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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, GM1 Gangliosidosis or GM2 Gangliosidosis

PHASE3RECRUITING

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 and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis.

Study details:

This is a Phase 3 randomized, double-blinded, placebo-controlled study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis. Treatment specific procedures will be described in the disease-specific subprotocol.

Individual subprotocols may have additional eligibility requirements, safety and efficacy procedures, or endpoints, which will be described in the corresponding subprotocols. For information specific to each individual subprotocol included in this trial, please refer to the corresponding, separate, clinicaltrials. gov records: Niemann-Pick type C disease NCT07082725 and GM1 gangliosidosis or GM2 gangliosidosis NCT07082543.

Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

  1. Male and female participants, aged 4 years and older with a diagnosis of the late-infantile or juvenile form of NPC disease. Detailed inclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC (NCT07082725).
  2. Male and female participants, aged 4 years and older with a diagnosis of GM1 or GM2 (Tay-Sachs, Sandhoff, or GM2AB variant disease) gangliosidosis of late-infantile/ juvenile onset. Detailed inclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx (NCT07082543).

Exclusion criteria

  1. Detailed exclusion criteria are presented in the NPC disease-specific subprotocol AZA-001-301-NPC
  2. Detailed exclusion criteria are presented in the GM1/GM2 gangliosidosis-specific subprotocol AZA-001-301-GMx

Eligibility

Age eligible for study : NA

Healthy volunteers accepted : No

Gender eligible for study: ALL

Things to know

Study dates

Study start:

Primary completion:

Study completion finish:

study type

Study type

TREATMENT

phase

Phase

    PHASE3

trial

Study ID

NCT07054515

Intervention or treatment

DRUG: AZ-3102

DRUG: Placebo

Conditions

  • Niemann-Pick Type C Disease
  • GM1 Gangliosidosis
  • GM2 Gangliosidosis

  • Condition: Niemann-Pick Type C Disease, GM1 Gangliosidosis and more

  • DRUG: AZ-3102 and other drugs

  • North Adelaide, South Australia, Australia and more

  • Sponsor: Azafaros A.G.

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Are you running this trial?

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Research sites nearby

Closest Location:

Women's and Children's Hospital

Select from list below to view details:

  • Women's and Children's Hospital

    North Adelaide, South Australia, Australia

  • Royal Melbourne Hospital

    Parkville, Victoria, Australia

  • Royal Children's Hospital Melbourne - PIN

    Parkville, Victoria, Australia

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Who is running the clinical trial?

AA

Azafaros A.G.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/ArmIntervention/Treatment
EXPERIMENTAL: Subprotocol NCT07082725: Intervention Group NPC
  • Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
  • (Recruiting)
Drug: AZ-3102, Drug: Placebo
    EXPERIMENTAL: Subprotocol NCT07082543: Intervention Group GM1/GM2
    • Participants will be randomized to receive either nizubaglustat or placebo at a 2:1 ratio (nizubaglustat:placebo).
    • (Active, not recruiting - No longer looking for participants)
    Drug: AZ-3102, Drug: Placebo

      What is the study measuring?

      Primary Outcome

      MeasureDescriptionTime Frame
      Number of participants allocated to subprotocol AZA-001-301-NPCNot SpecifiedBaseline to month 18
      Number of participants allocated to subprotocol AZA-001-301-GMxNot SpecifiedBaseline to month 18

      Secondary Outcome

      Are you running this trial?If you're a clinic or sponsor, you can claim this study.
      Claim this trial

      References

      Clinical Trials Gov: 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

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