Clinical Trials Logo
  1. Home
  2. Niemann-Pick Disease, Type C
  3. NCT05758922

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease — RAINBOW  

Niemann-Pick Disease, Type C Clinical Trial

Official title:
Randomized, Double Blind, Placebo Controlled, Multicenter, 12 Weeks Phase 2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Clinical Trial Summary

This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients. If approved by the country health authorities, a double-blind extension period will be proposed to the patients who complete the 12-week study.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05758922
Study type Interventional
Source Azafaros A.G.
Contact
Status Active, not recruiting
Phase Phase 2
Start date April 24, 2023
Completion date December 2024
View Details
See also
  Status Clinical Trial Phase
Completed NCT03759639 - N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) Phase 2
Withdrawn NCT04189601 - Complement Activation in the Lysosomal Storage Disorders
Completed NCT02435030 - A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C N/A
Completed NCT02534844 - VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease Phase 2/Phase 3
Recruiting NCT05588167 - Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Withdrawn NCT03687476 - Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease Phase 2
Enrolling by invitation NCT05368038 - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed NCT01899950 - Longitudinal Study of Cognition With Niemann-Pick Disease, Type C N/A
Terminated NCT00668564 - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Phase 2
Active, not recruiting NCT05163288 - A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C Phase 3
Active, not recruiting NCT02612129 - Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C Phase 2/Phase 3
Recruiting NCT03471143 - Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C Phase 1/Phase 2
Terminated NCT04958642 - Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease Phase 2/Phase 3
Terminated NCT03879655 - Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 Phase 2/Phase 3
Completed NCT00975689 - Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine Phase 1/Phase 2
Withdrawn NCT01306604 - Biomarker for Niemann Pick Type C Disease (BioNPC)
Recruiting NCT00344331 - Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Completed NCT03910621 - Safety and Efficacy of Miglustat in Chinese NPC Patients Phase 4
Available NCT04316637 - Early Access Program With Arimoclomol in US Patients With NPC