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Enzyme Replacement Therapy

PR001 for Gaucher Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Prevail Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Parent/legal guardian has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations
Clinical diagnosis of GD2
Must not have
Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives
Contraindications to general anesthesia or sedation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline until event or study completion, up to year 5
Awards & highlights
No Placebo-Only Group

Summary

This trial is studying a new drug for infants with a rare disease called Gaucher disease. The study will last about 5 years, and patients will be monitored for safety and efficacy.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Potential side effects may include reactions related to general anesthesia or sedation required for treatment administration, intolerance to contrast agents used during MRI or CT scans as part of evaluation procedures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My guardian understands the study's risks and can consent for me.
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I have been diagnosed with GD2.
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My condition is confirmed as GD2 with specific GBA1 mutations.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have a brain or nervous system condition that is not related to my GD symptoms.
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I cannot have general anesthesia or sedation due to health reasons.
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I have never received gene or cell therapy.
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I am currently using or have used growth disorder treatments.
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I can walk on my own without help.
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My brain scans show a condition that makes certain injections unsafe.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline until event or study completion, up to year 5
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline until event or study completion, up to year 5 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change in Clinical Global Impressions (Severity)
Change in adaptive behavior and functioning
Change in behavioral symptoms
+5 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Low DoseExperimental Treatment4 Interventions
Group II: High DoseExperimental Treatment4 Interventions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Methylprednisolone
2015
Completed Phase 4
~2280
Sirolimus
2013
Completed Phase 4
~2750
Prednisone
2014
Completed Phase 4
~2500

Find a Location

Who is running the clinical trial?

Eli Lilly and CompanyIndustry Sponsor
2,680 Previous Clinical Trials
3,466,369 Total Patients Enrolled
1 Trials studying Gaucher Disease
15 Patients Enrolled for Gaucher Disease
Prevail TherapeuticsLead Sponsor
4 Previous Clinical Trials
173 Total Patients Enrolled
1 Trials studying Gaucher Disease
15 Patients Enrolled for Gaucher Disease
Travis Lewis, MD, PhDStudy DirectorPrevail Therapeutics
Sarah Neuhaus, D.O.Study DirectorPrevail Therapeutics

Media Library

PR001 (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04411654 — Phase 1 & 2
Gaucher Disease Research Study Groups: High Dose, Low Dose
Gaucher Disease Clinical Trial 2023: PR001 Highlights & Side Effects. Trial Name: NCT04411654 — Phase 1 & 2
PR001 (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04411654 — Phase 1 & 2
~7 spots leftby May 2028
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