Niemann-Pick Disease Type CPhase 2/3HARBORNCT07885917
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
A Phase 2/3 study testing AZ-3102 in people with Niemann-Pick Disease Type C and GM2 Gangliosidosis, run by Azafaros B.V..
Trial summary
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. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis
- What is being tested?
- AZ-3102
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 218 participants
- Where
- No sites listed yet
- Study dates
- 10 Dec 2026to 1 Apr 2030
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- A positive serum pregnancy test (for women of childbearing potential).
- 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).
- Use of any other treatment with a similar mode of action to nizubaglustat (GCS or NLGase inhibition), e.g., miglustat or venglustat.
- Participation in another interventional trial or non-interventional study or in an Early Access Program.
About this study
Official title: 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
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.
Read the detailed description
This is a multi-center, global, open-label, extension trial in participants with Niemann-Pick disease type C (NPC), GM1 gangliosidosis, GM2 gangliosidosis, or Gaucher disease type 3 (GD3) who previously participated in a clinical trial of nizubaglustat (AZ-3102) and wish to continue treatment. All participants will receive nizubaglustat once daily.
The primary objective is to assess the long-term safety and tolerability of continued daily administration of nizubaglustat. The trial will also investigate the effect of nizubaglustat on disease biomarkers and explore the effect of long-term treatment on neurological aspects of disease progression.
Participants may remain in the trial under marketing authorization has been granted for nizubaglustat in their country or until the sponsor decides to stop the trial.
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Being tested
- AZ-3102
- Keywords
- NPC, GM1, GM2, GD3, Nizubaglustat, HARBOR, Rare Lysosomal Disorders, AZ-3102, GM1 gangliosidosis, GM2 gangliosidosis, Gaucher Disease Type 3, Niemann-Pick disease type C
- Medical subject headings
- Niemann-Pick Disease, Type C, Gangliosidoses, GM2, Gangliosidosis, GM1, Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AZ-3102Drug
Daily oral intake of nizubaglustat (AZ-3102) dispersible tablets
| Study group | Role | What they receive |
|---|---|---|
| All participants will receive nizubaglustat | Experimental | Not described Receives: Drug: AZ-3102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence and severity of treatment-emergent adverse events (TEAEs)
Change in vital signs parameters over time
Measured: Through study completion, an average of 4 years
The Incidence and severity of treatment-emergent adverse events (TEAEs)
Change in laboratory safety parameters over time.
Measured: Through study completion, an average of 4 years
Secondary outcomes (4)
Change in concentrations
Absolute and percent change in the concentrations of Plasma lyso-monosialoganglioside GM1 (Lyso-GM1; for participants with GM1) since the first dose of nizubaglustat
Measured: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Plasma Lyso-GM2 (for participants with GM2) since the first dose of nizubaglustat
Measured: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Plasma N-palmitoyl-O-phosphocholine-serine (PPCS; for participants with NPC) since the first dose of nizubaglustat
Measured: Through study completion, an average of 4 years
Change in concentrations
Absolute and percent change in the concentrations of Glucosylsphingosine (GlcSph; for participants with GD3) since the first dose of nizubaglustat
Measured: Through study completion, an average of 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start10 Dec 2026 (estimated)
- Primary completion1 Apr 2030 (estimated)
- Study completion1 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Niemann-Pick Disease Type CStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 218 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Azafaros B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07885917
- Sponsor's protocol ID
- AZA-001-302-MI
- Other IDs
- 2026-527156-12-00, 2026-527156-12, Universal Trial Number (UTN)
- Acronym
- HARBOR
- Status last verified
- Oct 2026
- First submitted
- 14 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No