Trial · NCT00418561
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 21
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From study drug administration up to Week 28 | 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. |
| Primary | Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Baseline, Week 26 | 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. |
| Primary | Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Baseline, Week 26 | 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. |
| Primary | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Baseline up to Week 26 | 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. |
| Primary | Change From Baseline in Mullen's Scales of Early Learning at Week 26 | Baseline, Week 26 | 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. |
| Primary | Maximum Plasma Drug Concentration (Cmax) of Recombinant Human Arylsulphatase A (rhASA) | 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 | |
| Primary | Arylsulfatase A (ASA) Activity in Leukocytes | Pre-dose and post-dose at 24 hours on Day 0 and at Weeks 8 and 26 | |
| Secondary | Change From Baseline in Nerve Conduction Velocity at Week 26 | Baseline, Week 26 | 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. |
| Secondary | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline, Week 26 | |
| Secondary | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Baseline up to Week 26 | 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. |
| Secondary | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Baseline, Week 26 | 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). |
| Other | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Baseline up to Week 26 | 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. |
| Other | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Coagulation | Baseline up to Week 26 | 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. |
| Other | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Genotyping | Baseline up to Week 26 | Number of participants with at least 1 shift from baseline to Week 26 are reported. Abbreviations: CSF=Cerebrospinal fluid; NFP=Neurofilament proteins. |
| Other | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Hematology | 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. |
| Other | Number of Participants With Abnormal Findings in Urine Analysis | Baseline up to Week 26 | 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. |
| Other | Number of Participants With Clinically Significant Abnormal Electrocardiogram (ECG) Findings | Baseline up to Week 26 | Abnormal ECG findings were considered as clinically significant at the discretion of investigator. |
| Other | Change From Baseline in Chitotriosidase at Week 26 | Baseline, Week 26 | |
| Other | Change From Baseline in Neurofilament Proteins (NFP), Glial Fibrillary Acidic Protein (GFAP) and Tauprotein in Cerebrospinal Fluid (CSF) at Week 26 | Baseline, Week 26 | |
| Other | Change From Baseline in Amplitude at Week 26 | Baseline, Week 26 | Abbreviations: MN=Median Nerve; PN=Peroneal Nerve; SN=Sural Nerve; Dig.=Digit; APB=abductor pollicis brevis; EDB=extensor digitorum brevis. |
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.
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