Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Idursulfase Weekly (0.5 mg/kg)	Idursulfase 0.5 milligram per kilogram (mg/kg) administered once-weekly by intravenous infusion for one year (52 infusions).
Idursulfase Every Other Week (EOW) (0.5 mg/kg)	Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions).
Placebo	Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions).

Participant Flow:   Overall Study

	Idursulfase Weekly (0.5 mg/kg)	Idursulfase Every Other Week (EOW) (0.5 mg/kg)	Placebo
STARTED	32	32	32
COMPLETED	31	32	31
NOT COMPLETED	1	0	1
Death	1	0	1

Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat (ITT) population included all randomized participants.

Reporting Groups

	Description
Idursulfase Weekly (0.5 mg/kg)	Idursulfase 0.5 mg/kg administered once-weekly by intravenous infusion for one year (52 infusions).
Idursulfase EOW (0.5 mg/kg)	Idursulfase 0.5 mg/kg administered every other week by intravenous infusion for one year (26 infusions) along with placebo matching to idursulfase every other week (alternating with idursulfase) by intravenous infusion for one year (26 infusions).
Placebo	Placebo matching to idursulfase administered once-weekly by intravenous infusion for one year (52 infusions).
Total	Total of all reporting groups

Baseline Measures

	Idursulfase Weekly (0.5 mg/kg)	Idursulfase EOW (0.5 mg/kg)	Placebo	Total
Overall Participants Analyzed  [Units: Participants]	32	32	32	96
Age [1]  [Units: Years] Mean (Standard Deviation)	15.14  (6.293)	14.40  (7.019)	13.12  (6.908)	14.22  (6.729)
[1] Age at randomization.
Age, Customized [1]  [Units: Participants]
5 to 11 years	14	14	15	43
12 to 18 years	10	9	10	29
19 to 25 years	7	7	5	19
greater than equal to (>=) 26 years	1	2	2	5
[1] Age at randomization. The participant who was less than (<) 5 years old was considered for randomization to be in 5 to 11 years category. Participants who were greater than (>) 25 years old were considered for randomization to be in 19 to 25 years category.
Gender  [Units: Participants]
Female	0	0	0	0
Male	32	32	32	96
Region of Enrollment  [Units: Participants]
North America	11	11	12	34
South America	7	7	7	21
Europe	14	14	13	41

1.  Primary:

Ranked Adjusted 2-Component Composite Variable Score Based on Change From Baseline to Week 53   [ Time Frame: Baseline, Week 53 ]

2.  Secondary:

Change From Baseline in Mean Global Joint Range of Motion (JROM) Score at Week 53   [ Time Frame: Baseline, Week 53 ]

3.  Secondary:

Mean Combined Liver and Spleen Volume at Baseline   [ Time Frame: Baseline ]

4.  Secondary:

Percent Change From Baseline in Mean Combined Liver and Spleen Volume at Week 53   [ Time Frame: Baseline, Week 53 ]

5.  Secondary:

Change From Baseline in Mean Normalized Urine Glycosaminoglycan (GAG) Levels at Week 53   [ Time Frame: Baseline, Week 53 ]

6.  Secondary:

Mean Cardiac Left Ventricular Mass Index (LVMI) at Baseline   [ Time Frame: Baseline ]

7.  Secondary:

Percent Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 53   [ Time Frame: Baseline, Week 53 ]

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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