Azafaros B.V.
- Address
- J.H. Oortweg 21
Sponsor Overview
Specializations
Glycosphingolipid lysosomal storage disorders
Azafaros B.V. focuses on rare inherited disorders involving abnormal glycosphingolipid accumulation, with research centred on conditions that affect the nervous system and other organs.
- Niemann-Pick type C disease
- GM1 gangliosidosis
- GM2 gangliosidosis
The sponsor’s clinical activity is directed toward neurologic manifestations associated with these diseases, including movement impairment and progressive functional decline.
Niemann-Pick type C disease
Clinical research includes treatment evaluation in late-infantile and juvenile forms of NPC disease, with attention to disease-related neurological symptoms and patient function.
- Ataxia
- Juvenile-onset disease
- Late-infantile disease
Work in this area reflects a therapeutic interest in addressing central nervous system involvement in a rare metabolic disorder.
GM1 and GM2 gangliosidoses
The sponsor is also active in GM1 gangliosidosis and GM2 gangliosidosis, targeting the neurologic burden of these lysosomal storage diseases across paediatric and juvenile presentations.
- Late-infantile onset
- Juvenile onset
- Motor dysfunction
These studies align with therapeutic interest in disorders marked by progressive impairment of movement and neurological development.
Pharmacology and drug disposition
Azafaros B.V. also funds research into the absorption, metabolism, excretion, and bioavailability of nizubaglustat and AZ-3102, supporting its development in rare disease therapy.
- Metabolic profiling
- Pharmacokinetics
- Food effect
This area includes evaluation in healthy volunteers and healthy male subjects, with emphasis on understanding how the compound behaves in the body after oral administration.
Sponsored Trials
| Trial Name | Country | Status |
|---|---|---|
| Phase 1 Study on the Pharmacokinetics of [14C]-AZ-3102 in Glycosphingolipid Lysosomal Storage Disorders in Healthy Male Subjects |  The Netherlands | Not Recruiting |
| Phase 3 Study on the Efficacy and Safety of Nizubaglustat (AZ-3102) in Late-Infantile and Juvenile Niemann-Pick Type C and GM1/GM2 Gangliosidoses |  France Germany Italy Portugal Spain Sweden | Recruiting |
| Phase I Study on the Bioavailability and Food Effect of Nizubaglustat in Glycosphingolipid Lysosomal Storage Disorders | The Netherlands | Not Recruiting |
Data & Insights
This sponsor works with 2 molecules, of which 2 are not being studied by other sponsors.
This sponsor has worked with 8 sites across their trial portfolio.
Therapeutic Focus
- 1.Nutritional and Metabolic Diseases2 trials211 other sponsors
- 2.Congenital, Hereditary, and Neonatal Diseases and Abnormalities1 trial165 other sponsors
Partnered Research Centres
8 centres- Address
- Largo Professor Abel Salazar, 4050-011 Porto
- Address
- Via Giacomo Venezian 1, 20133 Milan
- Address
- 100 Boulevard Du General Leclerc, 92110 Clichy
- Address
- Passeig De La Vall D'Hebron 119-129, 08035 Barcelona
- Address
- Van Swietenlaan 6, 9728 NZ Groningen
- Address
- Behandlingsvagen 7, Harlanda, 416 50 Gothenburg
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