A study to find how well LY3884961 works and how safe it is in participants with Parkinson’s disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Prevail Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2025-06-16

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Prevail Therapeutics, A Wholly Owned Subsidiary of Eli Lilly & Company

External identifiers

EU CT number

2024-519587-40-00

ClinicalTrials.gov

NCT04127578

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacodynamic, Others, Safety, Efficacy

to evaluate the safety, tolerability, and immunogenicity of 2 dose levels of LY3884961 administered via suboccipital injection into the cisterna magna

Secondary objectives 2

1. to evaluate the effect of LY3884961 on GCase enzyme activity and GCase protein levels in blood and cerebrospinal fluid (CSF)
2. to evaluate the effect of LY3884961 on Glycolipid levels in blood and CSF

Conditions and MedDRA coding

Parkinson’s Disease with at Least One GBA1 Mutation

Study design 2 periods

Regulatory references

Plan to share IPD

No

IPD plan description

All patient data is anonymized/coded, Sponsor does not have access to the deidentified data. There is no intention to share deidentified patient data.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. 1. Men or women aged 35 to 80 years (inclusive)
2. 2. Body weight range of ≥40 kg (88 lbs) to ≤110 kg (242 lbs)
3. 3. Diagnosis of PD per UK Parkinson’s Disease Society Brain Bank Clinical Diagnostic Criteria
4. 4. Hoehn and Yahr Stage III-IV (when off of PD medication)
5. 5. Stable use of background medications at least 8 weeks prior to investigational product (IP) administration, including but not limited to those used for treatment of PD
6. 6. At least 1 pathogenic GBA1 mutation confirmed by the central laboratory. Patients with bi-allelic GBA1 mutations and/or monogenic forms of PD will not be eligible. Only patients with low (< lower limit of normal) GCase enzyme activity measured in the blood will be eligible for participation.
7. 7. Negative screening test for Mycobacterium tuberculosis (MTB)
8. 8. Patient and/or patient’s legally authorized representative has the ability to understand the purpose and risks of the study and provide written informed consent
9. 9. Patient has a reliable study partner (e.g., family member, friend) willing and able to participate in the study as a source of information on the patient’s health status and cognitive and functional abilities
10. 10. Women of childbearing potential cannot be pregnant or lactating/breastfeeding

Exclusion criteria 10

1. 1. The diagnosis of a significant central nervous system (CNS) disease other than PD
2. 2. Montreal Cognitive Assessment (MoCA) score of <14
3. 3. Spinal, cervical, or brain MRI/magnetic resonance angiography (MRA) indicating clinically significant abnormality
4. 4. Hypersensitivity or contraindications to corticosteroid and/or sirolimus use
5. 5. Concomitant disease or condition within 6 months of Screening that could interfere with, or treatment of which might interfere with, the conduct of the study or that would, in the opinion of the Investigator, pose an unacceptable safety risk to the patient or interfere with the patient’s ability to comply with study procedures
6. 6. Clinically significant abnormalities in laboratory test results at Screenin
7. 7. Participation within 3 months prior to Screening in another therapeutic investigational drug or device study with purported disease-modifying effects on PD, unless it can be documented that the patient received placebo.
8. 8. History of deep brain stimulator placement, focused ultrasound, or surgery for PD
9. 9. Any type of prior gene or cell therapy
10. 10. Participants who are legally incompetent due to severe PD symptoms and/or cognitive impairment are not eligible for participation.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Incidence and severity of treatment-emergent AEs and SAE
2. Incidence of procedure or treatment-emergent safety findings
3. Treatment emergent and change from baseline in immunogenicity of adeno-associated viruses serotype 9 and glucocerebrosidase

Secondary endpoints 2

1. Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in blood
2. Change from baseline in glycolipid and glucocerebrosidase levels, as well as glucocerebrosidase enzyme activity in cerebrospinal fluid

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Prevail Therapeutics Inc.

Sponsor organisation

Prevail Therapeutics Inc.

Address

430 East 29th Street Ste 1520

City

New York

Postcode

10016-8367

Country

United States

Scientific contact point

Organisation

Prevail Therapeutics Inc.

Contact name

Regulatory Department

Public contact point

Organisation

Prevail Therapeutics Inc.

Contact name

Regulatory Department

Third parties 20

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 18 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications