PR001 for Gaucher Disease

Not currently recruiting at 4 trial locations

Overseen By Prevail Therapeutics

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Prevail Therapeutics

Must not be taking: Substrate reduction therapy, Immunosuppressants

Disqualifiers: Significant CNS disease, Independent gait, Severe peripheral symptoms, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called LY3884961 (formerly PR001) for infants with Type 2 Gaucher disease (GD2), a rare genetic disorder affecting metabolism. The study aims to determine if a single dose of this treatment is safe and effective in improving health markers and reducing symptoms over five years. The trial includes two groups: one receiving a high dose and the other a low dose. Infants diagnosed with GD2 due to specific genetic mutations who have not yet achieved independent walking might be eligible to participate. As a Phase 1/Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this innovative therapy.

Will I have to stop taking my current medications?

The trial requires that participants do not use certain medications, including substrate reduction therapy for Gaucher disease and some other prohibited medications, herbals, or over-the-counter agents. The protocol does not specify all medications that must be stopped, so it's best to discuss your current medications with the study team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that PR001, also known as LY3884961, is being tested for its safety in treating Gaucher disease. In earlier studies, doctors assessed the treatment's safety, patient tolerance, and potential immune reactions. So far, these studies have not linked PR001 to any serious or unexpected side effects. Patients generally tolerate the treatment well, but like all new treatments, it requires careful monitoring to ensure safety. This information comes from early trials, so more data is needed to fully understand the safety of PR001. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Gaucher disease?

Researchers are excited about PR001 for Gaucher Disease because it represents a novel approach that targets the root cause of the condition rather than just managing symptoms. Unlike standard enzyme replacement therapies, PR001 is a gene therapy designed to deliver a functional copy of the GBA1 gene directly to cells. This could potentially correct the enzyme deficiency at its source, offering a more lasting and effective solution. Additionally, PR001 has the potential to be a one-time treatment, which is a significant advantage over the regular infusions required with current therapies.

What evidence suggests that PR001 might be an effective treatment for Gaucher disease?

Research has shown that PR001, administered in this trial in either a high or low dose, holds promise for treating Gaucher Disease, particularly Type 2, which affects infants. This treatment provides a functional version of the GBA gene, often faulty in individuals with this condition. Initial findings suggest that PR001 can improve enzyme activity, crucial for breaking down certain fats that can otherwise accumulate and cause harm. Although data is still emerging, researchers hope this gene therapy could lead to better health outcomes by addressing the disease's root cause. Ongoing studies are needed to confirm these early positive results. ^{1 2 3 6 7}

Who Is on the Research Team?

Hamzeh Migdadi, M.D.

Principal Investigator

Prevail Therapeutics

Are You a Good Fit for This Trial?

This trial is for infants with Type 2 Gaucher Disease (GD2) who have a clinical diagnosis confirmed by the central lab. They must not have had prior gene or cell therapy, and should not be on treatments that could affect the study results. Parents or guardians need to consent and provide information about the child's health.

Inclusion Criteria

My guardian understands the study's risks and can consent for me.

I have been diagnosed with GD2.

Patient has a reliable informant (i.e., parent/legal guardian) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales)

Exclusion Criteria

I can stop my blood thinner medication for 7 days before and 48 hours after certain procedures.

I am not on any immunosuppressants or steroids, except for skin conditions or asthma.

Your lab test results at screening show significant abnormalities.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of LY3884961 and are evaluated for safety, tolerability, immunogenicity, biomarkers, and efficacy

12 months

Follow-up

Participants are monitored for safety and changes in biomarkers and clinical outcomes

4 years

What Are the Treatments Tested in This Trial?

Interventions

• PR001

Trial Overview The safety and effectiveness of LY3884961 (formerly PR001) are being tested in this Phase 1/2 trial over approximately five years. The first year focuses on how well patients tolerate the drug, its side effects, immune response, biomarkers, and initial efficacy.

How Is the Trial Designed?

Group I: Low Dose Experimental Treatment 4 Interventions

Group II: High Dose Experimental Treatment 4 Interventions

Who Is Running the Clinical Trial?

Prevail Therapeutics

Lead Sponsor

Trials

5

Recruited

190+

Eli Lilly and Company

Industry Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05487599

NCT05487599 | A Clinical Trial of PR001 (LY3884961) in ... A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED). ClinicalTrials.gov ID NCT05487599. Sponsor ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04411654

Study Details | NCT04411654 | Phase 1/2 Clinical Trial of ... J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants ...

3. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT04411654

PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) An open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 ...

4. scge.mcw.edu scge.mcw.edu/platform/data/clinicalTrials/report/NCT04411654

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 ... During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, ...

5. trial.medpath.com trial.medpath.com/clinical-trial/4266d2dc80245ea8

Phase 1/2 Clinical Trial of PR001 in Infants With ... - MedPath During the first 12 months after dosing, patients will be evaluated for the effects of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and ...

6. gaucherdiseasenews.com gaucherdiseasenews.com/pr001/

PR001 for Gaucher disease type 1 PR001 is an experimental gene therapy being developed to ease symptoms and slow or halt disease progression in people with Gaucher disease type 1.

7. clinicaltrial.be clinicaltrial.be/en/details/4608?per_page=20&recruiting=1&enrolling_by_invitation=1&active_not_recruiting=1&not_yet_recruiting=0&completed=0&only_recruiting=0&only_eligible=0

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patient... During the first year, patients will be evaluated for the effect of LY3884961 on safety, tolerability, immunogenicity, biomarkers, and clinical ...