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Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

NCT04408625 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2026-04-30

No results posted yet for this study

Summary

Study J4B-MC-OKAA is a Phase 1/2, multi-center, open-label ascending dose, first-in-human study that will evaluate the safety and effect of intra-cisternal LY3884963 administration on progranulin protein (PGRN) levels in patients with frontotemporal dementia with progranulin mutations (FTD-GRN). Two escalating dose (low dose and medium dose) cohorts are planned, as well as one bridging cohort which will allocate patients to receive either low or medium dose. The duration of the study is 5 years. During the first year, patients will be evaluated for the effect of LY3884963 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will follow up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.

Conditions

  • Frontotemporal Dementia

Interventions

BIOLOGICAL

LY3884963

Participants will receive a single dose of LY3884963, administered intra cisterna magna

DRUG

Methylprednisolone

IV pulses every 2 weeks in the first 3 months.

DRUG

Optional Sirolimus

At the investigators discretion following steroid tolerability issues, patients may receive a loading dose, followed by maintenance dose, followed by dose tapering; administered as concomitant medication

DRUG

Optional Prednisone

If needed and at the investigator discretion, Oral Prednisone may be added to the immunosuppression regimen

Sponsors & Collaborators

Principal Investigators

  • Travis Lewis, MD, PhD · Prevail Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
30 Years
Max Age
85 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-09
Primary Completion
2029-11-30
Completion
2029-11-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • France
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04408625 on ClinicalTrials.gov