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New drug offers hope for brain symptoms in rare gaucher disease

NCT ID NCT07675031

Disease control Sponsor: Sanofi 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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 Jun 30, 2026 · Last updated Jul 01, 2026 · Updated 1 time

Summary

This program provides early access to the experimental drug venglustat for children and adults with Type 3 Gaucher disease who have brain-related symptoms. Participants must be at least 12 years old, weigh at least 15 kg, and be stable on standard enzyme replacement therapy. The goal is to offer a potential treatment for the neurological aspects of the disease, which currently have no approved therapies.

What this could mean

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

Active substance
venglustat
What this could lead to
If venglustat works, it could offer a treatment option for the brain-related symptoms of Type 3 Gaucher disease, for which no approved therapies currently exist.
What could go wrong
This is an early-access program, not a formal trial, so data on effectiveness and safety are still limited. The drug may not work for all patients or could have side effects.

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.

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

12 years and older

Sex

Anyone

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: * ≥ 12 years of age * Clinical diagnosis of GD3 with at least 1 CNS manifestation * Body weight ≥15 kg * On a stable ERT dose for at least 6 months * Clinically stable with respect to hematologic manifestations * Clinically stable with respect to spleen and liver volume * If the patient has a history of seizures, they must be well controlled without use of medications that are strong/moderate inducers or strong/moderate inhibitors of CYP3A * Documented negative pregnancy test * Contraceptive use consistent with local regulations. Exclusion criteria: * Inability to safely swallow (or chew and swallow) a venglustat tablet * Use of any investigational drugs within the last 30 days or 5 half-lives or gene therapy at any time * Current use of any unapproved therapy for GD * Pregnant or breastfeeding females * Drug contraindications * History of major organ transplant (eg, bone marrow or liver) * History of drug and/or alcohol abuse within the last year * Patients with severe depression and/or a history of a major affective disorder in the past year * Patients with suicidal ideation or behavior prior to study. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

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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.

Type 3 Gaucher disease

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 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 ↗

  3. 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: •••••@•••••