Trial · NCT00418561
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
NCT00418561, Phase 1, Completed, Shire; results first posted 2015-08-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD). Trials, record NCT00418561. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00418561
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
- NCT number
- NCT00418561
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 13 participants, actual
- Lead sponsor
- Shire
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Interventions
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- First posted
- 2007-01-05
- Last updated
- 2021-06-25
- Other registries' IDs
- eudract:2006-005341-11
- Results first posted
- 2015-08-13
- Limitations and caveats
ASA activity in leukocytes could not be included in basic results format as the results were presented graphically. Due to quick disappearance of rhASA from plasma, rhASA levels were not possible to report.
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| 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. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From study drug administration up to Week 28 | Intent-to-treat (ITT) population included all participants who received at least 1 dose of study drug. | participants | NUMBER |
| PRIMARY | Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Baseline, Week 26 | ITT | percent (%) change | MEAN |
| PRIMARY | Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Baseline, Week 26 | ITT | percent (%) change | MEAN |
| PRIMARY | Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Baseline up to Week 26 | ITT. Here, the number of participants analyzed are the participants evaluable for this outcome. | participants | NUMBER |
| PRIMARY | Change From Baseline in Mullen's Scales of Early Learning at Week 26 | Baseline, Week 26 | ITT | percent (%) change | MEAN |
| SECONDARY | Change From Baseline in Nerve Conduction Velocity at Week 26 | Baseline, Week 26 | ITT. Here, N signifies the number of participants who were evaluable for the respective category. | meters per second | MEAN |
| SECONDARY | Number of Participants Who Had Undergone Nerve Biopsy and Had a Normal Nerve at Both Baseline and Week 26 | Baseline, Week 26 | ITT. | participants | NUMBER |
| SECONDARY | Number of Participants With Shift From Baseline to Week 26 in Magnetic Resonance Imaging (MRI)-Loes Scores | Baseline up to Week 26 | ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome. | participants | NUMBER |
| SECONDARY | Change From Baseline in Paediatric Evaluation of Disability Inventory (PEDI) Scores at Week 26 | Baseline, Week 26 | ITT. Here, N signifies the number of participants who were evaluable for the respective category. | units on a scale | MEAN |
| OTHER_PRE_SPECIFIED | Number of Participants With Shift From Baseline to Week 26 in Clinical Laboratory Evaluations: Biochemistry | Baseline up to Week 26 | ITT. Here, number of participants analyzed in the Cohort 2 are the participants evaluable for this outcome. | participants | NUMBER |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group634
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 1 | 4 | participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 2 | 5 | participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Cohort 3 | 4 | participants | ||
| Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Cohort 1 | -2.36 | 95% Confidence Interval -7.62 to 2.91 | percent (%) change | |
| Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Cohort 2 | -8.29 | 95% Confidence Interval -13.55 to -3.02 | percent (%) change | |
| Change From Baseline in Gross Motor Function Measure (GMFM) at Week 26 | Cohort 3 | -10.90 | 95% Confidence Interval -16.17 to -5.64 | percent (%) change | |
| Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Cohort 1 | 24.55 | 95% Confidence Interval 0.44 to 48.66 | percent (%) change | |
| Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Cohort 2 | -3.77 | 95% Confidence Interval -22.33 to 14.80 | percent (%) change | |
| Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatide at Week 26 | Cohort 3 | -4.32 | 95% Confidence Interval -28.17 to 19.53 | percent (%) change | |
| Number of Participants With Shift From Baseline to Week 26 in Sulfatide Levels in Urine | Cohort 1 | Negative(0) to negative(0) | 0 | participants |
1 to 10 of 634Source ClinicalTrials.gov
See all 634 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 4 | |
| Overall Study | STARTED | Cohort 2 | 5 | |
| Overall Study | STARTED | Cohort 3 | 4 | |
| Overall Study | COMPLETED | Cohort 1 | 4 | |
| Overall Study | COMPLETED | Cohort 2 | 4 | |
| Overall Study | COMPLETED | Cohort 3 | 4 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2 | 1 | |
| Overall Study | NOT COMPLETED | Cohort 3 | 0 |
Source ClinicalTrials.gov
Adverse events126
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Bronchitis acute | Infections and infestations | Cohort 1 | 1 | 4 | 1 |
| other | Bronchitis acute | Infections and infestations | Cohort 2 | 0 | 5 | 0 |
| other | Bronchitis acute | Infections and infestations | Cohort 3 | 0 | 4 | 0 |
| other | Tonsillitis | Infections and infestations | Cohort 1 | 1 | 4 | 1 |
| other | Tonsillitis | Infections and infestations | Cohort 2 | 0 | 5 | 0 |
| other | Tonsillitis | Infections and infestations | Cohort 3 | 1 | 4 | 1 |
| other | Nasopharyngitis | Infections and infestations | Cohort 1 | 1 | 4 | 1 |
| other | Nasopharyngitis | Infections and infestations | Cohort 2 | 0 | 5 | 0 |
| other | Nasopharyngitis | Infections and infestations | Cohort 3 | 1 | 4 | 1 |
| other | Influenza | Infections and infestations | Cohort 1 | 0 | 4 | 0 |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | ||
| Total, all-cause mortality | Cohort 2 | ||
| Total, all-cause mortality | Cohort 3 | ||
| Total, other adverse events | Cohort 1 | 4 | 4 |
| Total, other adverse events | Cohort 2 | 5 | 5 |
| Total, other adverse events | Cohort 3 | 4 | 4 |
| Total, serious adverse events | Cohort 1 | 1 | 4 |
| Total, serious adverse events | Cohort 2 | 2 | 5 |
| Total, serious adverse events | Cohort 3 | 2 | 4 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
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 trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00418561
GET https://conductscience.org/api/trials/v1/NCT00418561/registered?page=2