MOR-008 Safety & Effects of BMN110 in Patients with Morquio A Syndrome
Research type
Research Study
Full title
A Randomized, Double-Blind, Pilot Study of the Safety and Physiological Effects of Two Doses of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)
IRAS ID
99666
Contact name
Simon Jones
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2011-005682-20
ISRCTN Number
n/a
Research summary
The purpose of this study is to evaluate the safety of 2 doses of BMRN 110 in 25 patients greater than 7 years of age with Morquio A Syndrome (MPS IVA) who are able to walk at least 200 meters on the 6WMT.
This study will evaluate the effect of both doses on 6MWTs and 3MSC tests,
investigate changes in physiological correlation by assessment of exercise on a cardiopulmonary exercise test (CPET), lung function on RFTs, pain (Adolescent Pediatric Pain Tool, APPT) and muscle strength tests (MST). The PK of both doses of BMN 110 will be assessed. This study has a 4-week screening period and a 27-week treatment period. The patients will be split into 2 groups.
Group A will perform all study procedures, including the CPET and will be randomised in a ratio of 2:1 to receive either 2.0 mg/kg/week of BMN 110 or 4.0 mg/kg/week of BMN 110. Enrolment for group A will be completed prior to the enrolment of Group B. 10 patients from Group A will be enrolled in Cohort B upon completion of enrolment. Group B will perform all study procedures, except for CPET and will be randomized 1:1 to receive either 2.0 mg/kg/week of BMN 110 or 4.0 mg/kg/week of BMN 110.
Screening Period. Radiograph of the cervical spine or a neck/spine MRI, medical history; a physical exam, anthropometric measurements; weight, and vital sign and measurements
Weeks 0 and 24.Physical examination (including assessment of skeletal deformities
Week 24 (or the Early Termination Visit [ETV]) Overnight monitoring with a home sleep testing device, ECG, and ECHO.
Week 26 (or ETV)A urine pregnancy test.
Week 24 (or ETV) Patient self-rating (Patient Impression Questionnaire [PIQ])
Weeks 12 and 24 (or ETV)The 6MWT and 3MSC test, pain scores, and RFTs.
Weeks 12 and 24. Biochemical markers of bone and cartilage metabolism and blood inflammatory biomarkers.
Weeks 12 and 26 (or ETV) Clinical laboratory.
Weeks 1, 2, 4, 6, 12, and 24 (or ETV) Assessments of plasma and urine GAG and urine creatinine.
Weeks 2, 4, 6, 12, and 24 (or ETV). Assessment of immunogenicity.
Weeks 0 and 23. Blood samples for PK analysis from all patients.
Study drug infusion, vital signs, AEs and concomitant medications will be assessed weekly. CPET and MST will each be performed on separate days at Week 25 (+ 14-day window) (or ETV).REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
12/YH/0126
Date of REC Opinion
19 Mar 2012
REC opinion
Further Information Favourable Opinion