A Phase 2 interventional study of AZ-3102 in Gaucher Disease Type 3, sponsored by Azafaros B.V.. Not yet recruiting at 7 sites in 3 countries. Open to participants aged 4 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-11-12.
Sponsored by Azafaros B.V. · Phase 2, Interventional, and Treatment
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Among 13 studies in Gaucher Disease with a site in India — 2 open to participants now, 2 under way, none of them phase 3. Gaucher Disease · India.
Among the 13 studies in Gaucher Disease with a site in India, this one is the only one that started in 2027, is one of 2 in phase 2, and was the 13th registered.
177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.
Browse Gaucher Disease studies →2 of the 13 studies in Gaucher Disease with a site in India are open now. Recruiting first, the newest first:
Genzyme, a Sanofi Company · Sites in India, United States, Argentina and 49 more countries
A study can be open while its sites in India have not opened yet or have stopped. Only those recruiting at a site in India →
Of the 26 Gaucher Disease studies recruiting now, 11 (42%) set no upper age limit and 11 (42%) stop at 75 or younger; 15 take children under 18; 4 take healthy volunteers.
This study: 4 Years to 30 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
13 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 1 · Phase 2 3 · Phase 3 4 · Phase 4 1
First in 1991; this study’s year is marked. 1 started in 2027.
This study's planned enrollment of 20 is close to the median of 20 across 100 interventional studies indexed under Gaucher Disease.
Median enrollment 37 across the 8 interventional studies, 21 across the 5 observational. Of the 9 that completed or were terminated, 6 (67%) have results posted.
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12 of 13 have an industry lead sponsor. The 6 most frequent:
Azafaros B.V. is the lead sponsor of 8 studies on the registry; 4 are open to participants now.
Azafaros B.V. has 2 studies filed under Gaucher Disease — fewer than ten, so there is no comparison to draw.
None of the studies under way reaches primary completion within a year.
Among the 13 studies in Gaucher Disease with a site in India, from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive Nizubaglustat (AZ-3102) once daily during Study Period 1 (24 weeks), with a possible extension into Study Period 2 (72 weeks).
Drug: AZ-3102
Daily oral intake of AZ-3102 orodispersible tablets
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
Assessed using magnetic resonance imaging (MRI)
Time frame: 24 weeks
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Jan 2027; primary completion for Jan 2028, 12 months later.
Start moved once since we started tracking on Sep 25, 2026: Sep 2026 → Jan 2027, on Nov 12, 2026.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Nov 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Nizubaglustat
Azafaros B.V.