Health Trials

Gaucher Disease, Type 3

3

Review clinical trials related to Gaucher Disease, Type 3. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

A phase I/II clinical study to evaluate the tolerance, safety and efficacy of VGN-R08b intracerebroventricular injection in patients with type III Gaucher's disease

Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 2-18Biological sex: AllType: InterventionalSponsor: Shanghai Vitalgen BioPharma Co., Ltd.Updated: May 22, 2026
Eligibility criteria

The signing of the informed consent form should be completed when the subject is... [+8]

There are other serious neurological disorders that may cause symptoms of Gauche... [+8]

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Status: Recruiting

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

Participants needed: 10
Trial details
Phase: Phase 1Age: 18-50Biological sex: FemaleType: InterventionalSponsor: University of California, San FranciscoUpdated: Mar 17, 2026Locations: 1
Eligibility criteria

Live male or female fetuses at 18 0/7 weeks to 34 6/7 weeks gestation [+4]

Fetuses with a concurrent severe structural anomaly [+10]

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Status: Recruiting

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Recent studies have has shown that magnetic resonance spectroscopy (MRS) can provide validated neuronal markers in patients with Type 1 GD (GD1) who are on stable therapy. However, alterations in neurometabolites in adult patients with GD3, who have established neurological involvement, are not well understood. The goal of this study is to characterize neurometabolite profiles in adult patients with GD3 using MRS to identify novel biomarkers that can demonstrate treatment response. Additionally, a secondary aim is to evaluate relationships between neurometabolites and disease parameters, such as genotype, enzyme levels and Gaucher disease (GD) biomarkers.

Participants needed: 5
Trial details
Age: 18-80Biological sex: AllType: ObservationalSponsor: University of MinnesotaUpdated: Jan 8, 2026Locations: 1
Eligibility criteria

All participants must be 18 years or older. [+2]

Medically unstable conditions as determined by the investigators. [+6]

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