Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01609062
• Date of registration: 24/05/2012
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
• Scientific 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)
• Date of first enrolment: April 2012
• Target sample size: 25
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01609062
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Canada, Germany, United Kingdom, United States

Contacts

• Name: Adam Shaywitz, MD PhD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion criteria

Inclusion Criteria:

- Is willing and able to provide written, signed informed consent (or patient's legally
authorized representative) after the nature of the study has been explained and prior
to performance of any research- related procedure. Also, patients who do not meet
country and local age requirements for informed consent must be willing and able to
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 performance of any research-related procedure.

- Has documented clinical diagnosis of Morquio A Syndrome (MPS IVA) based on clinical
signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte
N-acetylgalactosamine-6-sulfatase (GALNS) enzyme activity or genetic testing
confirming diagnosis of MPS IVA.

- Is at least 7 years of age

- Is able to walk = 200 meters as assessed by the 6-minute Walk Test (6MWT)

- If sexually active, is willing to use an acceptable method of contraception while
participating in the study

- If female of childbearing potential, must have a negative pregnancy test at the
Screening Visit and be willing to have additional pregnancy tests during the study

- Is willing and able to perform all study procedures, including cardiopulmonary
exercise testing (CPET)

Exclusion Criteria:

- Inability to perform an exercise test due to limited mobility

- Body weight greater than 95 kg at Screening

- Severe, untreated sleep apnea as measured during Screening with a home sleep testing
device

- Patients with a history of, or current condition of sleep apnea or sleep disordered
breathing under adequate treatment may be enrolled if approved by the medical monitor.

- Requirement for supplemental oxygen

- Use of ventilator assistance in the 3 months prior to study entry

- Use of positive airway pressure (continuous positive airway pressure, CPAP, or bilevel
airway pressure) for treatment of sleep apnea or sleep disordered breathing is allowed
if settings have been stable for at least 1 month prior to study entry, and is
approved by the medical monitor.

- Has a concurrent disease or condition, including but not limited to, symptomatic
cervical spine instability, clinically significant spinal cord compression, or severe
cardiac disease that would interfere with study participation, or pose a safety risk,
as determined by the Investigator

- Has previous hematopoietic stem cell transplant (HSCT)

- Has received previous treatment with BMN 110

- Has a known hypersensitivity to BMN 110 or its excipients

- Has had major surgery within 3 months prior to study entry or is planning to have a
major surgery during the duration of the study

- Use of any other investigational product (IP) or investigational medical device within
30 days prior to the beginning of the Screening Period or requires any investigational
agent prior to completion of all scheduled study assessments

- Is pregnant or breastfeeding during the Screening Period or planning to become
pregnant (self or partner) at any time during the study

- Has a concurrent disease or condition that may interfere with study participation or
safety, and/or ability to perform study procedures as determined by the Investigator

- Has any condition that, in the view of the Investigator, poses a safety risk to the
patient

- Has any condition that, in the view of the Investigator, places the patient at high
risk of poor treatment compliance or of not completing the study



Age minimum: 7 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis IVA

Morquio A Syndrome

MPS IVA

Intervention(s)

Drug: BMN 110

Primary Outcome(s)

Safety Evaluation [Time Frame: Entire Study Period, up to 192 weeks or ETV (early termination visit)]

Secondary Outcome(s)

Muscle Strength Testing (MST) - Elbow Flexion Test [Time Frame: Baseline, Week 25 and 52]

3-minute Stair Climb Test (3MSCT) [Time Frame: Baseline, Week 12, 24, and 52]

Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise [Time Frame: Baseline, Week 25 and 52]

Adolescent Pediatric Pain Tool (APPT) - Pain Intensity [Time Frame: Baseline, Week 12, 24, and 52]

Muscle Strength Testing (MST) - Knee Extension Test [Time Frame: Baseline, Week 25 and 52]

Cardiopulmonary Exercise Testing (CPET) - O2 Pulse [Time Frame: Baseline, Week 25 and 52]

Cardiopulmonary Exercise Testing (CPET) - Peak Workload [Time Frame: Baseline, Week 25 and 52]

Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency [Time Frame: Baseline, Week 25 and 52]

Normalized Urine Keratan Sulfate (uKS) [Time Frame: Baseline, Week 12, 24, and 52]

Muscle Strength Testing (MST) - Knee Flexion Test [Time Frame: Baseline, Week 25 and 52]

Respiratory Function Test (MVV and FVC) [Time Frame: Baseline, Week 12, 24, and 52]

6-minute Walk Test (6MWT) [Time Frame: Baseline, Week 12, 24, and 52]

Secondary ID(s)

MOR-008

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 01/02/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01609062