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ACTIVE NOT RECRUITING Phase 1

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

NCT04408625 · View on ClinicalTrials.gov ↗

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 Studied

Interventions

  • DRUG Methylprednisolone
  • BIOLOGICAL LY3884963
  • DRUG Optional Sirolimus
  • DRUG Optional Prednisone

Study Locations (12)

Other

  • UZ Leuven, Neurologie Herestraat 49 — Leuven
  • Centre Mémoire de Ressources — Lille
  • Le Ber, Institut du Cerveau et de la Moelle Epinière — Paris
  • Hospital Clinic de Barcelona, Villaroel 170 Servicio de Neurología — Barcelona
  • Hospital Universitario de Donostia, Servicio De Neurologia, Consultas Externas Neurologia, San Sebastian, Guipúzcoa — Donostia / San Sebastian
  • University College London,Queen Square, Dementia Research Building, London, — London

Florida

  • k2 Medical Research-Maitland — Maitland
  • PPD Phase 1 Clinic, 100 West Gore Street, Suite 202 — Orlando

Massachusetts

  • Lahey Hospital & Medical Center, 41 Burlington Mall Road — Burlington

Pennsylvania

  • Hospital of the University of Pennsylvania, 3 West Gates Building, 3400 Spruce Street — Philadelphia

New South Wales

  • Royal Prince Alfred Hospital, Brain & Mind Research Institute, 94 Mallet Street — Camperdown

Marseille

  • AP-HM Hôpital de La Timone — Saint-Pierre

Trial Details

FieldValue
Enrollment Target 35 participants
Start Date 2020-11-09
Est. Completion 2029-11-30
Phase Phase 1

Sponsor

Prevail Therapeutics

5 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04408625

The ClinicalTrials.gov registry entry for NCT04408625 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Prevail Therapeutics, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 1 condition, with Frontotemporal Dementia appearing as the primary indexed condition, and to 4 interventions — of which Methylprednisolone is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT04408625 reports 12 study locations spanning 6 distinct geographic areas — top geographies include Other, Florida, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT04408625 about?

NCT04408625 is a clinical study titled "Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)". 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...

What is the current status of trial NCT04408625?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 35 participants. The study started on 2020-11-09. Estimated completion is 2029-11-30.

What conditions does trial NCT04408625 study?

This clinical trial studies the following conditions: Frontotemporal Dementia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT04408625?

The interventions under investigation include: Methylprednisolone (DRUG), LY3884963 (BIOLOGICAL), Optional Sirolimus (DRUG), Optional Prednisone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT04408625?

This trial is sponsored by Prevail Therapeutics, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT04408625 being conducted?

This trial has 12 study locations across Florida, Massachusetts, Pennsylvania, New South Wales, Marseille. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial