AZ-3102: Clinical Trials

2 active not recruiting, 2 not yet recruiting, 1 recruiting. 3 Phase 2, 3 Phase 3.

By phase and status

Phase	Trials	Status
Phase 2	3	Not yet recruiting 2, Active not recruiting 1
Phase 3	3	Active not recruiting 1, Not yet recruiting 1, Recruiting 1

Follow AZ-3102: new trials by email Literature Review of AZ-3102 trials Research Report on AZ-3102

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders AZ-3102 - Niemann-Pick Disease Type C, GM2 Gangliosidosis	Phase 2 / Phase 3	Not yet recruiting	Azafaros B.V.	218	2026-12-10	2026-10-21
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3) AZ-3102 - Gaucher Disease Type 3	Phase 2	Not yet recruiting	Azafaros B.V.	20	2026-09-28	2026-07-20
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease AZ-3102 - GM2 Gangliosidosis, Niemann-Pick Type C Disease	Phase 2	Active not recruiting	Azafaros B.V.	21	2026-02-04	2026-10-21
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 AZ-3102, Placebo - Niemann-Pick Type C Disease, GM1 Gangliosidosis	Phase 3	Recruiting	Azafaros B.V.	147	2025-06-30	2026-09-24
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 AZ-3102, Placebo - Gangliosidoses, GM2, Gangliosidosis, GM1	Phase 3	Active not recruiting	Azafaros B.V.	75	2025-06-30	2026-09-22

Conditions studied

Gangliosidoses, GM2 (4)  ·  Gangliosidosis, GM1 (3)  ·  Niemann-Pick Disease, Type C (3)  ·  Gaucher Disease (2)

Sponsors

Azafaros B.V. (5)

Countries

Brazil (3)  ·  India (3)  ·  Turkey (Türkiye) (3)  ·  Argentina (2)  ·  Australia (2)  ·  Canada (2)  ·  France (2)  ·  Germany (2)  ·  Italy (2)  ·  Pakistan (2)  ·  Portugal (2)  ·  Spain (2)  ·  Sweden (2)  ·  Switzerland (2)  ·  United States (2)