BridgeMD
New treatment option for Type II Gaucher Disease
Official title An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
ClinicalTrials.gov ID: NCT06272149
What this study is testing
What is VGN-R08b?
VGN-R08b is an investigational medicine, being studied as a potential treatment for type ii gaucher disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 24
You may be able to join if
- 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...
You likely can't join if
- 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.
- 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...
- Clinically active infection (including HIV, HBV, HCV or syphilis).
- For those receiving enzyme replacement therapy and/or substrate reduction therapy and/or ambroxol for Gaucher disease, stable treatment ≤2 months...
- Use of strong inhibitors or inducers of cytochrome CYP3A4 or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
See the full eligibility criteria
- 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.
- 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.
- 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...
- Clinically active infection (including HIV, HBV, HCV or syphilis).
- 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.
- Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed).
- Patients with anti-AAV9 neutralizing antibody titer over 1:5.
- Brain MRI (magnetic resonance imaging) showing clinically significant abnormality considered to prevent intracisternal injection.
- Contraindication to sedation during surgery or imaging studies (PET).
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 months to 24 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.