Mucopolysaccharidosis VII Disease Monitoring Program

Part of paid clinical trials in Orange, California.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT03604835
Status
Recruiting
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Conditions

  • MPS 7
  • MPS VII
  • Mucopolysaccharidosis VII
  • Sly Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • No Intervention — OTHER
    Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.

Study Details

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

Key Dates

Start date
Jan 29, 2018
Status verified
Jun 2026
Primary completion
Apr 30, 2032
Completion
Apr 30, 2032

Study Design

Enrollment
50 participants (estimated)

Arms

  • Arm: Patients with MPS VII receiving vestronidase-alfa
    via prescription, or early access/ compassionate use program
  • Arm: Patients with MPS VII not receiving vestronidase-alfa
    no treatment or treatment other than vestronidase alfa

Primary Outcome Measure

Clinical Course of MPS VII Disease [ Time Frame: 10 years ]

Central Contacts

Locations (7)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange CountyOrangeCalifornia92868-
Children's National Health SystemWashington D.C.District of Columbia20010-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of MichiganAnn ArborMichigan48109-
New York University Langone Medical CenterNew YorkNew York10016-
University of Utah Medical CenterSalt Lake CityUtah84112-
Seattle Children's HospitalSeattleWashington98105-

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County· Orange, CAChildren's National Health System· Washington D.C., DCAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILUniversity of Michigan· Ann Arbor, MINew York University Langone Medical Center· New York, NYUniversity of Utah Medical Center· Salt Lake City, UT

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