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Tests treatment safety and results for Niemann-Pick Type C Disease
Official 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)
ClinicalTrials.gov ID: NCT07082725
What this study is testing
What is Nizubaglustat?
Nizubaglustat is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for niemann-pick type c disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 3: a large, late-stage study
- Time commitment: about 18 months
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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
You likely can't join if
- A history of medical conditions other than NPC disease that, in the opinion of the Principal Investigator, would confound scientific rigor or the...
- 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
See the full 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...
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Minneapolis, Minnesota, United States
- Rochester, Minnesota, United States
- Dallas, Texas, United States
- Fairfax, Virginia, United States
- Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina
- Córdoba, Córdoba Province, Argentina
- North Adelaide, South Australia, Australia
- Parkville, Victoria, Australia
- Rio de Janeiro, Rio de Janeiro, Brazil
- Porto Alegre, Rio Grande do Sul, Brazil
- Curitiba, Brazil
- Calgary, Alberta, Canada
- Edmonton, Alberta, Canada
- Montreal, Quebec, Canada
- Höchheim, Germany
- Ernākulam, Kerala, India
- New Delhi, National Capital Territory of Delhi, India
- Jaipur, Rajasthan, India
- Vellore, Tamil Nadu, India
+ 14 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 18 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Minneapolis, Minnesota, United States; Rochester, Minnesota, United States; Dallas, Texas, United States; Fairfax, Virginia, United States; Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina and 28 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.