Trial Outcomes & Findings for Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD) (NCT NCT00418561)

Last Updated: 2021-06-25

Results Overview

An adverse event (AE) is any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant, participating in a clinical study with study drug, regardless of causal relationship. TEAEs were AEs occurred after study drug administration that were absent before treatment or that worsened relative to pre-treatment state, up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks) completed.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

13 participants

Primary outcome timeframe

From study drug administration up to Week 28

Results posted on

2021-06-25

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Overall Study STARTED	4	5	4
Overall Study COMPLETED	4	4	4
Overall Study NOT COMPLETED	0	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Overall Study Participant's (guardian's) decision	0	1	0

Baseline Characteristics

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Total n=13 Participants Total of all reporting groups
Age, Continuous	36.25 months STANDARD_DEVIATION 9.32 • n=5 Participants	41.80 months STANDARD_DEVIATION 10.13 • n=7 Participants	30.75 months STANDARD_DEVIATION 7.27 • n=5 Participants	36.69 months STANDARD_DEVIATION 9.59 • n=4 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	8 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	5 Participants n=4 Participants

PRIMARY outcome

Timeframe: From study drug administration up to Week 28

Population: Intent-to-treat (ITT) population included all participants who received at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	4 participants	5 participants	4 participants

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: ITT

GMFM was measured using GMFM-88 item scores and summed to calculate a total GMFM-88 score. For each GMFM-88 item, the score was between 0 (minimal) to 3 (maximum). The total GMFM-88 score was between 0 (minimal) and 264 (maximum). The decrease in GMFM score over time indicates worsening of disease over time. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26	-2.36 percent (%) change Interval -7.62 to 2.91	-8.29 percent (%) change Interval -13.55 to -3.02	-10.90 percent (%) change Interval -16.17 to -5.64

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: ITT

Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26	24.55 percent (%) change Interval 0.44 to 48.66	-3.77 percent (%) change Interval -22.33 to 14.8	-4.32 percent (%) change Interval -28.17 to 19.53

PRIMARY outcome

Timeframe: Baseline up to Week 26

Population: ITT. Here, the number of participants analyzed are the participants evaluable for this outcome.

Number of participants with shifts between negative (value=0) and positive (value=1) values in urine sulfatide levels from baseline at Week 26 is reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=3 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=2 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=2 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine Negative(0) to negative(0)	0 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine Negative(0) to positive(1)	0 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine Positive(1) to negative(0)	0 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine Positive(1) to positive(1)	3 participants	2 participants	2 participants

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: ITT

Mullen's Scales of Early Learning is used to assess performance and learning ability in young children. The scale consisted of 144 items that had specific scoring criteria for each item. The scores were converted to T-scores with a decrease in score indicating worsening of disease. Relative change from baseline at Week 26 was calculated as percentage change from baseline divided by the age-difference in months between first and last visit. Adjusted mean and 95 percent (%) confidence intervals were reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Mullen's Scales of Early Learning at Week 26	16.28 percent (%) change Interval 0.72 to 31.84	0.26 percent (%) change Interval -15.49 to 16.02	-4.92 percent (%) change Interval -20.26 to 10.41

PRIMARY outcome

Timeframe: Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours on Day 0, 40 minutes post-dose at Week 4, Pre-dose and post-dose at 20, 40, 90 minutes, 3, 6 and 8 hours at Week 8

Population: Due to quick disappearance of rhASA from plasma, rhASA levels were not possible to report.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26

Population: Data were not available to report as ASA activity in leukocytes was presented graphically, as per planned analysis.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: ITT. Here, "N" signifies the number of participants who were evaluable for the respective category.

An electrophysiological evaluation using standard electrophysiological and electromyography to measure the speed and extent of nerve conduction and units are expressed in meters per second. Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; FH=fibular hemimelia; L LM=left lateral medial; R LM=right lateral medial; MC=medial collateral.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Nerve Conduction Velocity at Week 26 MN, Elbow Wrist: Baseline (N=4,5,4)	20.38 meters per second Standard Deviation 6.99	25.78 meters per second Standard Deviation 17.72	15.95 meters per second Standard Deviation 5.68
Change From Baseline in Nerve Conduction Velocity at Week 26 MN, Elbow Wrist: Change at Week 26 (N=4,4,4)	-4.00 meters per second Standard Deviation 1.10	2.62 meters per second Standard Deviation 4.42	-3.65 meters per second Standard Deviation 3.33
Change From Baseline in Nerve Conduction Velocity at Week 26 MN, Dig. II Wrist: Baseline (N=3,5,4)	39.83 meters per second Standard Deviation 7.22	36.82 meters per second Standard Deviation 16.64	23.30 meters per second Standard Deviation 11.99
Change From Baseline in Nerve Conduction Velocity at Week 26 MN, Dig. II Wrist: Change at Week 26 (N=3,4,4)	-11.7 meters per second Standard Deviation 5.78	-0.25 meters per second Standard Deviation 4.39	-1.33 meters per second Standard Deviation 4.72
Change From Baseline in Nerve Conduction Velocity at Week 26 PN, Dig. Ankle FH: Baseline (N=4,4,4)	20.70 meters per second Standard Deviation 9.08	32.23 meters per second Standard Deviation 21.41	14.13 meters per second Standard Deviation 5.92
Change From Baseline in Nerve Conduction Velocity at Week 26 PN, Dig. Ankle FH: Change at Week 26 (N=4,3,4)	-7.85 meters per second Standard Deviation 3.92	-2.43 meters per second Standard Deviation 1.59	-2.65 meters per second Standard Deviation 3.61
Change From Baseline in Nerve Conduction Velocity at Week 26 SN, Sensory L LM - MC: Baseline (N=4,4,4)	26.88 meters per second Standard Deviation 9.29	36.13 meters per second Standard Deviation 20.61	29.18 meters per second Standard Deviation 15.93
Change From Baseline in Nerve Conduction Velocity at Week 26 SN, Sensory L LM - MC: Change at Week 26 (N=3,3,4)	-5.57 meters per second Standard Deviation 4.18	3.70 meters per second Standard Deviation 9.79	-9.33 meters per second Standard Deviation 10.64
Change From Baseline in Nerve Conduction Velocity at Week 26 SN, Sensory R LM - MC: Baseline (N=4,4,4)	31.35 meters per second Standard Deviation 6.46	34.58 meters per second Standard Deviation 17.74	27.48 meters per second Standard Deviation 13.91
Change From Baseline in Nerve Conduction Velocity at Week 26 SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0)	NA meters per second Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.	NA meters per second Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.	NA meters per second Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: ITT.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 Baseline	0 participants	2 participants	0 participants
Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 Week 26	0 participants	2 participants	0 participants

SECONDARY outcome

Timeframe: Baseline up to Week 26

Population: ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome.

Loes scoring system is used to grade the demyelinating abnormalities on brain MRI. A total of 17 locations of the brain were scored from 0 (normal appearance) to 2 (dense appearance). The total score ranged from 0 to 34 with a score of 14 or greater being considered severe. Number of participants with any shift of score between 0 to 2 for each of the 17 locations (Parieto Occipital \[PO\]-Periventricular \[P\], Central \[C\], Subcortical \[Sc\]; Anterior Temporal \[AT\]-P, C, Sc; Frontal \[F\]-P, C, Sc; Corpus Callosum \[CC\]-Splenium \[S\], Genus \[G\]; Projection Fibers \[PF\]-Capsular interna \[CI\] ant, CI post, Brainstem \[B\]; Cerebellum \[Cb\]-Cortex, Atrophy; Basal Ganglia \[BG\]-BG, Thalamus \[T\]; Cerebral Atrophy \[CA\]-CA), are only reported.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=4 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, C: 2 to 2	3 participants	4 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, Sc: 0 to 0	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, Sc: 0 to 2	0 participants	2 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, Sc: 1 to 2	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, P: 1 to 2	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, P: 2 to 2	3 participants	4 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, C: 2 to 2	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 0 to 0	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 0 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 0 to 2	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 1 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 1 to 2	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PO, Sc: 2 to 2	3 participants	2 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, P: 0 to 2	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, P: 1 to 2	0 participants	1 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, P: 2 to 2	3 participants	3 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, C: 0 to 2	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, C: 1 to 2	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, C: 2 to 2	3 participants	4 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 0 to 0	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 0 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 0 to 2	0 participants	2 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 1 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 1 to 2	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores AT, Sc: 2 to 2	2 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, P: 0 to 2	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, P: 1 to 2	0 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, P: 2 to 2	3 participants	3 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, C: 1 to 2	1 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores F, Sc: 2 to 2	2 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, S: 1 to 0	0 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, S: 1 to 2	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, S: 2 to 0	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, S: 2 to 1	2 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, S: 2 to 2	0 participants	1 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, G: 0 to 2	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, G: 1 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, G: 1 to 2	0 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, G: 2 to 1	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CC, G: 2 to 2	2 participants	2 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI ant: 0 to 0	2 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI ant: 0 to 1	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI ant: 1 to 1	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 0 to 0	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 0 to 1	1 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 1 to 1	0 participants	2 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 1 to 2	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 2 to 1	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, CI post: 2 to 2	2 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 0 to 0	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 0 to 1	0 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 0 to 2	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 1 to 0	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 1 to 1	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 1 to 2	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores PF, B: 2 to 2	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Cortex: 0 to 0	2 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Cortex: 0 to 1	0 participants	2 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Cortex: 1 to 1	2 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Atrophy: 0 to 0	1 participants	1 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Atrophy: 0 to 1	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Cb, Atrophy: 1 to 1	2 participants	2 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, Bg: 0 to 0	2 participants	3 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, Bg: 0 to 1	0 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, Bg: 1 to 0	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, Bg: 1 to 1	2 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, T: 0 to 0	2 participants	3 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, T: 1 to 0	0 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores Bg, T: 1 to 1	2 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CA, CA: 0 to 0	1 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CA, CA: 0 to 1	0 participants	2 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CA, CA: 1 to 1	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores CA, CA: 1 to 2	2 participants	1 participants	0 participants

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: ITT. Here, "N" signifies the number of participants who were evaluable for the respective category.

PEDI is used for the clinical evaluation of functional capabilities, performance and changes in functional skills in children with disabilities. It consisted of 20 items scored on a scale from 0 (total assistance) to 5 (independent). Total score ranged from 0-100 with higher scores indicating better functioning. None, child, rehab, extensive are items in 3 domains (self-care, mobility and social functioning).

Outcome measures

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome.

Number of participants with at least 1 shift from baseline to Week 26, are reported. Abbreviations: ALT=Alanine transaminase; CK=Creatine kinase; AP=Amyloid P component; LDH=Lactate dehydrogenase.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=4 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: Low to low	2 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: Low to normal	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: Normal to low	0 participants	1 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: Normal to normal	0 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: High to low	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry ALT-Serum: High to normal	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Amylase-Serum: Normal to normal	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry AP-Serum: Normal to normal	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Calcium-Serum: Normal to normal	4 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry CK-Serum: Normal to normal	4 participants	3 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry CK-Serum: Normal to high	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry CK-Serum: High to normal	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry CK-Serum: High to high	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Creatinine-Serum: Normal to normal	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Iron-Serum: Low to normal	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Iron-Serum: Normal to low	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Iron-Serum: Normal to normal	3 participants	3 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Iron-Serum: Normal to high	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry LDH-Serum: Normal to normal	3 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry LDH-Serum: High to normal	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Magnesium-Serum: Normal to normal	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Phosphate-Serum: Normal to normal	2 participants	3 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Phosphate-Serum: Normal to high	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Phosphate-Serum: High to normal	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Potassium-Serum: Normal to normal	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Sodium-Serum: Normal to normal	4 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry Sodium-Serum: High to normal	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry T Bilirubin-Serum: Normal to normal	4 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry T Bilirubin-Serum: High to normal	0 participants	1 participants	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: ITT. Data for Cohorts 2 and 3 were not reported since there were no participants with shift from baseline to Week 26 in coagulation evaluations.

Number of participants with at least 1 shift from baseline to Week 26 are reported. The shift reported below for Cohort 1 was from low level at baseline to low level at Week 26.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Coagulation	4 participants	—	—

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: ITT. The number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome. Here, "N" signifies the number of participants who were evaluable for the respective category.

Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: CSF=Cerebrospinal fluid; NFP=Neurofilament proteins.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=4 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin CSF: Normal to normal (N=3,4,4)	0 participants	2 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin CSF: Normal to high (N=3,4,4)	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin CSF: High to high (N=3,4,4)	2 participants	2 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin index: Normal to normal (N=3,4,4)	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin index: High to high (N=3,4,4)	3 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin Serum: Low to low (N=3,4,4)	2 participants	2 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin Serum: Low to normal (N=3,4,4)	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin Serum: Normal to low (N=3,4,4)	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Albumin Serum: Normal to normal (N=3,4,4)	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Chitotriosidase CSF: Low to low (N=4,4,4)	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Chitotriosidase CSF: High to high (N=4,4,4)	3 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping NFP CSF: Normal to high (N=4,4,4)	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping NFP CSF: High to high (N=4,4,4)	3 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Sulfatide CSF: High to high (N=3,4,4)	3 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Tauprotein CSF: High to low (N=4,4,4)	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping Tauprotein CSF: High to high (N=4,4,4)	4 participants	3 participants	4 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: Abs=Absolute count; ERCS=Erythrocytes; MCHC=Mean corpuscular hemoglobin concentration; MCH=Mean cell hemoglobin.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=4 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Basophils Abs - Blood: Normal to normal (N=4,4,4)	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Eosinophil Abs - Blood: Normal to normal (N=4,4,4)	4 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology ERCS - Blood: Low to low (N=4,4,4)	1 participants	0 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology ERCS - Blood: Normal to normal (N=4,4,4)	3 participants	4 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Haemoglobin - Blood: Low to low (N=4,4,4)	1 participants	1 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Haemoglobin - Blood: Low to normal (N=4,4,4)	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Haemoglobin - Blood: Normal to low (N=4,4,4)	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Haemoglobin - Blood: Normal to normal (N=4,4,4)	1 participants	3 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Lymphocyte Abs - Blood: Low to low (N=4,4,4)	0 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Lymphocyte Abs - Blood: Normal to low (N=4,4,4)	2 participants	2 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Lymphocyte Abs - Blood: Normal to normal (N=4,4,4)	2 participants	1 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology MCHC - Blood: Normal to normal (N=4,4,4)	3 participants	3 participants	4 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology MCHC - Blood: High to normal (N=4,4,4)	1 participants	1 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology MCH - Blood: Low to low (N=4,4,4)	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology MCH - Blood: Normal to normal (N=4,4,4)	4 participants	4 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Monocytes Abs - Blood: Normal to normal (N=4,4,4)	4 participants	4 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Monocytes Abs - Blood: High to normal (N=4,4,4)	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Neutropil Abs - Blood: Normal to normal (N=4,4,4)	4 participants	4 participants	3 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Neutropil Abs - Blood: Normal to high (N=4,4,4)	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Thrombocytes - Blood: Normal to low (N=4,3,4)	1 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Thrombocytes - Blood: Normal to normal (N=4,3,4)	2 participants	0 participants	2 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology Thrombocytes - Blood: High to normal (N=4,3,4)	1 participants	3 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology T Leucocytes - Blood: Low to normal (N=4,4,4)	0 participants	0 participants	1 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology T Leucocytes - Blood: Normal to low (N=4,4,4)	2 participants	2 participants	0 participants
Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology T Leucocytes - Blood: Normal to normal (N=4,4,4)	2 participants	2 participants	3 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: ITT

The parameters analyzed in urine were albumin/protein, glucose, leucocytes, acetoacetate/ketones, nitrite and pH. Urine analysis findings were considered abnormal as judged by the investigator.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Abnormal Findings in Urine Analysis	0 participants	0 participants	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: ITT

Abnormal ECG findings were considered as clinically significant at the discretion of investigator.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings	0 participants	0 participants	0 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 26

Population: ITT. Here, "N" signifies the number of participants who were evaluable for the respective category.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Chitotriosidase at Week 26 Baseline (N=4, 5, 4)	924.0 nanomole/hour/milliliter Standard Deviation 1431	1481 nanomole/hour/milliliter Standard Deviation 1165	367.0 nanomole/hour/milliliter Standard Deviation 179.0
Change From Baseline in Chitotriosidase at Week 26 Change at Week 26 (N=4, 4, 4)	-76.0 nanomole/hour/milliliter Standard Deviation 393.9	-228 nanomole/hour/milliliter Standard Deviation 692.7	94.75 nanomole/hour/milliliter Standard Deviation 103.6

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 26

Population: ITT. Here, "N" signifies the number of participants who were evaluable for the respective category.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 NFP: Baseline (N=4, 5, 4)	5436 nanogram/milliliter Standard Deviation 3698	7848 nanogram/milliliter Standard Deviation 4510	12558 nanogram/milliliter Standard Deviation 3529
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 NFP: Change at Week 26 (N=4, 4, 4)	1134 nanogram/milliliter Standard Deviation 8098	-3505 nanogram/milliliter Standard Deviation 2553	-4020 nanogram/milliliter Standard Deviation 9061
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 GFAP: Baseline (N=4, 5, 4)	1758 nanogram/milliliter Standard Deviation 397.4	1014 nanogram/milliliter Standard Deviation 524.3	1415 nanogram/milliliter Standard Deviation 625.7
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 GFAP: Change at Week 26 (N=4, 4, 4)	330.0 nanogram/milliliter Standard Deviation 380.3	402.5 nanogram/milliliter Standard Deviation 460.5	502.5 nanogram/milliliter Standard Deviation 285.4
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 Tauprotein: Baseline (N=4, 5, 4)	1148 nanogram/milliliter Standard Deviation 143.4	1014 nanogram/milliliter Standard Deviation 530.9	1610 nanogram/milliliter Standard Deviation 543.4
Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 Tauprotein: Change at Week 26 (N=4, 4, 4)	-288 nanogram/milliliter Standard Deviation 692.3	-273 nanogram/milliliter Standard Deviation 228.7	-118 nanogram/milliliter Standard Deviation 907.6

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, Week 26

Population: ITT. Here, "N" signifies the number of participants who were evaluable for the respective category.

Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; APB=abductor pollicis brevis; EDB=extensor digitorum brevis.

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Amplitude at Week 26 MN, Wrist-APB: Baseline (N=4,5,4)	5.63 millivolts Standard Deviation 1.73	7.88 millivolts Standard Deviation 2.20	4.30 millivolts Standard Deviation 1.05
Change From Baseline in Amplitude at Week 26 MN, Wrist-APB: Change at Week 26 (N=4,4,4)	0.00 millivolts Standard Deviation 2.84	0.65 millivolts Standard Deviation 0.74	-0.35 millivolts Standard Deviation 1.66
Change From Baseline in Amplitude at Week 26 MN, Elbow-APB: Baseline (N=4,5,4)	3.85 millivolts Standard Deviation 1.23	6.34 millivolts Standard Deviation 2.71	2.65 millivolts Standard Deviation 0.60
Change From Baseline in Amplitude at Week 26 MN, Elbow-APB: Change at Week 26 (N=4,4,4)	0.15 millivolts Standard Deviation 2.05	0.33 millivolts Standard Deviation 0.57	-0.55 millivolts Standard Deviation 0.65
Change From Baseline in Amplitude at Week 26 MN, Dig. II Wrist: Baseline (N=4,5,4)	2.68 millivolts Standard Deviation 1.30	11.64 millivolts Standard Deviation 14.89	1.78 millivolts Standard Deviation 0.83
Change From Baseline in Amplitude at Week 26 MN, Dig. II Wrist: Change at Week 26 (N=4,4,4)	-0.32 millivolts Standard Deviation 1.00	3.53 millivolts Standard Deviation 6.60	-0.93 millivolts Standard Deviation 0.85
Change From Baseline in Amplitude at Week 26 PN, Ankle EDB: Baseline (N=4,4,4)	1.85 millivolts Standard Deviation 0.91	5.40 millivolts Standard Deviation 2.55	1.30 millivolts Standard Deviation 0.68
Change From Baseline in Amplitude at Week 26 PN, Ankle EDB: Change at Week 26 (N=4,3,4)	-0.72 millivolts Standard Deviation 1.66	-0.43 millivolts Standard Deviation 1.56	0.08 millivolts Standard Deviation 0.96
Change From Baseline in Amplitude at Week 26 PN, FH EDB: Baseline (N=4,4,4)	2.43 millivolts Standard Deviation 1.80	4.98 millivolts Standard Deviation 2.95	0.95 millivolts Standard Deviation 0.74
Change From Baseline in Amplitude at Week 26 PN, FH EDB: Change at Week 26 (N=4,3,4)	-1.68 millivolts Standard Deviation 2.06	-0.63 millivolts Standard Deviation 1.46	0.16 millivolts Standard Deviation 0.75
Change From Baseline in Amplitude at Week 26 SN, Sensory L LM - MC: Baseline (N=4,4,4)	5.25 millivolts Standard Deviation 6.31	41.18 millivolts Standard Deviation 51.50	1.65 millivolts Standard Deviation 2.28
Change From Baseline in Amplitude at Week 26 SN, Sensory L LM - MC: Change at Week 26 (N=4,3,4)	-2.45 millivolts Standard Deviation 4.18	0.53 millivolts Standard Deviation 21.75	-0.78 millivolts Standard Deviation 2.34
Change From Baseline in Amplitude at Week 26 SN, Sensory R LM - MC: Baseline (N=4,4,4)	5.35 millivolts Standard Deviation 5.45	50.05 millivolts Standard Deviation 56.13	1.73 millivolts Standard Deviation 2.01
Change From Baseline in Amplitude at Week 26 SN, Sensory R LM - MC: Change at Week 26 (N=0,0,0)	NA millivolts Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.	NA millivolts Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.	NA millivolts Standard Deviation NA Since there were no participants evaluable for SN, Sensory R LM - MC category at Week 26, data could not be reported.

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to Week 26

Population: Physical examination results were not summarized since data were collected in participant's listing only as planned.

Physical examination included general appearance, skin, head, ears, eyes, nose and throat, lymph nodes, heart, lungs, abdomen, extremities/joints, hip, neurological, mental status and, if appropriate, breasts, external genitalia, pelvic and rectal, and in addition weight, height and head circumference were recorded.

Outcome measures

Outcome data not reported

Adverse Events

Cohort 1

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Cohort 2

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Cohort 3

Serious events: 2 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1 n=4 participants at risk Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 participants at risk Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 participants at risk Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Metabolism and nutrition disorders Malnutrition	0.00% 0/4 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	40.0% 2/5 • Number of events 2 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	50.0% 2/4 • Number of events 2 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	0.00% 0/4 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	20.0% 1/5 • Number of events 1 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	25.0% 1/4 • Number of events 1 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)
Infections and infestations Bronchitis acute	25.0% 1/4 • Number of events 1 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	0.00% 0/5 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)	0.00% 0/4 From study drug administration up to Week 28 until evaluation (when last cohort had 26-week evaluation and data management performed within 4 weeks)

Other adverse events

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If a multicentre publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicentre Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Publication restrictions are in place

Restriction type: OTHER

Measure	Cohort 1 n=4 Participants Single dose of 25 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), thereafter, received repeated doses of 50 U/kg recombinant human Arylsulphatase A, once in every 2 weeks for a period of 26 weeks, as an intravenous (IV) infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 2 n=5 Participants Repeated doses of 100 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 30 minutes. Dosage adjustment was done monthly to account for changes in body weight.	Cohort 3 n=4 Participants Repeated doses of 200 U/kg recombinant human Arylsulphatase A (Metazym, rhASA), once in every 2 weeks for a period of 26 weeks, as an IV infusion over 60 minutes. Dosage adjustment was done monthly to account for changes in body weight.
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, None: Baseline (N=4,5,4)	6.25 units on a scale Standard Deviation 0.96	6.00 units on a scale Standard Deviation 1.41	4.75 units on a scale Standard Deviation 2.06
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, None: Change at Week 26 (N=4,4,4)	-0.75 units on a scale Standard Deviation 1.50	-1.50 units on a scale Standard Deviation 1.73	-1.50 units on a scale Standard Deviation 1.91
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Child: Baseline (N=4,5,4)	1.75 units on a scale Standard Deviation 0.96	2.00 units on a scale Standard Deviation 1.41	3.25 units on a scale Standard Deviation 2.06
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Child: Change at Week 26 (N=4,4,4)	0.75 units on a scale Standard Deviation 1.50	1.25 units on a scale Standard Deviation 1.50	0.75 units on a scale Standard Deviation 2.22
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Rehab: Baseline (N=4,5,4)	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Rehab: Change at Week 26 (N=4,4,4)	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0	0 units on a scale Standard Deviation 0
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Extensive: Baseline (N=4,5,4)	0 units on a scale Standard Deviation 0	0.20 units on a scale Standard Deviation 0.45	0 units on a scale Standard Deviation 0
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Self-care, Extensive: Change at Week 26 (N=4,4,4)	0 units on a scale Standard Deviation 0	0.25 units on a scale Standard Deviation 0.50	0.50 units on a scale Standard Deviation 0.58
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, None: Baseline (N=4,5,4)	4.25 units on a scale Standard Deviation 1.89	3.60 units on a scale Standard Deviation 1.14	5.00 units on a scale Standard Deviation 0.82
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, None: Change at Week 26 (N=4,4,4)	-0.75 units on a scale Standard Deviation 1.71	-0.50 units on a scale Standard Deviation 1.29	-1.25 units on a scale Standard Deviation 1.71
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Child: Baseline (N=4,5,4)	2.50 units on a scale Standard Deviation 1.91	2.40 units on a scale Standard Deviation 1.67	1.25 units on a scale Standard Deviation 1.26
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Child: Change at Week 26 (N=4,4,4)	0.00 units on a scale Standard Deviation 1.41	0.25 units on a scale Standard Deviation 1.26	-0.50 units on a scale Standard Deviation 0.58
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Rehab: Baseline (N=4,5,4)	0.00 units on a scale Standard Deviation 0.00	1.00 units on a scale Standard Deviation 1.41	0.75 units on a scale Standard Deviation 0.96
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Rehab: Change at Week 26 (N=4,4,4)	1.00 units on a scale Standard Deviation 0.82	0.25 units on a scale Standard Deviation 0.50	0.50 units on a scale Standard Deviation 1.73
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Extensive: Baseline (N=4,5,4)	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Mobility, Extensive: Change at Week 26 (N=4,4,4)	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00	1.25 units on a scale Standard Deviation 1.50
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, None: Baseline (N=4,5,4)	4.25 units on a scale Standard Deviation 0.96	4.60 units on a scale Standard Deviation 0.55	4.75 units on a scale Standard Deviation 0.50
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, None: Change at Week 26 (N=4,4,4)	0.75 units on a scale Standard Deviation 0.96	-0.25 units on a scale Standard Deviation 0.50	0.25 units on a scale Standard Deviation 0.50
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Child: Baseline (N=4,5,4)	1.50 units on a scale Standard Deviation 2.38	0.00 units on a scale Standard Deviation 0.00	0.25 units on a scale Standard Deviation 0.50
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Child: Change at Week 26 (N=4,4,4)	-1.50 units on a scale Standard Deviation 2.38	0.00 units on a scale Standard Deviation 0.00	-0.25 units on a scale Standard Deviation 0.50
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Rehab: Baseline (N=4,5,4)	0.00 units on a scale Standard Deviation 0.00	0.40 units on a scale Standard Deviation 0.55	0.00 units on a scale Standard Deviation 0.00
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Rehab: Change at Week 26 (N=4,4,4)	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Extensive: Baseline (N=4,5,4)	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00	0.00 units on a scale Standard Deviation 0.00
Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 Social, Extensive: Change at Week 26 (N=4,4,4)	0.00 units on a scale Standard Deviation 0.00	0.25 units on a scale Standard Deviation 0.50	0.00 units on a scale Standard Deviation 0.00