Gaucher Disease Type 3Phase 2NCT07715084
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A Phase 2 study testing AZ-3102 in people with Gaucher Disease Type 3, run by Azafaros B.V. at 7 sites in 3 countries.
Trial summary
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3). Read more about this study
- Who is it for?
- Ages 4 to 30 years
- Conditions the study is for
- Gaucher Disease Type 3
- 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
- 20 participants
- Where
- 7 sitesin 3 countries
- Study dates
- 28 Sep 2026to Jul 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have a confirmed diagnosis of GD3 disease
- Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening
- Be aged 4-30 years old at the time of informed consent
- Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly
- Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance
- Male participants with a female partner of childbearing potential who are willing to follow contraceptive guidance
Who cannot join
- Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments
- History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results
- Body weight <10 kg at Screening
- Presence of a neurologic disease other than GD3
- Transfusion dependence
- Prior splenectomy
Show all 13Show fewer
- The presence of severe renal impairment at Screening
- Prior use of an investigational drug within 3 months before Screening
- Prior participation in a clinical study involving gene therapy or stem cell transplantation
- Current treatment, or treatment in the last 12 months before Screening, with enzyme replacement therapy (ERT) and/or substrate reduction therapy (SRT)
- A positive serum pregnancy test (for women of childbearing potential)
- Electrocardiogram (ECG) with an average Fridericia-corrected QT interval (QTcF) of >450 msec for males and >470 msec for females at Screening
- Known allergies to azasugars or any study drug excipient
Trial locations and contacts
7 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 3 countries
India3 sites
SMS Medical College Jaipur
Jaipur, 302004
Maula a Azad medical college Delhi
New Delhi, 110002
Sir Ganga Ram Hospital
New Delhi, 110060
Pakistan2 sites
University of Child Health Sciences and the Children's Hospital
Lahore
Clininal Trial Unit, Rehman Medical Institute
Peshawar
Turkey (Türkiye)2 sites
Ege Universitesi Tip Fakultesi Hastanesi
Bornova, İzmir, 35100
Gazi University Medical Faculty Hospital
Yenimahalle, 06560
About this study
Official title: 96-week, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
- Conditions
- Gaucher Disease Type 3
- Being tested
- AZ-3102
- Keywords
- Nizubaglustat
- Medical subject headings
- 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 AZ-3102 orodispersible tablets
| Study group | Role | What they receive |
|---|---|---|
| Nizubaglustat | Experimental | Participants will receive Nizubaglustat (AZ-3102) once daily during Study Period 1 (24 weeks), with a possible extension into Study Period 2 (72 weeks). Receives: Drug: AZ-3102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in hemoglobin levels from Baseline
Measured: 24 weeks
Change in platelet count from Baseline
Measured: 24 weeks
Change in total spleen volume from Baseline
Assessed using magnetic resonance imaging (MRI)
Measured: 24 weeks
Secondary outcomes (8)
Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Measured: 24 weeks
Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline
Scale ranges from 1 to 7, where a higher score equals a worse outcome.
Measured: 24 weeks
Clinical Global Impression of Change (CGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
Measured: 24 weeks
Participant/Caregiver Global Impression of Change (PGI-C) score
Scale ranges from 1 to 7, where a 1 equals improvement and 7 equals a deterioration.
Measured: 24 weeks
Peak plasma concentration (Cmax) of Nizubaglustat
Measured: 24 weeks
Area under the plasma concentration versus time curve (AUC0-24) for Nizubaglustat
Measured: 24 weeks
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylceramide (GlcCer) C16:0 and C18:0
Measured: 24 weeks
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylsphingosine (glcSph)
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2026
- Study start28 Sep 2026 (estimated)
- Primary completionJan 2028 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease Type 3Studies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 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
- NCT07715084
- Sponsor's protocol ID
- AZA-001-202-GD3
- Status last verified
- Jul 2026
- First submitted
- 8 Jun 2026
- Last updated
- 20 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 96-week, Open-label, Phase 2 Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
- ClinicalTrials.gov ID
- NCT07715084
- Acronym / sponsor ID
- AZA-001-202-GD3
- Conditions
- Gaucher Disease Type 3
- Keywords
- Nizubaglustat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Azafaros B.V.Industry (pharma, biotech, devices)
- Study start
- 28 Sep 2026 (estimated)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 20 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nizubaglustat | Experimental | Drug: AZ-3102Participants will receive Nizubaglustat (AZ-3102) once daily during Study Period 1 (24 weeks), with a possible extension into Study Period 2 (72 weeks). |
Who may qualify
- Ages
- 4 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Have a confirmed diagnosis of GD3 disease
- Have at least 2 impaired domains in the Modified Severity Scoring Tool (mSST) at Screening
- Be aged 4-30 years old at the time of informed consent
- Have the following symptoms of GD3 identified at Screening: GD-related anemia, thrombocytopenia, splenomegaly
- Female participants of childbearing potential who are sexually active and willing to follow contraceptive guidance
Key exclusion criteria (13 listed)
- Any condition at Screening or Baseline that, in the opinion of the Principal Investigator, could interfere with study assessments
- History of medical conditions other than GD3 that, in the opinion of the Principal Investigator, could confound scientific rigor or interpretation of results
- Body weight <10 kg at Screening
- Presence of a neurologic disease other than GD3
- Transfusion dependence
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AZ-3102Daily oral intake of AZ-3102 orodispersible tablets | Drug | Nizubaglustat |
Primary outcome
Change in hemoglobin levels from Baseline
Time frame: 24 weeks
Change in platelet count from Baseline
Time frame: 24 weeks
Change in total spleen volume from Baseline
Time frame: 24 weeks
Assessed using magnetic resonance imaging (MRI)
8 secondary outcomes
Change in the Clinical Global Impression of Severity (CGI-S) score compared to Baseline
Time frame: 24 weeks
Change in the Participant/caregiver Global Impression of Severity (PGI-S) score compared to Baseline
Time frame: 24 weeks
Clinical Global Impression of Change (CGI-C) score
Time frame: 24 weeks
Participant/Caregiver Global Impression of Change (PGI-C) score
Time frame: 24 weeks
Peak plasma concentration (Cmax) of Nizubaglustat
Time frame: 24 weeks
Area under the plasma concentration versus time curve (AUC0-24) for Nizubaglustat
Time frame: 24 weeks
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylceramide (GlcCer) C16:0 and C18:0
Time frame: 24 weeks
Change in plasma and cerebrospinal fluid (CSF) concentrations of glucosylsphingosine (glcSph)
Time frame: 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AZ-3102 in people with Gaucher Disease Type 3, run by Azafaros B.V. at 7 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 20 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in India, Pakistan, Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patient Advocacy Representative (Please reach out by email, info@azafaros.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in hemoglobin levels from Baseline (time frame: 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2026.