NCT06272149RecruitingEarly phase 1
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
An Exploratory Clinical Trial to Evaluate the Tolerability and Safety of VGN-R08b Via Intracerebroventricular Injection in Patients With Type II Gaucher Disease
Source: ClinicalTrials.gov · Last updated 22 Feb 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Status (registry)
- Recruiting
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- First submitted
- 17 Jul 2023
- Start date
- 1 Mar 2023
- Primary completion date
- 15 Jan 2025
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
- Diseases
- Gaucher disease
- NCT number
- NCT06272149
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 6
- From 1 Mar 2023 to the primary completion date, 15 Jan 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- For those receiving enzyme replacement therapy and/or substrate reduction therapy and/or ambroxol for Gaucher disease, stable treatment ≤2 months before enrollment.
- Use of strong inhibitors or inducers of cytochrome CYP3A4 or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
- Any type of prior gene or cell therapy.
- Immunizations (live vaccines) in the prior 4 weeks.
- Patients with anti-AAV9 neutralizing antibody titer over 1:5.
- Contraindication to sedation during surgery or imaging studies (PET).
Other illness
- Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
- Clinically active infection (including HIV, HBV, HCV or syphilis).
- Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed).
- Presence of other significant medical conditions that would create an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
Other
- Achieved independent gait.
- Severe visceral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
- Brain MRI (magnetic resonance imaging) showing clinically significant abnormality considered to prevent intracisternal injection.
Who the study is looking for
- Infants with age of ≤24 months.
- Historical diagnosis of Gaucher disease confirmed by GCase enzyme activity test, and with GBA1 biallelic mutations.
- Neurological signs and/or symptoms consistent with diagnosis of GD2.
- Parent(s)/legal guardian(s) of subject must give their consent for subject to enroll in the study.
- Parent(s)/legal guardian(s) of the subject must agree to comply with the requirements of the study, including providing disease information and support disease assessment of symptoms.
Age in the registry: 0 months to 24 months · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Xinhua Hospital Affiliated To Shanghai Jiao tong University School of Medicine Coordinator of this site? Confirm its status | Shanghai | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.