- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2032
- U.S. locations
- 7
- States / cities
- Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
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- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Interventions
- UX003
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 4
- States / cities
- Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Interventions
- UX003
- Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 1 Day to 5 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 3
- States / cities
- Washington D.C., District of Columbia • New York, New York • Salt Lake City, Utah
- Conditions
- Mucopolysaccharidosis Type 7
- Interventions
- UX003
- Drug
- Lead sponsor
- Joyce Fox
- Other
- Eligibility
- Male only
- U.S. locations
- 1
- States / cities
- New Hyde Park, New York
- Conditions
- MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
- Interventions
- UX003, Placebo
- Drug · Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 5 Years to 35 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 4
- States / cities
- Oakland, California • Orange, California • Miami, Florida + 1 more