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. Author manuscript; available in PMC: 2010 Jul 14.
Published in final edited form as: J Pediatr Rehabil Med. 2010;3(2):89–100. doi: 10.3233/PRM-2010-0113

Table 1.

Summary of study populations

Study Study Time
Period (Yrs)		 Study Design Weeks of
study:
Efficacy		 Number of
Patients
Enrolled/
Completed		 Dose of
rhASB		 Age (yrs)
Mean ± SD
(range yrs)		 Sex (M/F) Height (cm)
Mean ± SD		 Number of
Patients
Withdrawn:
Time of
Withdrawal		 Baseline Urinary
GAG (µg/mg creatinine)
Mean ± SD
Phase 1/2 2001–05 Double-Blind,
Randomized, Dose
Comparison/Open-
Label Extension		 240 7/5 0.2 mg/kg
1.0 mg/kg		 12.0 ± 3.8
(7 to 16)		 4/3 107.5 ± 21.5 1 (0.2 mg/kg)
after Week 3;
1 (0.2 mg/kg)
after Week 32		 365 ± 148
Survey Study
(patients not
on ERT)		 2002–03 Cross sectional study
Of patients not on ERT		 123/121 None 13.9 ± 10
(4 to 56)		 58/63    115 ± 26 321 ± 200
Phase 2 2002–06 Open-Label,
Non-randomized		 144 10/10 1.0 mg/kg 12.1 ± 5.3
(6 to 21)		 7/3 103.7 ± 14.4 0 336 ± 116
Phase 3 2003–06 Double-Blind,
Placebo-Controlled,
Randomized/Open-
Label Extension		 96 39/38 1.0 mg/kg 13.7 ± 6.5
(rhASB)
10.7 ± 4.4
(placebo)
(5 to 29)		 13/26 104.4 ± 2.9
(rhASB)
100.3 ± 13.5
(placebo)		 1 (placebo
group) after
Week 5		 346 ± 128
(rhASB)
330 ± 114
(placebo)