NCT05758922: RAINBOW

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

Completed Phase 2 Last updated 12 May 2026

What this trial tests

Phase 2 trial testing AZ-3102 (Dose 1) in GM2 Gangliosidosis in 13 participants. Completed in 5 May 2026.

Timeline

24 April 2023

Primary endpoint
19 April 2024

5 May 2026

Quick facts

Lead sponsor	Azafaros A.G.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	13
Start date	24 April 2023
Primary completion	19 April 2024
Estimated completion	5 May 2026
Sites	3 locations across Brazil

Drugs / interventions tested

• AZ-3102 (Dose 1) — full drug profile →
• Placebo
• AZ-3102 (Dose 2)

Conditions studied

• GM2 Gangliosidosis — all drugs for GM2 Gangliosidosis →
• Niemann-Pick Disease, Type C — all drugs for Niemann-Pick Disease, Type C →

Sponsor

Azafaros A.G. — full company profile →

Who can join

12 and older, any sex, with GM2 Gangliosidosis or Niemann-Pick Disease, Type C. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Safety/tolerability: Incidence and severity of treatment emergent adverse events
  Time frame: Through study completion, up to Week 12
• Assessment of pharmacokinetic (PK) parameters in plasma: Cmax
  Time frame: Through study completion, up to Week 12
• Assessment of PK parameters in plasma: Tmax
  Time frame: Through study completion, up to Week 12
• Assessment of PK parameters in plasma: AUC0-24h
  Time frame: Concentration versus time curve calculated from time 0 to 24 hours (AUC0-24h)

Sponsor's own description

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. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Extracellular Vesicles as Tools for Crossing the Blood-Brain Barrier to Treat Lysosomal Storage Diseases.
   Lerussi G, Villagrasa-Araya V, Moltó-Abad M, Del Toro M, et al · · 2025 · cited 7× · PMID 39860010 · DOI 10.3390/life15010070
2. Inborn Errors of Metabolism with Ataxia: Current and Future Treatment Options.
   Bremova-Ertl T, Hofmann J, Stucki J, Vossenkaul A, et al · · 2023 · cited 2× · PMID 37759536 · DOI 10.3390/cells12182314
3. An Australian standard of care for Niemann-Pick disease type C.
   Tchan M, Smith N, Peters H, Van Velsen E, et al · · 2026 · PMID 41824299 · DOI 10.1111/imj.70370