None listed
Conditions
Interventions
Drug : Ambroxol is an oral administration drug.
1. Gaucher disease patients who have been treated with standard-of-care therapy for the disease, i.e., enzyme replacement therapy (imiglucerase, Abcerti
Sponsors
Asan Medical Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Male and female subjects aged between 2 and 60 years old, inclusive 2) Patients diagnosed with Gaucher disease by genetic testing or enzyme activity assay 3) Voluntarily signed written informed consent by patients or by both patients and their representatives
Exclusion criteria
Exclusion criteria: 1) Patients with severe renal impairment (eGFR < 30 mL/min/1.73 m2) 2) Patients with severe hepatic impairment (Child-Pugh class C) 3) Patients with genetic predisposition to lactose intolerance, galactose intolerance, Lapp lactase deficiency, or glucose-galactose malabsorption 4) Patients with known active hepatitis, HIV positive, or other uncontrolled infectious diseases 5) Patients who are unable to orally administer the study drug 6) Patients with hypersensitivities to the study drug or any of its components 7) Pregnant and lactating women, men and women of childbearing potential who are not willing to practice abstinence or use appropriate methods of contraception during the study 8) Patients who participated in any clinical trials other than GD-AMBX-01 within 90 days from treatment with the investigational product 9) Patients who are unable to participate in the study for other reasons, according to the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events (AEs) (Renal dysfunction, hepatic disease, digestive disorder, allergy and anaphylactoid reaction, Stevens-Johnson syndrome, Lyell syndrome, purulent rhinitis, etc.);Laboratory tests (hematology, blood chemistry, blood coagulation, and urinalysis);Vital signs (blood pressure, pulse, body temperature) and body measurements (body mass index);Electrocardiogram (ECG) and chest X-ray | — |
Secondary
| Measure | Time frame |
|---|---|
| Primary efficacy endpoint_Change in modified severity scoring tool (mSST) score;Secondary efficacy endpoints_Change in residual enzyme activity of GBA before treatment with enzyme replacement therapy;Secondary efficacy endpoints_Change in residual enzyme activity of GBA after treatment with enzyme replacement therapy;Secondary efficacy endpoints_Change in latency and threshold of brainstem auditory evoked response (BAER);Secondary efficacy endpoints_Change in N-acetyl acid/creatinine (NAA/Cr) and choline/creatinine (Cho/Cr) on brain magnetic resonance spectroscopy (MRS);Secondary efficacy endpoints_Improvement rate of ocular motility disorders;Secondary efficacy endpoints_Change in angle of esotropia;Secondary efficacy endpoints_Change in biomarkers of Gaucher disease (acid phosphatase, angiotensin converting enzyme, and chitotriosidase);Secondary efficacy endpoints_Change in hemoglobin concentrations;Secondary efficacy endpoints_Change in platelet levels;Secondary efficacy endpoints_Change in bone mineral density (BMD);Secondary efficacy endpoints_Change in intelligence test scores;Secondary efficacy endpoints_Change in the frequency of seizures;Secondary efficacy endpoints_Change in the size of liver and spleen;Exploratory endpoints_Transfer ratio of ambroxol (CSF to serum ambroxol concentration ratio);Exploratory endpoints_Pharmacokinetic parameter: serum Cmax, Tmax, t1/2, CL, AUCtau, etc. | — |
Countries
Korea, Republic of
Contacts
Public ContactYoo Wook
Asan Medical Center
Outcome results
None listed