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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)

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 Niemann-Pick type C disease

Conditions

Niemann-Pick Type C Disease
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Related Conditions:

Niemann-Pick Type C Disease

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 Niemann-Pick type C disease

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 Niemann-Pick Type C Disease (NPC)

Condition
Niemann-Pick Type C Disease
Intervention / Treatment

-

Contacts and Locations

Oakland

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

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

  • * Signed informed consent
  • * Confirmed diagnosis of NPC disease
  • * Patient is unable or unwilling to take miglustat, or is, in the opinion of the investigator, unsatisfactorily treated with miglustat
  • * Male and female participants aged 4 years and older at the time of informed consent
  • * Onset of neurological symptoms from 2 to 15 years
  • * Disability level at Baseline: Ataxic disturbances with a total SARA score of ≥3 and ≤30 at Baseline
  • * Female 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 NPC disease 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)
  • * Current treatment with miglustat, provided the patient has been using the recommended dose for most of the past 12 months AND is, in the opinion of the investigator, satisfactorily treated with miglustat. Any participants receiving miglustat are required to undergo a 1-month washout period before starting study medication

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