A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome)

Conditions: Mucopolysaccharidosis Type IV A
MedDRA version: 9.1Level: LLTClassification code 10028095Term: Mucopolysaccharidosis IV

Registration Number: EUCTR2008-007365-23-GB

Lead Sponsor: BioMarin Pharmaceutical Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: Not specified

Inclusion Criteria

-Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay.
-Willing and able to provide written, signed informed consent, or in the case of subjects under the age of 16 years, 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.
-Between 5 and 18 years of age, inclusive.
-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 been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.
-Willing to perform all study procedures as physically possible.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Previous hematopoietic stem cell transplant (HSCT).
-Has known hypersensitivity to BMN 110 or its excipients.
-Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
-Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
-Concurrent disease or condition that would interfere with study participation or safety, including, but not limited to, symptomatic cervical spine instability.
-Any condition that, in the view of the Principal Investigator (PI), places the subject at high risk of poor treatment compliance or of not completing the study.