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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT07082725
Registration number
NCT07082725
Ethics application status
Date submitted
27/06/2025
Date registered
24/07/2025
Date last updated
3/09/2025
Titles & IDs
Public title
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)
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Scientific title
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
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Secondary ID [1]
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2024-515778-28-00
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Secondary ID [2]
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AZA-001-301-NPC
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Niemann-Pick Type C Disease
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Condition category
Condition code
Metabolic and Endocrine
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Nizubaglustat
Treatment: Drugs - Placebo
Experimental: Nizubaglustat - Once daily oral dispersible tablets
Placebo comparator: Placebo - Matching placebo
Treatment: Drugs: Nizubaglustat
AZ-3102
Treatment: Drugs: Placebo
Matching placebo
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
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Assessment method [1]
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Total SARA comprises eight categories with a cumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia)
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Timepoint [1]
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Baseline to month 18
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Primary outcome [2]
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Change from baseline in functional SARA score
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Assessment method [2]
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Functional SARA uses an abbreviated scale that scores 0 to 16, with higher scores indicating more severe impairment
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Timepoint [2]
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Baseline to month 18
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Secondary outcome [1]
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Change from baseline in SARA score for gait/posture
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Assessment method [1]
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Assessed on a 9-point scale with higher scores indicating inability to complete the task
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Timepoint [1]
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Baseline to months 6, 12, and 18
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Secondary outcome [2]
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Change from baseline in SARA score for speech
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Assessment method [2]
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Assessed on a 7-point scale with higher scores indicating unintelligible speech/anarthria
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Timepoint [2]
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Baseline to months 6, 12, and 18
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Secondary outcome [3]
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Change from baseline in SARA score for kinetics
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Assessment method [3]
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Assessed on a 5-point scale with higher scores indicating more severe impairment
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Timepoint [3]
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Baseline to months 6, 12, and 18
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Secondary outcome [4]
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Change from baseline in Vineland Adaptive Behavior Scale (VABS)
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Assessment method [4]
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Assesses four domains of function: communication, daily living skills, socialization, and motor skills
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Timepoint [4]
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Baseline to months 6, 12, and 18
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Secondary outcome [5]
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Change from baseline in Penetration-Aspiration Scale (PAS)
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Assessment method [5]
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Assessed on an 8-point ordinal scale, with 1 representing the lowest and 8 the highest/most severe score
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Timepoint [5]
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Baseline to months 6, 12, and 18
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Secondary outcome [6]
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Change from baseline in 9-Hole Peg Test (9-HPT-D)
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Assessment method [6]
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Assessed by the number of pegs per second placed in a 50-second, 9-HPT using the dominant hand
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Timepoint [6]
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Baseline to months 6, 12, and 18
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Secondary outcome [7]
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Change from baseline in NPC-Clinical Severity Scale (NPC-CSS)
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Assessment method [7]
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Scores can range from 0 to 61, with a higher score indicating more severe clinical impairment
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Timepoint [7]
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Baseline to months 6, 12, and 18
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Secondary outcome [8]
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Change from baseline in Goal Attainment scale (GAS)
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Assessment method [8]
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Assesses individual goals achieved during the study
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Timepoint [8]
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Baseline to months 6, 12, and 18
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Secondary outcome [9]
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Change from baseline in Clinician Global Impression of Change (CGI-C)
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Assessment method [9]
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Assessed on a 7-point scale with higher scores indicating lower improvement
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Timepoint [9]
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Baseline to months 6, 12, and 18
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Secondary outcome [10]
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Change from baseline in Participant/Caregiver Global Impression of Change (PGI-C)
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Assessment method [10]
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Assessed on a 7-point scale with higher scores indicating lower improvement
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Timepoint [10]
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Baseline to months 6, 12, and 18
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Secondary outcome [11]
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Change from baseline in Seizure frequency and duration, as per the seizure diary
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Assessment method [11]
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Timepoint [11]
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Baseline to months 6, 12, and 18
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Secondary outcome [12]
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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
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Assessment method [12]
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Timepoint [12]
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Baseline to month 18
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Secondary outcome [13]
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Time-to-event comparison between nizubaglustat and placebo over the study duration for pre-defined detrimental events of an increase in functional SARA of =1.5 points
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Assessment method [13]
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Timepoint [13]
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Baseline to month 18
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Secondary outcome [14]
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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
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Assessment method [14]
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Timepoint [14]
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Baseline to month 18
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Secondary outcome [15]
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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
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Assessment method [15]
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Timepoint [15]
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Baseline to month 18
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Secondary outcome [16]
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Maximum observed plasma concentration (Cmax)
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Assessment method [16]
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Timepoint [16]
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Baseline and months 1,18, and 21
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Secondary outcome [17]
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Time to Cmax (Tmax)
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Assessment method [17]
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Timepoint [17]
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Baseline and months 1,18, and 21
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Secondary outcome [18]
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Plasma trough concentration (Ctrough)
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Assessment method [18]
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Timepoint [18]
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Baseline and month 1
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Secondary outcome [19]
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Area under the plasma concentration-time curve from time of dosing (zero) to 24 hours post-dose (AUC0-24) at baseline
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Assessment method [19]
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Timepoint [19]
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Baseline (Day 1)
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Secondary outcome [20]
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Accumulation ratio for Cmax
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Assessment method [20]
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Timepoint [20]
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Baseline and months 1,18, and 21
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Secondary outcome [21]
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Change from baseline in plasma concentration of glucosylceramide (GlcCer) C16:0; C18:0
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Assessment method [21]
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Timepoint [21]
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Baseline and months 1, 6, 12, and 18
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Eligibility
Key inclusion 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
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Minimum age
4
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/06/2025
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
4/11/2027
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Actual
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Sample size
Target
72
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA,VIC
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Recruitment hospital [1]
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Women's and Children's Hospital - North Adelaide
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Recruitment hospital [2]
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Royal Children's Hospital Melbourne - PIN - Parkville
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Recruitment postcode(s) [1]
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5006 - North Adelaide
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Virginia
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Country [3]
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Argentina
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State/province [3]
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Buenos Aires
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Country [4]
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Argentina
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State/province [4]
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Córdoba Province
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Country [5]
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Brazil
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State/province [5]
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Rio de Janeiro
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Country [6]
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Brazil
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State/province [6]
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Rio Grande do Sul
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Country [7]
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Brazil
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State/province [7]
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Curitiba
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Country [8]
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Canada
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State/province [8]
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Alberta
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Country [9]
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Canada
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State/province [9]
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Quebec
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Country [10]
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France
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State/province [10]
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Bouches-du-Rhône
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Country [11]
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India
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State/province [11]
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Kerala
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Country [12]
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India
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State/province [12]
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Maharashtra
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Country [13]
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India
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State/province [13]
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National Capital Territory of Delhi
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Country [14]
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India
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State/province [14]
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Rajasthan
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Country [15]
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Italy
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State/province [15]
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Milan
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Country [16]
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Portugal
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State/province [16]
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Porto District
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Country [17]
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Spain
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State/province [17]
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Barcelona
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Country [18]
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Spain
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State/province [18]
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Madrid
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Country [19]
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Switzerland
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State/province [19]
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Canton of Bern
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Country [20]
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Turkey (Türkiye)
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State/province [20]
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Adana
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Country [21]
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Turkey (Türkiye)
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State/province [21]
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Ankara
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Country [22]
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Turkey (Türkiye)
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State/province [22]
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Izmir
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Country [23]
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United Kingdom
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State/province [23]
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London
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Country [24]
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United Kingdom
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State/province [24]
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Middlesex
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Azafaros A.G.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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 Niemann-Pick type C disease
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Trial website
https://clinicaltrials.gov/study/NCT07082725
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Patient Advocacy Representative
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Address
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Country
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Phone
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Please reach out by email
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT07082725
Download to PDF