Registered outcomes13

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

1 to 13 of 13

List	Outcome	Time frame	Description
Primary	Safety Evaluation	Entire Study Period, up to 192 weeks or ETV (early termination visit)	The primary objective of the study is to evaluate the safety of weekly infusions of BMN 110; the safety variables included Adverse Events (AEs). The primary outcome measure data is presented in more detail under the Adverse Events section.
Secondary	6-minute Walk Test (6MWT)	Baseline, Week 12, 24, and 52	Change from baseline to Week 12, 24, and 52 as measured in distance walked (meters) in 6MWT.
Secondary	3-minute Stair Climb Test (3MSCT)	Baseline, Week 12, 24, and 52	Change from baseline to Week 12, 24, and 52 as measured in speed (stairs/min) in 3MSCT.
Secondary	Respiratory Function Test (MVV and FVC)	Baseline, Week 12, 24, and 52	Respiratory Function was assessed by spirometry in accordance with American Thoracic Society standards. Percent change from baseline to Week 12, 24, and 52 as measured by Maximum Voluntary Ventilation (MVV, L/min) Percent change from baseline to Week 12, 24, and 52 as measured by Forced Vital Capacity (FVC, L)
Secondary	Normalized Urine Keratan Sulfate (uKS)	Baseline, Week 12, 24, and 52	Urinary KS was measured by a quantitative method and normalized using the sample urinary creatinine measurement. Percent change from baseline to Week 12, 24, and 52 in normalized urine keratan sulfate (ug/mg).
Secondary	Cardiopulmonary Exercise Testing (CPET) - Duration of Exercise	Baseline, Week 25 and 52	Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Change from baseline to Week 25 and 52 as measured by the CPET Duration of Exercise (min)
Secondary	Cardiopulmonary Exercise Testing (CPET) - Peak Workload	Baseline, Week 25 and 52	Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Peak workload (watt)
Secondary	Cardiopulmonary Exercise Testing (CPET) - O2 Pulse	Baseline, Week 25 and 52	Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET O2 pulse (ml/beat)
Secondary	Cardiopulmonary Exercise Testing (CPET) - Aerobic Efficiency	Baseline, Week 25 and 52	Subjects performed maximal incremental exercise testing using an electronically braked upright cycle ergometer. Cycle ergometry is a method of CPET that may be feasible in subjects who have orthopedic, peripheral vascular, or neurological limitations that restrict weight bearing. Percent change from baseline to Week 25 and 52 as measured by the CPET Aerobic Efficiency (ml/watt). Note that decline in Aerobic Efficiency translate into an improvement
Secondary	Muscle Strength Testing (MST) - Knee Extension Test	Baseline, Week 25 and 52	Change from baseline to Week 25 and 52 as measured by the peak force in MST knee extension test (newton meters).
Secondary	Muscle Strength Testing (MST) - Knee Flexion Test	Baseline, Week 25 and 52	Percent change from baseline to Week 25 and 52 as measured by the peak force in MST knee flexion test (newton meters).
Secondary	Muscle Strength Testing (MST) - Elbow Flexion Test	Baseline, Week 25 and 52	Percent change from baseline to Week 25 and 52 as measured by the peak force in MST elbow flexion test (newton meters).
Secondary	Adolescent Pediatric Pain Tool (APPT) - Pain Intensity	Baseline, Week 12, 24, and 52	The APPT is a validated, multidimensional tool to evaluate pain in children, adolescents, and young adults. The complete APPT is measured in three parts - Part 1 of the APPT scale determines the subject's pain locations using a body template. Part 2 of the APPT scale determines the intensity of the pain using a 10 cm visual analog scale (VAS) with the lowest point of the scale (0) labeled No Pain and the highest point on the scale (10) labeled Worst Possible Pain. Intermediate regions of the sale were labeled with 3 intermediate descriptors (Little Pain, Medium Pain, and Large Pain). Part 3 of the APPT scale characterizes the pain by tracking the number and percentage of words selected by subjects to describe their pain from a total of 57 choices. Part 2 corresponds most closely to other typically used pain scales (based on VAS) and for this reason the results from Part 2 are presented here. Change from baseline to Week 12, 24, and 52 in pain intensity.

Source ClinicalTrials.govtrials-2026-11-12-0.5.0

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this list from software.

Twenty rows a page, as JSON. No key.

GET https://conductscience.org/api/trials/v1/NCT01609062/registered

API reference For AI assistants