SphinCS GmbH

Höchheim 65239

4 studies enrolling now · 10 studies all time

What they study most

AB Variant Gangliosidosis GM2, Fabry Disease, GM1 Gangliosidosis, GM2 Gangliosidosis, Gangliosidoses, GM2, Gangliosidosis, GM1, Gaucher Disease, Type 2, Glycogen Storage Disease Type II Infantile Onset, Mucopolysaccharidosis II, Niemann-Pick Disease, Type C1, Niemann-Pick Type C Disease, Pompe Disease

Studies at this site

• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis — Recruiting now
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) — Recruiting now
• Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) — Recruiting now
• A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 — Recruiting now
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis — Running, not enrolling
• A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Running, not enrolling
• A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) — Running, not enrolling
• Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease — Completed
• Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 — Status unconfirmed
• Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 — Completed