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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up year 5

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ year 5

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~year 5

This trial's timeline: 3 weeks for screening, Varies for treatment, and year 5 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Immunogenicity of AAV9 and GCase in CSF

Immunogenicity of AAV9 and GCase in blood

Number of Adverse Events (AEs), Serious Adverse Events (SAEs), and Adverse Events leading to discontinuation

Secondary outcome measures

Change in Clinical Global Impressions (Severity)

Change in GCase (glucocerebrosidase) enzyme activity levels in blood

Change in GCase enzyme activity levels in CSF (cerebrospinal fluid)

+11 more

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

~2370

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Eli Lilly and CompanyIndustry Sponsor

2,615 Previous Clinical Trials

3,201,043 Total Patients Enrolled

1 Trials studying Gaucher Disease

15 Patients Enrolled for Gaucher Disease

Prevail TherapeuticsLead Sponsor

3 Previous Clinical Trials

58 Total Patients Enrolled

1 Trials studying Gaucher Disease

15 Patients Enrolled for Gaucher Disease

Travis Lewis, MD, PhDStudy 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this clinical trial extend to seniors aged eighty or above?

"This medical trial is specifically targeting infants aged 0 - 24 months. There are 134 clinical trials for minors and 430 for senior citizens available at this time."

Answered by AI

Who meets the criterion for inclusion in this research project?

"For this clinical trial, up to 15 children between the ages of 0-24 months with a confirmed diagnosis of Gaucher Disease will be accepted. In addition, applicants must possess bi-allelic mutations in GBA1 consistent with GD2 and have an understanding parent/legal guardian who can provide informed consent as well as information on the patient's health status if requested."

Answered by AI

How many persons are engaged in this clinical examination?

"To join this study, 15 participants meeting the designated criteria are required. Those interested can register at sites such as Children's Hospital of Pittsburgh on 4401 Penn Avenue in Pittsburgh and University of Minnesota Masonic Children's Hospital located on 2450 Riverside Avenue in Minneapolis."

Answered by AI

Are there any other relevant investigations conducted concerning PR001?

"Currently, PR001 is the subject of 516 separate clinical trials with 125 in Phase 3. Most are held at medical centres located in Duarte, California; however, there are 18250 sites across the world hosting this research."

Answered by AI

Is enrollment currently open for this research endeavor?

"Affirmative, clinicaltrials.gov displays that this experiment is actively seeking participants. This research was originally posted on June 29th 2021 and has been revised as of July 26th 2022. It requires the enrollment of 15 people from 3 distinct medical institutions."

Answered by AI

What has PR001 been predominantly utilized for?

"PR001 is the most commonly prescribed medicine for scalp structure conditions. However, it can also be beneficial to those suffering from organ transplantation complications, thyroiditis and ulcerative colitis."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

2021. "Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04411654.

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