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Clinical Trials/NCT07082543
NCT07082543Active, not recruitingPhase 3

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

Azafaros B.V.51 sites in 15 countries75 target enrollmentStarted: June 30, 2025Last updated:
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Interventions
Drugs

Trial Snapshot

Phase
Phase 3
Status
Active, not recruiting
Sponsor
Enrollment
75
Locations
51
Primary Endpoint
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

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

Detailed Description

Please see NCT #07054515 for information on the AZA-001-301 Master Protocol

PRIMARY OBJECTIVE

The primary objective of this study is to demonstrate superior efficacy on ataxic manifestations with oral nizubaglustat dosing compared with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

SECONDARY OBJECTIVES

I. To assess additional efficacy in ataxic and non-ataxic manifestations comparing nizubaglustat dosing with placebo when administered over 18 months in participants with late-infantile and juvenile forms of GM1/GM2 gangliosidosis

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Eligibility Criteria

Ages
4 Years to — (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion 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

Exclusion Criteria

  • •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)

Arms & Interventions

Placebo

Placebo Comparator

Intervention: Placebo (Drug)

Nizubaglustat

Experimental

Once daily oral dispersible tablets

Intervention: AZ-3102 (Drug)

Outcomes

Primary Outcomes

Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score

Time Frame: Baseline to month 18

Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)

Change from baseline in functional SARA score

Time Frame: Baseline to month 18

Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment

Secondary Outcomes

  • Change from baseline in SARA score for gait/posture(Baseline to months 6, 12, and 18)
  • Change from baseline in SARA score for speech(Baseline to months 6, 12, and 18)
  • Change from baseline in SARA score for kinetics(Baseline to months 6, 12, and 18)
  • Change from baseline in Vineland Adaptive Behavior Scale (VABS)(Baseline to months 6, 12, and 18)
  • Change from baseline in Penetration-Aspiration Scale (PAS)(Baseline to months 6, 12, and 18)
  • Change from baseline in 9-Hole Peg Test (9-HPT-D)(Baseline to months 6, 12, and 18)
  • Change from baseline in Goal Attainment Scale (GAS)(Baseline to months 6, 12, and 18)
  • Change from baseline in Clinician Global Impression of Change (CGI-C)(Baseline to months 6, 12, and 18)
  • Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)(Baseline to months 6, 12, and 18)
  • Change from baseline in Seizure frequency and duration, as per the seizure diary(Baseline to months 6, 12, and 18)
  • Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in total SARA score of ≥2 points(Baseline to month 18)
  • Time-to-event comparison between nizubaglustat and placebo over the course of the study for pre-defined detrimental events of an increase in functional SARA of ≥1.5 points(Baseline to month 18)
  • Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of a decrease in PAS of ≥1 point(Baseline to month 18)
  • Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of participants leaving the study due to participant/caregiver perception of disease progression/lack of efficacy(Baseline to month 18)
  • Maximum observed plasma concentration (Cmax)(Baseline and months 1,18, and 21)
  • Time to Cmax (Tmax)(Baseline and months 1,18, and 21)
  • Plasma trough concentration (Ctrough)(Baseline and month 1)
  • Area under the plasma concentration-time curve from time of dosing (zero) to 24 hours post-dose (AUC0-24) at baseline(Baseline (Day 1))
  • Accumulation ratio for Cmax(Baseline and months 1,18, and 21)
  • Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0(Baseline to months 1, 6, 12, and 18)

Investigators

Sponsor
Azafaros B.V.
Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (51)

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