Could a daily pill replace IV infusions for gaucher type 3?
NCT ID NCT05222906
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 3 trial tests venglustat, a daily oral drug, against the standard IV treatment Cerezyme in 43 people with Gaucher disease type 3. Participants must have been on enzyme replacement therapy for at least 3 years. The goal is to see if venglustat can stabilize or improve neurological symptoms while keeping the body stable.
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 it works, this could offer a daily pill option for Gaucher type 3 patients, potentially replacing or reducing the need for regular IV infusions.
What could go wrong
This is a small, early-phase 3 trial with only 43 participants. It may not show clear benefit over the current standard treatment, and side effects are still being studied.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GAUCHER'S DISEASE TYPE III are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
47-87, boulevard de l'hôpital - Investigational Site Number: 2500003
Paris, 75013, France
-
Azienda Ospedaliera Universitaria (AOU) "Federico II" - Investigational Site Number: 3800002
Naples, 80131, Italy
-
Children's Hospital Research Institute of Manitoba - Investigational Site Number: 1240001
Winnipeg, Manitoba, R3E 3P4, Canada
-
Cukurova University Medical School Hospital-Investigational Site Number : 7920001
Adana, 01790, Turkey (Türkiye)
-
Debreceni Egyetem, Klinikai Központ, Reumatológiai Klinika - Investigational Site Number: 3480001
Debrecen, H-4032, Hungary
-
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - Investigational Site Number : 3800003
Milan, Lombardy, 20122, Italy
-
Gazi University Medical Hospital-Investigational Site Number : 7920002
Ankara, 06500, Turkey (Türkiye)
-
Hopital Necker - Investigational Site Number: 2500001
Paris, 75015, France
-
Hospital de Ninos - Investigational Site Number: 320001
Buenos Aires, 1426, Argentina
-
Investigational Site Number : 8260001
London, NW3-2PF, United Kingdom
-
Istanbul University Medical Faculty Hospital-Investigational Site Number : 7920004
Istanbul, 34093, Turkey (Türkiye)
-
Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001
Fairfax, Virginia, 22030, United States
-
National Taiwan University Hospital-Investigational Site Number: 1580001
Taipei, Taiwan, 10041, China
-
Odawara Municipal Hospital-Investigational Site Number : 3920002
Odawara, Kanagawa, 250-8558, Japan
-
Peking Union Medical College Hospital - Investigational Site Number: 1560001
Beijing, 100005, China
-
SphinCS GmbH - Investigational Site Number: 2760001
Hochheim am Main, 65239, Germany
-
Texas Oncology - Medical City Dallas Site Number : 8400008
Dallas, Texas, 75230, United States
-
The First Affiliated Hospital - Investigational Site Number: 1560002
Guangzhou, 510080, China
-
Tohoku University School of Medicine - Investigational Site Number: 3920001
Sendai, 980-8574, Japan
-
University of Iowa - Investigational Site Number: 8400002
Iowa City, Iowa, 52242, United States
-
Xinhua Hospital - Investigational Site Number: 1560004
Shanghai, 200092, China
-
Yale University School of Medicine - Investigational Site Number: 8400003
New Haven, Connecticut, 06511, United States