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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04408625
Other study ID # J4B-MC-OKAA (PRV-FTD101)
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date November 9, 2020
Est. completion date August 31, 2029

Study information

Verified date May 2024
Source Prevail Therapeutics
Contact Prevail Therapeutics
Phone (917) 336-9310
Email prevail.patients@lilly.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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Study Design


Intervention

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

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Sponsors (2)

Lead Sponsor Collaborator
Prevail Therapeutics Eli Lilly and Company

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  France,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events Leading to discontinuation 5 Years
Primary Sum of adverse reactions (ARs) and suspected ARs 5 years
Primary Sum of serious ARs and serious suspected ARs 5 years
Primary Incidence of procedure or treatment-emergent AEs Measured by brain and spine MRI 5 years
Primary Change in PGRN immunogenicity in blood PGRN: progranulin protein. Measured by level of antibodies and ELISPOT Baseline and Month 12
Primary Change in PGRN immunogenicity in CSF CSF: cerebrospinal fluid Baseline and Month 12
Primary Change in AAV9 immunogenicity in blood Measured by level of antibodies and ELISPOT. Baseline and Month 12
Primary Change in AAV9, PGRN, and NfL immunogenicity in CSF Measured by levels of antibodies. Baseline and Month 12
Primary Change in PGRN levels in blood Baseline and Month 12
Primary Change in PGRN levels in CSF Baseline and Month 12
Secondary Change in CDR plus NACC FTLD CDR: Clinical Dementia Rating staging instrument. NACC FTLD: National Alzheimer's Coordinating Center frontotemporal lobar degeneration domains Baseline and Month 12
Secondary Change in NfL levels in blood NfL: neurofilament light chain Baseline and Month 12
Secondary Change in NfL levels in CSF Baseline and Month 12
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