Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gauc… (NCT05222906) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
United States, Argentina43 participantsStarted 2022-04-18
Plain-language summary
This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and \<18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.
Who can participate
Age range12 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* The 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, is deemed clinically stable for at least 1 year by the Investigator and is within the therapeutic goals as all of the following:
* 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)
* 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
* Adult participant is ≥18 years of age
* Pediatric participant is ≥12 years \<18 years of age
* The participant has a clinical diagnosis of GD3 and a documented deficiency of acid beta-glucosidase activity confirming this diagnosis.
* The participant has a modified SARA score of 1 or above.
* The presence of gaze palsy, predominantly horizontal, with slow or absent saccades.
* If the participant has a history of seizures, they are well controlled under appropriate medication not identified as a strong or moderate inducer or inhibitor of CYP3A.
* Participants ≥ 30 kg of weight
* Contraception for sexually active male or female participants; not pregnant or breastfeeding; no sperm donating for male participant
* Signed written informed assent/consent
Exclusion Criteria:
* The participant is blood transfus…
What they're measuring
1
Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score
Timeframe: From baseline to Week 52
2
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score