ClinicalTrials.gov record NCT07715084

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age4 years old to 30 years old
SexAll
LocationIndia, Pakistan, Turkey
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a 96-week, open-label Phase 2 study testing daily oral Nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3. The initial treatment period is 24 weeks, with a possible 72-week extension. The study measures blood counts, spleen volume by MRI, clinical severity scores, safety, and drug levels.

What it is studying. The study is testing the safety and effects of Nizubaglustat (AZ-3102) in people with Gaucher disease type 3. Key measurements include changes in hemoglobin, platelet count, and total spleen volume at 24 weeks; clinical global impression and participant/caregiver impression scores; drug levels in blood; and changes in specific disease-related molecules in plasma and cerebrospinal fluid (CSF).

What the researchers hope to learn. Scientific goals: to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in Gaucher disease type 3 by measuring blood counts, spleen volume, clinical severity scales, pharmacokinetic parameters, and levels of glucosylceramide and glucosylsphingosine in plasma and CSF.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • AZ-3102

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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

Exclusion criteria

  • 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
  • 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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants take daily oral Nizubaglustat (AZ-3102) tablets for an initial 24-week period with a possible 72-week extension (total 96 weeks). Study visits include blood tests (for hemoglobin, platelet count and drug levels), MRI scans to measure spleen volume, and clinician- and participant/caregiver-rated questionnaires. Because cerebrospinal fluid (CSF) concentrations are measured, participants may undergo CSF collection (spinal tap). The registry does not provide a detailed visit schedule; safety monitoring and follow-up are performed during the study.

How the study is structured. Open-label, single-group Phase 2 interventional study lasting 96 weeks total. Participants take daily oral AZ-3102 during a first 24-week treatment period and may continue in a 72-week extension. There is no placebo or masking; all enrolled participants receive the study drug. Main outcomes are assessed at 24 weeks with continued monitoring during the extension.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in India, Pakistan, Turkey. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Gaucher disease

Clinical trials64 trials listed for Gaucher diseaseEvery trial the registry lists for Gaucher disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Gaucher disease researchLatest monthly update: September 2026. Includes: Platelet release. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 28, 2026 (estimated)
Primary completion
January 2028 (estimated)
Study completion
July 2029 (estimated)
Target enrollment
20 participants (estimated)
Last updated on registry
Jul 20, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Azafaros B.V.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (7)

  • SMS Medical College Jaipur

    Jaipur, 302004 · India

  • Maula a Azad medical college Delhi

    New Delhi, 110002 · India

  • Sir Ganga Ram Hospital

    New Delhi, 110060 · India

  • University of Child Health Sciences and the Children's Hospital

    Lahore · Pakistan

  • Clininal Trial Unit, Rehman Medical Institute

    Peshawar · Pakistan

  • Ege Universitesi Tip Fakultesi Hastanesi

    Bornova, İzmir, 35100 · Turkey (Türkiye)

  • Gazi University Medical Faculty Hospital

    Yenimahalle, 06560 · Turkey (Türkiye)

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
at Screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
Baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Principal Investigator
The doctor or scientist responsible for running the study at a site or overall.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Electrocardiogram
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this website; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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