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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

Description

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis

Conditions

Gangliosidoses, GM2Gangliosidosis, GM1
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Related Conditions:

Gangliosidoses, GM2Gangliosidosis, GM1

Study Overview

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Study Details

Study overview

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2 gangliosidosis

18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

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

Condition
Gangliosidoses, GM2
Intervention / Treatment

-

Contacts and Locations

Oakland

Children's Hospital and Research Center at Oakland, Oakland, California, United States, 94609

Rochester

Mayo Clinic Children's Center - PIN, Rochester, Minnesota, United States, 55905

Fairfax

Lysosomal Rare Disorders Research and Treatment Center, Fairfax, Virginia, United States, 22030-7404

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Confirmed GM1 gangliosidosis or Tay-Sachs, Sandhoff, or GM2AB variant
  • * Male and female participants aged 4 years and older at the time of informed consent
  • * Onset of neurological symptoms from 1 to 10 years
  • * Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
  • * Females of childbearing potential who are sexually active willing to follow the contraceptive guidance
  • * Male participants with a female partner of childbearing potential willing to follow the contraceptive guidance
  • * A history of medical conditions other than GM1 or GM2 gangliosidosis that, in the opinion of the Principal Investigator, would confound scientific rigor or the interpretation of results
  • * Body weight of \<10 kg
  • * The presence of another neurologic disease
  • * The presence of moderate or severe hepatic impairment
  • * The presence of moderate or severe renal impairment
  • * Platelet count of \<100x10\^9/L
  • * The dose of any anti-epileptic treatment(s) was not stable (required a change in dose within the previous 3 months) and/or a new anti-epileptic treatment (drug or procedure) was prescribed in the month before Baseline
  • * Prior use of an investigational drug within the 3 months before Screening; or prior participation in a clinical study involving gene therapy or stem cell transplantation within 2 years prior to Screening
  • * A positive serum pregnancy test (for women of childbearing potential)

Ages Eligible for Study

4 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Azafaros A.G.,

Study Record Dates

2027-11-04