Halted Phase I and Phase II (Integrated)- Other Type 1 Gaucher Disease (Peripheral/Non-neuronopathic Manifestations)

A dose finding study of LY3884961 in patients with Gaucher disease

EU CTIS ID: 2022-500281-10-02

What this study is testing

To evaluate the safety and tolerability of LY3884961

  • Phase I and Phase II (Integrated)- Other

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • 1. Age ≥ 18years at the time of informed consent. 2. Bi-allelic pathogenic GBA1 variants must be centrally confirmed. 3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening. 4. Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 5. 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 long-term follow-up. 6. Patients must agree to abstain from blood donations for at least the first year of the study; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.

You likely can't join if

  • 1. Clinically significant neurological signs and symptoms and/or behavioral disturbances. 2. Active and progressive bone disease expected to require surgical treatment in the next 6 months 3. History of total splenectomy or planned total splenectomy during the first 18 months of the study 4. Splenomegaly > 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI). 5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins 6. Thrombocytopenia with platelet count < 40 × 103 per μL 7. Severe hyperlipidemia (triglycerides > 1,000 mg/dL) 8. Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment 9. History of certain cancers within 5 years of Screening 10. 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 with the conduct of the study. 11. Women, who are pregnant (i.e., positive serum or urine pregnancy result at Screening and or Check-in) or breastfeeding or intending to become pregnant during the course of the trial. 12. 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 13. Any type of prior gene or cell therapy 14. Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression 15. Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer (unless it can be documented that the patient received placebo) 16. Have an anti-AAV9 antibody titer of >1:40 as determined by the central laboratory. 17. Clinically significant abnormalities in laboratory test results at Screening 18.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

  • Australia
  • United Kingdom
  • Brazil
  • United States

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, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Australia; United Kingdom; Brazil; United States. 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 EU CTIS; the study team decides eligibility.