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New pill aims to tackle rare genetic disorder affecting brain and body

NCT ID NCT07715084

Disease control Sponsor: Azafaros B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 20, 2026 · Last updated Jul 21, 2026 · Updated 1 time

Summary

This study tests an experimental drug called nizubaglustat (AZ-3102) in people aged 4 to 30 with Gaucher disease type 3, a rare genetic condition that can cause anemia, low platelets, an enlarged spleen, and neurological problems. Participants take the drug daily as a dissolvable tablet for 96 weeks. Researchers track changes in blood counts, spleen size, and overall symptom severity to see if the drug is safe and effective.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
an experimental oral drug called nizubaglustat (AZ-3102)
What this could lead to
If successful, nizubaglustat could become a new oral treatment option for people with Gaucher disease type 3, potentially improving blood counts and reducing spleen enlargement.
What could go wrong
This is a small, early-phase trial with only 20 participants and no placebo group, so results may not confirm effectiveness. The drug may also cause side effects not yet fully understood.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Jul 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 30 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type III

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Gaucher disease, type 3

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    7 sites in 3 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • 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)

  • Maula a Azad medical college Delhi

    New Delhi, 110002, India

  • SMS Medical College Jaipur

    Jaipur, 302004, India

  • Sir Ganga Ram Hospital

    New Delhi, 110060, India

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

    Lahore, Pakistan

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