Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series
Key facts
- Where
- Amsterdam, HollandiaSee the site
- Phase
- 3. fázis
Where in Hollandia
- Amsterdam UMC StichtingAmsterdam, HollandiaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Amsterdam UMC Stichting
- Trial code
- EU 2024-514012-28-00
- Record updated
- 2024. november 25.
- Original description
Effect of ambroxol on cerebrospinal fluid (CSF) Lyso-GL1 in GD3 patients receiving ambroxol.Read full original descriptionShow less
Effect of ambroxol on cerebrospinal fluid (CSF) Lyso-GL1 in GD3 patients receiving ambroxol.
Inclusion: The patient or the parent(s) / legal guardian(s) must provide written informed consent before start of the study; Male and female patients with documented deficiency of GCase activity and GBA genotype fitting GD3; Male and female patients of all ages; Able to travel to the study site; Patients receive ERT with treatment ongoing at the time of enrollment.
Exclusion: The patient has received an investigational product within 30 days prior to enrollment; Known hypersensitivity reactions, intolerance or adverse reactions to ambroxol or to the inactive ingredients; Any condition or abnormality which may, in the opinion of the investigator, compromise the safety of patients; The patient is transfusion dependent; Pregnancy, because there are no sufficient data for the use of ambroxol in pregnant women (see Summary of Product Characteristics (SPC)); The patient is lactating. Ambroxol crosses into the breast milk. As there is no adequate experience in humans to date, ambroxol should not be used in lactation in a study setting (see SPC); The patient is unwilling or, in the investigator's opinion, unable to adhere to the requirements of the study; The patient is unable to swallow powder and has no other enteral access (e.g. gastrostomy).