Memantine for Down Syndrome

MR
AC
Overseen ByAlberto C Costa, MD, PhD
Age: 18 - 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?

This trial explores whether higher doses of memantine, a drug approved for Alzheimer's disease, can enhance cognitive function in adolescents and young adults with Down syndrome. Researchers aim to determine if this treatment can improve memory and thinking skills while also assessing its safety and metabolism in the body. The trial suits individuals with confirmed Down syndrome who are in good health, can swallow pills, and have a caregiver to assist with study requirements. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to contribute to groundbreaking research.

Do I have to stop taking my current medications for the trial?

The trial requires participants to stop taking any experimental drugs for Down syndrome before joining. If you are currently on psychotropic drugs (medications affecting mood, perception, or behavior), you cannot participate in the trial.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Earlier research found memantine to be well tolerated by teenagers and young adults with Down syndrome. At a dose of 20 mg per day, no major safety issues emerged. However, this dose did not improve thinking or memory in these individuals. The FDA has already approved memantine for treating Alzheimer's disease, indicating its general safety.

This trial tests higher doses of memantine. As this is an early stage of research, the primary focus is on collecting safety information. Initial signs suggest that memantine might be well tolerated even at higher doses, but this trial aims to provide more detailed information about its safety in people with Down syndrome.12345

Why do researchers think this study treatment might be promising?

Unlike standard treatments for Down syndrome, which primarily focus on managing symptoms through supportive therapies and medications for specific health issues, memantine is unique because it targets cognitive improvement directly. Memantine acts on the brain's NMDA receptors, which play a role in memory and learning. Researchers are excited about memantine because it could potentially enhance cognitive function, offering a new avenue for improving quality of life for individuals with Down syndrome. This approach distinguishes it from existing options that do not address cognitive deficits as directly.

What evidence suggests that memantine might be an effective treatment for Down syndrome?

Research has shown that memantine can improve thinking skills and slow the decline of Alzheimer's disease. However, studies on individuals with Down syndrome have produced mixed results. Some research found no significant improvement in thinking skills for people with Down syndrome, even when higher doses were used and tolerated well. Specifically, adolescents and young adults with Down syndrome showed no improvement in cognitive abilities. While memantine is safe, its effectiveness in enhancing thinking skills for people with Down syndrome remains uncertain. This trial will investigate the effects of escalating doses of memantine on cognitive function in individuals with Down syndrome.13456

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?

This trial is for adolescents and young adults with Down syndrome (Trisomy 21 or complete unbalanced translocation), in generally good health, able to swallow pills, have a reliable caregiver, and can understand English. Participants must not be pregnant and must not have certain genetic variations of Down syndrome.

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

Trial Overview

The study tests different higher-than-standard doses of memantine (an Alzheimer's drug) to see if it is safe and well-tolerated in people with Down syndrome. It also looks at how the drug affects thinking skills and measures its levels in the blood.

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+

National Institute on Aging (NIA)

Collaborator

Trials
1,841
Recruited
28,150,000+

Citations

Memantine for dementia in people with Down syndrome - PMC

Memantine is thought to improve cognitive function and slow the decline of AD over time.The effects of memantine on AD are reported to be beneficial.

The role of Memantine in slowing cognitive decline in ...

Our study finds no statistically significant therapeutic effect of Memantine in comparison to placebo in Down syndrome patients.

Escalating Doses of Memantine in Down Syndrome (MEDS ...

Safety, efficacy, and tolerability of memantine for cognitive and adaptive outcome measures in adolescents and young adults with Down syndrome ...

4.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/34942135/

a randomised, double-blind, placebo-controlled phase 2 trial

Memantine was well tolerated, but cognition-enhancing effects were not recorded with a 20 mg/day dose in adolescents and young adults with Down syndrome.

Memantine Therapy for Down Syndrome Shows Little ...

A dose of 20 mg/day of memantine was safe but did not have cognitive-enhancing effects in adolescents and young adults with Down syndrome.

Safety, efficacy, and tolerability of memantine for cognitive ...

Memantine was well tolerated, but cognition-enhancing effects were not recorded with a 20 mg/day dose in adolescents and young adults with Down syndrome.