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LY3884961

Phase 1

Gaucher Disease, Type 2 | Gene therapy | Rare Disease |Eli Lilly and Company|Last Updated: Jul 16, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment7

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
RECRUITING15 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
PHASE1RECRUITING
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 Sites9

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 StatesAustraliaBrazilGermanySpainUnited KingdomIsrael
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Recent Changes (Last 90 Days)

LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 17, 2026NCT04127578primaryCompletionDate: changed
LOWJul 13, 2026NCT05487599lastUpdatePostDate: changed
LOWJul 13, 2026NCT05487599lastUpdatePostDate: changed
MEDIUMMay 26, 2026NCT05487599primaryCompletionDate: changed
LOWMay 26, 2026NCT04127578primaryCompletionDate: changed
LOWMay 26, 2026NCT04411654primaryCompletionDate: changed
LOWMay 24, 2026NCT05487599studyFirstPostDate: changed
LOWMay 24, 2026NCT04127578studyFirstPostDate: changed
LOWMay 24, 2026NCT04411654studyFirstPostDate: changed

Frequently asked questions about LY3884961

What is LY3884961 used for?

LY3884961 is an investigational gene therapy being studied for Gaucher Disease, Parkinson Disease, and Gaucher Disease Type 2. It is being evaluated in clinical trials for patients with Parkinson's disease who have at least one GBA1 mutation, as well as for infants with Type 2 Gaucher disease and adults with peripheral manifestations of Gaucher disease.

Who makes LY3884961?

LY3884961 is being developed by Eli Lilly and Company (ticker: LLY). The company is conducting clinical trials of this gene therapy across multiple indications, including Parkinson's disease and Gaucher disease.

What phase is LY3884961 in?

LY3884961 is in Phase 1 clinical development. It is an investigational gene therapy that has not been approved by regulatory authorities. The drug is currently being studied in early-stage clinical trials for Gaucher disease and Parkinson's disease.

What clinical trials is LY3884961 in?

LY3884961 is being studied in three clinical trials: NCT04127578 (PROPEL) in Parkinson's disease with GBA1 mutations, NCT04411654 (PROVIDE) in Type 2 Gaucher disease, and NCT05487599 (PROCEED) in peripheral manifestations of Gaucher disease. All trials are in Phase 1 and are active, with one still recruiting.

Is LY3884961 the same as PR001?

Yes, LY3884961 is also known as PR001. The clinical trials refer to the drug as PR001 (LY3884961), including the PROPEL, PROVIDE, and PROCEED studies. This gene therapy is being developed by Eli Lilly and Company for Gaucher disease and Parkinson's disease.