跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT07082725
NCT07082725招募中3 期

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.59 个研究点 分布在 14 个国家目标入组 72 人开始时间: 2025年6月30日最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
干预措施
相关药物

试验速览

阶段
3 期
状态
招募中
发起方
入组人数
72
试验地点
59
主要终点
Change from baseline in total Scale for the Assessment and Rating of Ataxia (SARA) score
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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

详细描述

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 NPC disease

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 NPC disease

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Parallel
主要目的
Treatment
盲法
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

入排标准

年龄范围
4 Years 至 —（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •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

排除标准

  • •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

研究组 & 干预措施

Placebo

Placebo Comparator

Matching placebo

干预措施: Placebo (Drug)

Nizubaglustat

Experimental

Once daily oral dispersible tablets

干预措施: Nizubaglustat (Drug)

结局指标

主要结局

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

时间窗: 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

时间窗: Baseline to month 18

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

次要结局

  • 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 NPC-Clinical Severity Scale (NPC-CSS)(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 study duration 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 and months 1, 6, 12, and 18)
  • 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 NPC-Clinical Severity Scale (NPC-CSS)(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 study duration 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 and months 1, 6, 12, and 18)

研究者

发起方
Azafaros B.V.
申办方类型
Industry
责任方
Sponsor

研究点 (59)

Loading locations...

相似试验

招募中
3 期
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
NCT07054515Azafaros B.V.147
进行中（未招募）
3 期
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
NCT07082543Azafaros B.V.75
已完成
3 期
Prophylaxis of Diarrhea in Adult Cancer Patients Receiving Targeted Cancer TherapyCancer Therapy-Related DiarrheaChemotherapy-related DiarrheaAdult Solid TumorProphylaxis of DiarrheaSymptomatic Relief of DiarrheaTargeted Therapy-related Diarrhea
NCT04538625Napo Pharmaceuticals, Inc.287
招募中
3 期
Ensartinib as Adjuvant Treatment in Anaplastic Lymphoma Kinase (ALK) Positive Non-small Cell Lung CancerNon-small Cell Lung Cancer
NCT05341583Betta Pharmaceuticals Co., Ltd.202
已完成
3 期
Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in the Treatment of Subjects With GPPGeneralized Pustular Psoriasis
NCT05352893Vanda Pharmaceuticals45