NCT07885917Not yet recruitingPhase 2/3
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
An Open-label, Roll-over Trial to Evaluate the Long-term Safety and Tolerability of Nizubaglustat (AZ-3102) in Participants With Rare Lysosomal Disorders Who Have Participated in Previous Nizubaglustat Trials and Wish to Continue Nizubaglustat Treatment
Source: ClinicalTrials.gov · Last updated 21 Oct 2026
Brief summary (registry, in English)
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to continue nizubaglustat treatment.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 2/3
- Study type
- Interventional
- Enrollment
- 218 (estimated)
- First submitted
- 14 Oct 2026
- Start date
- 10 Dec 2026
- Primary completion date
- 1 Apr 2030
- Lead sponsor
- Azafaros B.V.
- Diseases
- Frontotemporal dementia, Gaucher disease
- NCT number
- NCT07885917
- EU CT number
- 2026-527156-12-00
- EudraCT number
- 2026-527156-12
What you commit to
- Study type: Interventional · Phase 2/3
- Planned participants: 218
- From 10 Dec 2026 to the primary completion date, 1 Apr 2030
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Use of any other treatment with a similar mode of action to nizubaglustat (GCS or NLGase inhibition), e.g., miglustat or venglustat.
Other illness
- Any medical event or condition that prevents participation in the trial based on the judgment of the investigator (e.g., severe infection and severe cognitive impairment).
Lab values
- A positive serum pregnancy test (for women of childbearing potential).
Other
- Participation in another interventional trial or non-interventional study or in an Early Access Program.
Who the study is looking for
- Have participated in a trial of nizubaglustat and wish to continue nizubaglustat treatment
- Participation is considered beneficial by the investigator
- Female participants of childbearing potential who are willing to follow the protocol-defined contraceptive requirements
- Male participants who are willing to follow the protocol-defined contraceptive requirements
Age in the registry: 4 years to - · Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.