NCT02230566

Who might be able to join this trial:

• 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:

• 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.