This study will test the hypothesis that the FDA-approved AD drug memantine, at higher-than-standard doses, may be effective as a cognitive enhancer in adolescents and young adults with DS. The primary goal of this phase 1b clinical trial will be the assessment of the safety and tolerability of three memantine doses in persons with DS. In addition, we will assess the effect of this drug on cognitive test scores and plasma biomarkers of AD in the study participants. Finally, we will also investigate steady-state plasma levels of memantine and the time course of memantine plasma levels after a single dose in the study participants (pharmacokinetics, or PK). The data generated through this phase 1b study will provide the essential safety, PK, and preliminary efficacy signals required to advance a phase 2 trial evaluating high-dose memantine as a first-in-class therapeutic strategy in DS.

Who Is on the Research Team?

AC

Alberto C Costa, MD, PhD

Principal Investigator

University Hospitals Cleveland Medical Center

SL

Stephen L Ruedrich, MD

Principal Investigator

University Hospitals Cleveland Medical Center

Are You a Good Fit for This Trial?

Inclusion Criteria

I have Trisomy 21 or a full unbalanced translocation of chromosome 21.
* Laboratory findings within normal limits or judged clinically insignificant at baseline
* Vital signs within normal limits for age. Stable, medically treated hypotension will be allowed
See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive escalating doses of memantine (20 mg/day, 40 mg/day, and 60 mg/day) for 9 weeks at each dosing stage

27 weeks
Regular visits for dose escalation and monitoring

Washout

Participants undergo a washout period to evaluate the reversibility of any observed drug effect

9 weeks

Pharmacokinetics

A single 20 mg oral dose of memantine is administered to evaluate absorption, distribution, and excretion

197 hours
Multiple blood sampling visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Memantine

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: MemantineExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

University Hospitals Cleveland Medical Center

Lead Sponsor

Trials
348
Recruited
394,000+