An observational study in Gaucher Disease, Type 3, sponsored by University of Minnesota. Recruiting at 1 site in United States. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-08-04.
Sponsored by University of Minnesota · Observational
Among 92 studies in Gaucher Disease with a site in United States — 20 open to participants now, 27 under way, 2 of them phase 3. Gaucher Disease · United States.
Among the 92 studies in Gaucher Disease with a site in United States, this one is the 35th largest by planned enrollment of the 39 observational ones, is one of 3 that started in 2023, is one of 39 observational ones, reaches primary completion later than 6 of the 25 other live ones, and was the 80th registered.
177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.
This study's planned enrollment of 5 is below the median of 63 across 67 observational studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →20 of the 92 studies in Gaucher Disease with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 26 Gaucher Disease studies recruiting now, 11 (42%) set no upper age limit and 11 (42%) stop at 75 or younger; 15 take children under 18; 4 take healthy volunteers.
This study: 18 Years to 80 Years · all sexes.
The full table on the hub →92 studies; the count open now beside each. This study's row is marked.
First in 1988; this study’s year is marked. 3 started in 2023.
Median enrollment 30 across the 51 interventional studies, 60 across the 39 observational. Of the 50 that completed or were terminated, 20 (40%) have results posted.
45 of 92 have an industry lead sponsor. The 10 most frequent:
University of Minnesota is the lead sponsor of 1,216 studies on the registry; 197 are open to participants now.
Of its 136 completed or terminated interventional studies of FDA-regulated products, 95 (70%) have results posted.
University of Minnesota has 6 studies filed under Gaucher Disease — fewer than ten, so there is no comparison to draw.
5 of the studies in Gaucher Disease with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
| NCT02437396University of MinnesotaOxidative Stress and Inflammatory Biomarkers in Gaucher Disease | 2026-12-31 |
| NCT05586243University of MinnesotaMAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3) | 2026-12-31 |
| NCT05843552University of MinnesotaExtracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease | 2026-12-31 |
| NCT04411654Prevail TherapeuticsPhase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) | 2027-02 |
Among the 92 studies in Gaucher Disease with a site in United States, from their registry updates:
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 with GD3
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
This is an observational study looking at brain chemicals related to oxidative stress and inflammation using magnetic resonance spectroscopy (MRS).
Neurometabolite profile
Characterize neurometabolite profiles of up to 18 metabolites including N-acetyl aspartate (a validated biomarker of neuronal integrity), glutamate and GABA (neurotransmitters), myo-inositol, glutamine, lactate and choline (inflammation biomarkers) and glutathione (anti-oxidant) in individuals with GD3.
Time frame: baseline
Plan to share: No
No publications or documents are linked to this record.
Started Jan 2023 (actual). Primary completion is estimated for Dec 2026, 47 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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