Active not recruitingPHASE1, PHASE2INTERVENTIONAL

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

This study, called PROVIDE, is looking into a new treatment, LY3884961, for infants suffering from Type 2 Gaucher disease. This is an inherited condition that can cause severe problems. The study, which involves multiple hospitals, is a Phase 1/2 trial, meaning it's an early-stage study to check both the safety and effectiveness of the new treatment. Researchers will give a single dose of LY3884961 and then carefully monitor the babies for 12 months to see how they tolerate it, if their bodies react to it, and if it helps their condition. Following this, the children will be followed for another four years to keep an eye on their safety and any longer-term changes in their health. The total study duration for each child will be about five years.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Prevail Therapeutics

Enrolment target

Start

29 Jun 2021

Estimated completion

01 May 2028

Gaucher Disease, Type 2

What is this study about?

Gaucher disease is a rare inherited condition. In Type 2 Gaucher disease, crucial substances build up in the body's cells, leading to serious health problems, especially affecting the brain, and it can be very severe in babies. This clinical trial, known as PROVIDE, is exploring a new treatment called LY3884961.

The main goal of this study is to find out if this new treatment is safe for infants with Type 2 Gaucher disease and if it could help improve their condition. Because it's a Phase 1/2 study, it's an early step in testing the treatment, gathering initial information on how it works in people. This type of research is vital in developing new medicines for conditions where there are limited treatment options.

Researchers will be giving a single dose of the treatment and then carefully watching the babies over a long period. They'll be looking at various health markers, how the body reacts to the treatment, and any changes in the babies' overall health. The information gathered from this study will be crucial for understanding the potential of LY3884961 and deciding if further, larger studies should be undertaken.

Key takeaways

This is an early-stage study (Phase 1/2) testing a new treatment (LY3884961) for Type 2 Gaucher disease in babies.
The study aims to understand the safety and potential effectiveness of a single dose of the treatment.
Participation involves close medical monitoring for up to five years.
It's for infants aged 0 to 24 months with a confirmed diagnosis of Type 2 Gaucher disease.
The study involves multiple hospitals and will track a range of health indicators.
Parents or guardians must provide informed consent and participate in sharing health information.

Who may be eligible?

For an infant to take part in this study, they must have a confirmed diagnosis of Type 2 Gaucher disease through specific genetic tests. They need to be between birth and 24 months old. Importantly, a parent or legal guardian must be able to understand the study, give their permission for the child's participation, and be able to provide information about the child's health throughout the study.

There are also some reasons why an infant wouldn't be able to join. For example, if they have other serious brain conditions not caused by Gaucher disease, or if their Gaucher disease is causing severe problems in a way that might make the study unsafe for them. Also, if they've received certain other treatments, like gene therapy or specific medications, or if they have certain medical scan results that would make the treatment unsafe, they unfortunately wouldn't be eligible.

Essentially, the medical team needs to be confident that taking part in the study would be as safe as possible for your child and that their health condition allows for meaningful assessment of the new treatment.

Quick self-check

Is my child between birth and 24 months old?
Does my child have a confirmed diagnosis of Type 2 Gaucher disease?
Am I able to understand the study and provide information about my child's health?
Has my child avoided certain treatments like prior gene therapy or specific strong medications?
Does my child have any other severe health conditions that aren't Gaucher disease?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If your child takes part in this study, they will receive a single dose of the study treatment, LY3884961. After this, there will be a lot of regular check-ups and assessments over a period of 12 months. These will involve monitoring your child's safety, how their body is handling the treatment, and looking for any signs that the treatment might be helping. These assessments often include physical examinations, blood tests, and other specific tests to check their health.

Following the first year, your child will continue to be monitored for another four years. During this longer follow-up period, the focus will be on keeping an eye on their overall safety and any long-term changes in their condition. The total duration of participation in the study for each child will be approximately five years. Throughout this time, you, as their parent or guardian, will be asked to provide information about your child's health and development.

Potential risks and benefits

Participating in a clinical trial offers the chance to access a new and potentially beneficial treatment that isn't yet widely available. However, there are no guarantees that the treatment will help your child, and there could be side effects or risks that are currently unknown. The study team will explain all known risks. Your child will receive extra medical care and monitoring throughout the study. Remember, participation is completely voluntary, and you have the right to withdraw your child from the study at any time, for any reason, without it affecting their future medical care.

Locations (5)

UCSF Benioff Children's Hospital, 747 52nd St
Oakland, United States
University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue
Minneapolis, United States
Children's Hospital of Pittsburgh, 4401 Penn Avenue
Pittsburgh, United States
Lysosomal & Rare Disorders Research and Treatment Center
Fairfax, United States
Manchester Centre for Genomic Medicine, 6th Floor, St Mary's Hospital, Oxford Road
Manchester, United Kingdom

Common questions

What is Type 2 Gaucher disease?

It's a serious inherited condition where certain substances build up in the body's cells, leading to health problems, particularly affecting the brain in infants.

What is LY3884961?

It's a new treatment being tested in this study for infants with Type 2 Gaucher disease to see if it is safe and effective.

How long will my child be in the study?

Each child will be part of the study for about five years in total, with intense monitoring in the first year and less frequent checks for the next four years.

Will my child receive more than one dose of the treatment?

No, in this particular study, infants will receive a single dose of the treatment, and then their health will be monitored over time.

Can I take my child out of the study if I change my mind?

Yes, you have the right to withdraw your child from the study at any time, and their routine medical care will not be affected.