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Gene Therapy

Gene Therapy for Alzheimer's Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Lexeo Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of mild cognitive impairment due to Alzheimer's disease or mild to moderate dementia due to Alzheimer's disease
APOE4 homozygotes
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial is testing a gene therapy to treat patients with Alzheimer's who have two copies of the APOE4 gene. The trial will test different doses to see what is effective.

Who is the study for?
This trial is for APOE4 homozygotes with mild to moderate Alzheimer's-related cognitive impairment or dementia, who can consent and agree not to share study details on social media. Participants should not be on experimental meds or have active infections like hepatitis or HIV, and must avoid pregnancy during the study.Check my eligibility
What is being tested?
The trial tests LX1001 gene therapy in an open label, dose-ranging format for those diagnosed with varying stages of Alzheimer's disease due to APOE4 homogeneity. It aims to assess its safety and effectiveness in improving cognitive functions.See study design
What are the potential side effects?
While specific side effects are not listed here, gene therapies like LX1001 may cause immune reactions, injection site reactions, headache, nausea, fatigue and potential long-term risks which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with early or moderate Alzheimer's disease.
Select...
I have two copies of the APOE4 gene.
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I do not have any active infections, including hepatitis or HIV.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Proportion of participants with treatment-emergent adverse events and serious adverse events
The proportion of participants with treatment-emergent adverse events and serious adverse events at each dosage
Secondary outcome measures
Assess the levels of CSF biomarkers
Change from baseline in APOE2-APOE4 isoforms
Change from baseline in Clinical Dementia Rating Global and Sum of Boxes (CDR-SB)
+11 more

Trial Design

4Treatment groups
Experimental Treatment
Group I: Cohort 4: 1.4 x 10^14 gc (fixed dose)Experimental Treatment1 Intervention
Participants will receive 1.4 x 10^14 gc (fixed dose; approximately 3.4 × 10^11 gc/mL CSF based on an average CSF volume of 409 mL) of LX1001.
Group II: Cohort 3: 1.4 x 10^11 gc/mL CSFExperimental Treatment1 Intervention
Participants will receive 1.4 x 10^11 gc/mL CSF of LX1001.
Group III: Cohort 2: 4.4 x 10^10 gc/mL CSFExperimental Treatment1 Intervention
Participants will receive 4.4 x 10^10 gc/mL CSF of LX1001.
Group IV: Cohort 1: 1.4 x 10^10 gc/mL CSFExperimental Treatment1 Intervention
Participants will receive 1.4 x 10^10 gc/mL CSF of LX1001.

Find a Location

Who is running the clinical trial?

Alzheimer's Drug Discovery FoundationOTHER
21 Previous Clinical Trials
3,126 Total Patients Enrolled
Weill Medical College of Cornell UniversityOTHER
1,054 Previous Clinical Trials
1,316,513 Total Patients Enrolled
Lexeo TherapeuticsLead Sponsor
3 Previous Clinical Trials
34 Total Patients Enrolled

Media Library

LX1001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03634007 — Phase 1 & 2
Alzheimer's Disease Research Study Groups: Cohort 3: 1.4 x 10^11 gc/mL CSF, Cohort 4: 1.4 x 10^14 gc (fixed dose), Cohort 1: 1.4 x 10^10 gc/mL CSF, Cohort 2: 4.4 x 10^10 gc/mL CSF
Alzheimer's Disease Clinical Trial 2023: LX1001 Highlights & Side Effects. Trial Name: NCT03634007 — Phase 1 & 2
LX1001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03634007 — Phase 1 & 2
Alzheimer's Disease Patient Testimony for trial: Trial Name: NCT03634007 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many participants have been enrolled in this clinical trial so far?

"This trial necessitates that 15 participants, who meet the pre-set conditions for inclusion, register. Potential test subjects can enrol at either PPD Development in Orlando or Weill Cornell Medicine located in New york City."

Answered by AI

Are there vacancies available for enlistment in this research project?

"According to clinicaltrials.gov, this medical trial is still recruiting patients and has been since its posting on November 6th 2019. The most recent update was logged on the 29th of November 2022."

Answered by AI

Who else is applying?

What state do they live in?
New York
What site did they apply to?
Weill Cornell Medicine
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

Want to stay aware of potential cures of Alzheimer’s disease, as a single mother.
PatientReceived 1 prior treatment

What questions have other patients asked about this trial?

Can you use exiting data on me for screening, given extensive testing at NYC hospitals?
PatientReceived 1 prior treatment
Recent research and studies
clinicaltrials.govStudy
Gene Therapy for APOE4 Homozygote of Alzheimer's Disease
2019. "Gene Therapy for APOE4 Homozygote of Alzheimer's Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03634007.
All > Alzheimer Disease > Alzheimers Disease
~2 spots leftby Nov 2024
Unbiased ResultsWe believe in providing patients with all the options.
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