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LY3884961

Phase 1

Gaucher Disease | Gene therapy | Rare Disease |Eli Lilly and Company|Last Updated: Sep 18, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment8

FDA Designations

No designations recorded

Clinical trial landscape

LY3884961 · 3 trials · 4 indications

Phase 1 3
NCT05487599A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)Gaucher Disease
ACTIVE NOT_RECRUITING8 Analytics
NCT04411654Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)Gaucher Disease, Type 2
ACTIVE NOT_RECRUITING7 Analytics
NCT04127578Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)Parkinson Disease
ACTIVE NOT_RECRUITING32 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Gaucher DiseaseUnlock trial analytics
PHASE1ACTIVE NOT_RECRUITING
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
Gaucher Disease, Type 2Unlock trial analytics
PHASE1ACTIVE NOT_RECRUITING
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
Parkinson DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
5 years

Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) with AE's graded as mild, moderate, or severe.

Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation
Year 5
Immunogenicity of AAV9 and GCase in blood
Up to Year 2
Immunogenicity of AAV9 and GCase in CSF
Up to Year 1
Cumulative number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
5 years
Incidence of procedure or treatment-emergent AEs measured by brain MRI, spine MRI and nerve conduction study (NCS)
5 years
Treatment emergent immunogenicity of AAV9 in blood
Thru month 24
Change from baseline in immunogenicity of AAV9 in blood
Baseline and Month 24
Treatment emergent immunogenicity of GCase in blood
Thru Month 24

GCase (glucocerebrosidase)

Change from baseline in immunogenicity of GCase in blood
Baseline and Months 24

GCase (glucocerebrosidase)

Treatment emergent immunogenicity of Nfl in blood
Thru Month 24

GCase (glucocerebrosidase)

Change from baseline in immunogenicity of Nfl in blood
Baseline and Month 24

GCase (glucocerebrosidase)

Treatment emergent immunogenicity of AAV9 in CSF
Thru Month 24
Change in immunogenicity of AAV9 in CSF
Baseline and Month 12
Treatment emergent immunogenicity of GCase in CSF
Thru Month 24
Change in immunogenicity of GCase in CSF
Baseline, and Months 12

Secondary Endpoints

Spleen volume
5 years
Platelet count
5 years
GCase levels
5 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
LY3884961EXPERIMENTALLY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.
Low DoseEXPERIMENTAL -
High DoseEXPERIMENTAL -
Dose Level 1EXPERIMENTAL -
Dose Level 2EXPERIMENTAL -

Interventions

NameTypeDescription
LY3884961GENETIC• LY3884961 is a replication-incompetent recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.
MethylprednisoloneDRUGSingle IV pulse administered as concomitant medication.
SirolimusDRUGLoading dose, followed by maintenance doses, followed by dose tapering; administered as concomitant medication.
PrednisoneDRUGAdministered orally as concomitant medication, followed by dose tapering.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Age greater or equal to 18 years at the time of informed consent. 2. Bi-allelic pathogenic GBA1 variants must be centrally confirmed. 3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening. 4. Capable of giving ...

Countries:United StatesAustraliaGermanySpainUnited KingdomIsrael
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Recent Changes (Last 90 Days)

MEDIUMSep 19, 2026NCT04127578primaryCompletionDate: changed
LOWAug 20, 2026NCT04411654primaryCompletionDate: changed
LOWAug 20, 2026NCT04411654primaryCompletionDate: changed
LOWAug 20, 2026NCT04411654primaryCompletionDate: changed
LOWAug 20, 2026NCT04411654primaryCompletionDate: changed
HIGHAug 17, 2026NCT05487599Status: RECRUITING → ACTIVE_NOT_RECRUITING
HIGHAug 17, 2026NCT05487599Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 13, 2026NCT05487599lastUpdatePostDate: changed
LOWJul 13, 2026NCT05487599lastUpdatePostDate: changed

Frequently asked questions about LY3884961

What is LY3884961 used for?

LY3884961 is an investigational gene therapy being developed for Parkinson Disease, Gaucher Disease Type 2, and Gaucher Disease. It is in Phase 1 clinical trials for these conditions, which are being studied in patients with specific genetic mutations or disease manifestations.

Who makes LY3884961?

LY3884961 is being developed by Eli Lilly and Company, a biopharmaceutical company traded on the NYSE under the ticker LLY. The drug is currently in Phase 1 clinical development for rare disease indications.

What phase is LY3884961 in?

LY3884961 is in Phase 1 clinical development. It is an investigational gene therapy and has not been approved by regulatory authorities. It is being studied in early-stage clinical trials for Parkinson Disease and Gaucher Disease.

What clinical trials is LY3884961 in?

LY3884961 is being studied in three active Phase 1 clinical trials: NCT04127578 for Parkinson's Disease with GBA1 mutations, NCT04411654 for Type 2 Gaucher Disease in infants, and NCT05487599 for peripheral manifestations of Gaucher Disease. These trials are not yet recruiting participants.

Is LY3884961 the same as PR001?

Yes, LY3884961 is also known as PR001. Clinical trials reference the drug under both names, with PR001 used in trial titles and LY3884961 as the formal drug identifier.