BridgeMD
Venglustat in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3 with venglustat monotherapy extension
EU CTIS ID: 2023-508646-18-00
What this study is testing
Part 1: Biomarker evaluation/screening phase Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants Screen adult GD3 participants who qualify for treatment with venglustat in Parts 2, Part 3, and Part 4 Parts 2 and 3: Combination treatment phases Evaluate short-term (Part 2) and long-term (Part 3) safety and tolerability of venglustat in combination with Cerezyme in adult GD3 participants Evaluate the change in CSF central nervous system (CNS) biomarkers (glucosylceramide [GL-1] and lyso-glucosylceramide [lyso-GL-1]) from adult GD3 participants receiving venglustat in combination with Cerezyme (Part 2 only) Part 4: Extended treatment phase with monotherapy Evaluate safety and tolerability of venglustat monotherapy in adult GD3 participants who have remained systemically stable on venglustat in combination with Cerezyme
- Therapeutic exploratory (Phase II)
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
- -GD1 participant is ≥18 and ≤40 years of age.
- -Participant is willing to abstain from consumption of grapefruit, grapefruit juice, or grapefruit containing products for 72 hours prior to administration of the first dose of venglustat and for the duration of the treatment period.
- -Oculomotor apraxia characterized by a horizontal saccade abnormality.
- -Female participants of childbearing potential and male participants must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception for the duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of venglustat.
- -GD3 participant is ≥18 years of age.
- -Participant must provide written informed consent prior to any study-related procedures being performed.
You likely can't join if
- -Participant has myoclonic seizures.
- -Participant has had a major organ transplant (e.g., bone marrow or liver).
- -Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.
- -Participant, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., contraindications for magnetic resonance imaging).
- -Participant has had a partial or total splenectomy within 3 years prior to randomization.
- -Participant is blood transfusion-dependent.
See the full eligibility criteria
- -GD1 participant is ≥18 and ≤40 years of age.
- -Participant is willing to abstain from consumption of grapefruit, grapefruit juice, or grapefruit containing products for 72 hours prior to administration of the first dose of venglustat and for the duration of the treatment period.
- -Oculomotor apraxia characterized by a horizontal saccade abnormality.
- -Female participants of childbearing potential and male participants must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use 2 acceptable effective methods of contraception for the duration of the study and for at least 6 weeks for females and 90 days for males following their last dose of venglustat.
- -GD3 participant is ≥18 years of age.
- -Participant must provide written informed consent prior to any study-related procedures being performed.
- -Participant has a clinical diagnosis of Gaucher disease Type 1 (GD1) or Gaucher disease Type 3 (GD3) and documented deficiency of acid beta-glucosidase activity confirming this diagnosis.
- -Participant has received ERT (Cerezyme or other ERT; as deemed appropriate by local regulations) for at least 3 years prior to enrollment, on a stable dose for at least 6 months and is within the therapeutic goals defined below, and is deemed clinically stable for at least 1 year by the Investigator.
- -Participant has reached Gaucher disease therapeutic goals defined as all of the following to be eligible for this study: -Hemoglobin level of ≥11.0 g/dL for females and ≥12.0 g/dL for males. -Platelet count ≥100,000/mm3. -Spleen volume <10 multiples of normal (MN), or total splenectomy (provided the splenectomy occurred >3 years prior to randomization). -Liver volume <1.5 MN. -No bone crisis and free of symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathological fractures within 3 months prior to screening.
- -Participant has maintained GD therapeutic goals defined as all of the following to be eligible for entering Part 4 of this study: -Hemoglobin level of ≥11.0 g/dL for females and ≥12.0 g/dL for males -Platelet count ≥100 000/mm3 -Spleen volume <10 multiples of normal (MN), or total splenectomy -Liver volume <1.5 MN -No bone crisis and free of symptomatic bone disease such as bone pain attributable to osteonecrosis and/or pathological fractures within 3 months prior to entering Part 4
- -Participant, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin (β-hCG)] at baseline.
- -If participant has a history of seizures, except for myoclonic seizures, they are well controlled under appropriate medication not identified as a strong or moderate inducer or inhibitor of cytochrome P450 (CYP) 3A.
- -Participant has myoclonic seizures.
- -Participant has had a major organ transplant (e.g., bone marrow or liver).
- -Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.
- -Participant, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., contraindications for magnetic resonance imaging).
- -Participant has had a partial or total splenectomy within 3 years prior to randomization.
- -Participant is blood transfusion-dependent.
- -Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal, unless the participant has a diagnosis of Gilbert Syndrome.
- -Participant has any clinically significant disease, other than GD, including cardiovascular (congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect), hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia) or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation.
- -Participant has renal insufficiency, as defined by an estimated glomerular filtration rate <30 mL/min/1.73m2 at the screening visit.
- -Participant has received an investigational product within 30 days prior to enrollment.
- -Participant has a history of cancer, with the exception of basal cell carcinoma.
- -Participant is pregnant or lactating.
- -Participant has, according to World Health Organization (WHO) Grading, a cortical cataract > one-quarter of the lens circumference (Grade cortical cataract-2) or a posterior subcapsular cataract >2 mm (Grade posterior subcapsular cataract-2). Participants with nuclear cataracts will not be excluded.
- -Participant requires use of invasive ventilatory support.
- -Participant requires use of noninvasive ventilator support while awake for longer than 12 hours daily.
- -Participant is unable to receive treatment with Cerezyme due to a known hypersensitivity or is unwilling to receive Cerezyme treatment to ensure maintenance of Gaucher treatment goals.
- -Participant is currently receiving potentially cataractogenic medications (corticosteroids, psoralens used in dermatology with ultraviolet light therapy [PUVA], typical antipsychotics, and glaucoma medications) or any medication that may worsen the vision of a participant with cataract (eg, alphaadrenergic glaucoma medications).
- -Participant has received strong or moderate inducers or inhibitors of CYP3A within 15 days or 5 half-lives from screening, whichever is longer, prior to enrolment in Part 2. This also includes the consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat administration in Parts 2 and 3.
- -Participant is scheduled for in-patient hospitalization including elective surgery, during the study.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Japan
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. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Japan. 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.