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

NCT ID NCT07715084

Not yet recruiting Disease control Sponsor: Azafaros B.V. Source: ClinicalTrials.gov ↗

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.

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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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• 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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