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Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series
EU CTIS ID: 2024-514012-28-00
What this study is testing
Effect of ambroxol on cerebrospinal fluid (CSF) Lyso-GL1 in GD3 patients receiving ambroxol.
- Therapeutic confirmatory (Phase III)
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
- 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
You likely can't join if
- The patient is transfusion dependent
- 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
- 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
See the full eligibility criteria
- 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
- The patient is transfusion dependent
- 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
- 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)
- Any condition or abnormality which may, in the opinion of the investigator, compromise the safety of patients
The study team makes the final eligibility decision.
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, 0-17 years. The study team makes the final eligibility decision.
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.