Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT02230566) enrolled 12 participants in total — three people in each of four groups. All 12 participants completed the study. The trial was testing a drug called UX003 (given by infusion at a dose of 4 mg/kg) in people with a condition that causes a substance called dermatan sulfate to build up in the body. Different groups started on UX003 at different times, with some first receiving a placebo (an inactive treatment) for 8, 16, or 24 weeks before switching to UX003. The trial measured several things, including levels of a specific substance in the urine, physical function, breathing, and movement. The reported data shows that the main outcome — the change in urinary dermatan sulfate levels after 24 weeks on UX003 — showed an average reduction of about 65% from each participant's starting level. For a broader measure of overall physical function (called the Multi-Domain Responder Index, or MDRI, which combines six different physical tests into a single score ranging from -6 meaning greatest decline to +6 meaning greatest improvement), the reported average score was 0.5. For the six-minute walk test (how far someone can walk in six minutes), the reported average change from starting point was an increase of about 21 metres. For a breathing test measuring lung capacity as a percentage of what would be expected, the reported average change was 0. For another breathing measure (maximum ventilatory ventilation), the reported data shows no numerical result was provided. For shoulder movement (range of motion measured in degrees), the reported figures across six different measurements ranged from a change of approximately -1.5 to -9.4 degrees, suggesting no increase in shoulder movement range was recorded on average. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 32063397) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Rare Disease Trial, Completed
voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice
Who may and may not be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who may be able to join
- You have been diagnosed with MPS 7 (Mucopolysaccharidosis type 7), confirmed through a blood, skin cell, or genetic test.
- Your urine test shows a certain waste product (called glycosaminoglycans) at a level at least 3 times higher than normal for your age.
- You show clear physical signs of your condition, such as an enlarged liver or spleen, stiff joints, breathing or lung problems, or difficulty moving around (but you are still able to walk).
- You are between 5 and 35 years old (inclusive).
- You are willing and able to sign a consent form, or if you are under 18 (or 16 in some regions), a parent or legal guardian can sign on your behalf.
- If you are sexually active, you are willing to use reliable contraception throughout the study and for 30 days after the last dose.
- If you could potentially become pregnant, you have a negative pregnancy test before starting and are willing to have pregnancy tests during the study.
- You have never previously been treated with the study drug UX003.
Who may not be able to join
Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.
- You have had a successful bone marrow or stem cell transplant, or any donor cells are detectable in your body.
- You had major surgery in the last 3 months, or you have major surgery planned during the study that could make participation unsafe.
- You have had an allergic reaction to the study drug or any of its ingredients in the past (confirm with trial site).
- You are currently pregnant or breastfeeding, or you or your partner are planning to become pregnant at any point during the study.
- You have taken part in another clinical trial or used an experimental drug or device within the last 30 days before screening.
- You have a condition so serious that it requires immediate surgery or urgent treatment, or that could make it unsafe for you to take part.
- You have another illness, condition, or abnormal test result that, in the doctor's opinion, could put you at risk, affect your ability to complete the study, or raise additional safety concerns.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial was run
These are the locations recorded on the registry, and they are a historical record: this trial is not enrolling, so they are not places you can join it.
Primary endpoints
European Union (EU) and Rest of World: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate (DS) at UX003 Treatment Week 24
Other options
Data last synced from ClinicalTrials.gov: 18 August 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.