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New treatment option for Gaucher Disease
Official title A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
ClinicalTrials.gov ID: NCT05487599
What this study is testing
What is LY3884961?
LY3884961 is an investigational medicine, being studied as a potential treatment for gaucher disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Study J3Z-MC-OJAE is a Phase 1/2, multicenter, open-label, dose-finding study of LY3884961 evaluating the safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Age greater or equal to 18 years at the time of informed consent.
- Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
- On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
- Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and...
- Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception...
You likely can't join if
- Clinically significant neurological signs and symptoms and/or behavioral disturbances.
- Active and progressive bone disease expected to require surgical treatment in the next 6 months.
- History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).
- Splenomegaly \> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)
- Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.
- Thrombocytopenia with platelet count \< 40 × 10\^3 per μL.
See the full eligibility criteria
- Age greater or equal to 18 years at the time of informed consent.
- Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
- On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
- Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study, including the...
- Patients must agree to abstain from blood, tissue and organ donation; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.
- Clinically significant neurological signs and symptoms and/or behavioral disturbances.
- Active and progressive bone disease expected to require surgical treatment in the next 6 months.
- History of total splenectomy or planned total splenectomy during the first 18 months of the study. (Partial splenectomy not exclusionary).
- Splenomegaly \> 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI)
- Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins.
- Thrombocytopenia with platelet count \< 40 × 10\^3 per μL.
- Severe hyperlipidemia (triglycerides \> 1,000 mg/dL).
- Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment.
- History of certain cancers within 5 years of Screening.
- Concomitant disease, condition or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere...
- Women of childbearing potential, pregnant (i.e., positive serum pregnancy result at Screening and/or Check-in) or breastfeeding or intending to become pregnant during the course of the trial.
- Use of any GD-related chaperone therapy within 4 weeks prior to Screening or expected need to initiate chaperone therapy during at least the first 18 months of the study.
- Any type of prior gene or cell therapy.
- Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression.
- Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer.
- Have an anti-AAV9 antibody titer of \>1:40 as determined by central laboratory.
- Clinically significant abnormalities in laboratory test results at Screening.
- Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic)implants/cardiac pacemaker.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Chicago, Illinois, United States
- Durham, North Carolina, United States
- Fairfax, Virginia, United States
- Westmead, New South Wales, Australia
- Porto Alegre, Rio Grande do Sul, Brazil
- Höchheim, Germany
- Zaragoza, Spain
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Chicago, Illinois, United States; Durham, North Carolina, United States; Fairfax, Virginia, United States; Westmead, New South Wales, Australia; Porto Alegre, Rio Grande do Sul, Brazil and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.