A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injectio

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 3

Inclusion Criteria

Individuals eligible to participate in this study must meet all of the following inclusion criteria:
• Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing confirming diagnosis.
• Elevated uGAG excretion at a minimum of 2-fold over the mean normal for age.
• Between 5 and 30 years of age, inclusive.
• Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
• Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study.
• Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have had total hysterectomy, have been in menopause for at least two years, or have had tubal ligation at least one year prior to Screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
• Has undergone a successful bone marrow or stem cell transplant or has any degree of detectable chimaerism with donor cells.
• Any hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, place the subject at increased risk for adverse effects.
• Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
• Use of any investigational product (drug or device or combination product) within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments at any time during the study.
• Has a condition of such severity and acuity, in the opinion of the Investigator, that it warrants immediate surgical intervention or other treatment or may not allow safe study participation.
• Has a concurrent disease or condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or affect safety.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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