Overview

This trial is active, not recruiting.

Conditions mps 7, sly syndrome, mucopolysaccharidosis, mps vii
Treatment ux003
Phase phase 3
Sponsor Ultragenyx Pharmaceutical Inc
Start date November 2014
End date May 2016
Trial size 12 participants
Trial identifier NCT02230566, UX003-CL301

Summary

The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
Group A will receive 4 mg/kg UX003 every other week
ux003 recombinant human beta-glucuronidase
UX003 is a sterile liquid buffered saline formulation of rhGUS
(Experimental)
Group B will receive placebo every other week for the first 8 weeks followed by 4 mg/kg UX003 every other week
ux003 recombinant human beta-glucuronidase
UX003 is a sterile liquid buffered saline formulation of rhGUS
(Experimental)
Group C will receive placebo every other week for the first 16 weeks followed by 4 mg/kg UX003 every other week
ux003 recombinant human beta-glucuronidase
UX003 is a sterile liquid buffered saline formulation of rhGUS
(Experimental)
Group D will receive placebo every other week for the first 24 weeks followed by 4 mg/kg UX003 every other week
ux003 recombinant human beta-glucuronidase
UX003 is a sterile liquid buffered saline formulation of rhGUS

Primary Outcomes

Measure
Efficacy of UX003 (US only)
time frame: 48 weeks

Secondary Outcomes

Measure
Efficacy of UX003 (primary endpoint in EU and rest of world & secondary objective in US)
time frame: 24 weeks
Safety and Tolerability of UX003
time frame: 48 weeks
Efficacy of UX003 by MDRI
time frame: 24 weeks
Efficacy of UX003 by ICR
time frame: 24 weeks
Efficacy and Tolerability of UX003
time frame: 24 weeks

Eligibility Criteria

Male or female participants from 5 years up to 35 years old.

Inclusion Criteria: - Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme assay or genetic testing - Elevated uGAG excretion at a minimum of 3-fold over the mean normal for age (at Screening) - Apparent clinical signs of lysosomal storage disease as judged by the Investigator, including at least one of the following: enlarged liver and spleen, joint limitations, airway obstruction or pulmonary problems, limitation of mobility while still ambulatory - Aged 5 - 35 years, inclusive - Willing and able to provide written 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 acceptable highly effective methods of contraception while participating in the study and for 30 days following the last dose. - 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 not experienced menarche, or have had tubal ligation at least one year prior to Screening, or who have had total hysterectomy. - Naïve to treatment with UX003 Exclusion Criteria: - Undergone a successful bone marrow or stem cell transplant or has any degree of detectable chimaerism with donor cells - Major surgery within 3 months prior to study entry or planned major surgery during the study that may not allow safe participation in the study - Presence or history of any hypersensitivity to rhGUS or its excipients that, in the judgment of the Investigator, places 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) within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments. - Presence of a condition of such severity and acuity that, in the opinion of the Investigator, warrants immediate surgical intervention or other treatment or may not allow safe participation in the study. - Concurrent disease or condition, or laboratory abnormality 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 introduce additional safety concerns.

Additional Information

Official title A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
Principal investigator Paul Harmatz, MD
Description The Blind Start is a novel design whereby subjects will be randomized to one of 4 groups, each representing a different treatment sequence, and cross over to UX003 at different pre-defined time points in a blinded manner. All groups will receive a minimum of 24 weeks treatment with 4 mg/kg UX003 every other week.
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by Ultragenyx Pharmaceutical Inc.