Trial · NCT00681811

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

NCT00681811, Phase 2, Terminated, Shire; results first posted 2013-11-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD. Trials, record NCT00681811. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00681811

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
NCT number: NCT00681811
Status: Terminated
Phases: Phase 2
Enrollment: 11 participants, actual
Lead sponsor: Shire
Conditions: Late Infantile Metachromatic Leukodystrophy
Interventions: HGT-1111
First posted: 2008-05-21
Last updated: 2021-06-08
Other registries' IDs: eudract:2008-000084-41
Results first posted: 2013-11-08
Limitations and caveats: Early termination due to lack of efficacy.

Source ClinicalTrials.gov

Registered outcomes4

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Days of Exposure to HGT-1111	Baseline until end of study (Week 139)	End of study was defined as until HGT-1111 was commercially available, the participant's participation was discontinued, or the study was terminated by the Sponsor.
Secondary	Level of Cerebrospinal Fluid (CSF) Sulfatide	Baseline until end of study (Week 139)	Level of CSF sulfatide measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Secondary	Level of White Matter Metabolites	Baseline until end of study (Week 139)	Level of white matter metabolites \[N-acetyl Aspartate (NAA)\] measured at 6-month intervals in HGT-MLD-049 (NCT00681811).
Secondary	Score of Gross Motor Function Measurement (GMFM)	Baseline until end of study (Week 139)	Gross motor function was measured using GMFM-88 at 6-month intervals. The GMFM-88 item scores were 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) to 264 (maximum). Decrease in GMFM score indicates disease progression.

Source ClinicalTrials.gov

Outcomes4

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
SECONDARY	Level of Cerebrospinal Fluid (CSF) Sulfatide	Baseline until end of study (Week 139)	Safety population. Number of participants analysed signifies participants who were evaluable for the respective arms under this outcome measure.	nanomole per liter (nmol/L)	MEAN
SECONDARY	Level of White Matter Metabolites	Baseline until end of study (Week 139)	Safety population. Number of participants analysed signifies participants who were evaluable for the respective arms under this outcome measure. No participants were analysed after Month 18, hence, data were not available after Month 18.	nmol/L	MEAN
SECONDARY	Score of Gross Motor Function Measurement (GMFM)	Baseline until end of study (Week 139)	Safety population. Number of participants analysed signifies participants who were evaluable for the respective arms under this outcome measure.	scores on a scale	MEAN
PRIMARY	Days of Exposure to HGT-1111	Baseline until end of study (Week 139)	Safety population was defined as all enrolled participants who received at least one study infusion (or any portion of a dose) of HGT-1111.	Days	MEAN

Source ClinicalTrials.gov

Results by group24

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Level of Cerebrospinal Fluid (CSF) Sulfatide	100 U/kg HGT-1111 (Month 6)	685.0	Standard Deviation 522.79	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	200 U/kg HGT-1111 (Month 6)	732.5	Standard Deviation 545.06	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	100 U/kg HGT-1111 (Month 12)	745.0	Standard Deviation 525.12	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	200 U/kg HGT-1111 (Month 12)	1150.0	Standard Deviation 676.39	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	100 U/kg HGT-1111 (Month 18)	700.0	Standard Deviation 526.78	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	200 U/kg HGT-1111 (Month 18)	850.0	Standard Deviation 491.81	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	100 U/kg HGT-1111 (Month 24)	900.0	Standard Deviation NA	nanomole per liter (nmol/L)
Level of Cerebrospinal Fluid (CSF) Sulfatide	200 U/kg HGT-1111 (Month 24)	837.5	Standard Deviation 441.94	nanomole per liter (nmol/L)
Level of White Matter Metabolites	100 U/kg HGT-1111 (Month 6)	0.528	Standard Deviation 0.176	nmol/L
Level of White Matter Metabolites	200 U/Kg-HGT-1111(Month 6)	0.385	Standard Deviation 0.237	nmol/L

1 to 10 of 24Source ClinicalTrials.gov

See all 24 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	100 U/kg HGT-1111	6
Overall Study	STARTED	200 U/kg HGT-1111	5
Overall Study	COMPLETED	100 U/kg HGT-1111	3
Overall Study	COMPLETED	200 U/kg HGT-1111	1
Overall Study	NOT COMPLETED	100 U/kg HGT-1111	3
Overall Study	NOT COMPLETED	200 U/kg HGT-1111	4

Source ClinicalTrials.gov

Adverse events166

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	Pallor	Vascular disorders	100 U/kg HGT1111	0	6	0
other	Pallor	Vascular disorders	200 U/kg HGT1111	1	5	1
other	Flushing	Vascular disorders	100 U/kg HGT1111	1	6	1
other	Flushing	Vascular disorders	200 U/kg HGT1111	3	5	8
other	Disease progression	General disorders	100 U/kg HGT1111	0	6	0
other	Disease progression	General disorders	200 U/kg HGT1111	1	5	1
other	Adverse drug reaction	General disorders	100 U/kg HGT1111	0	6	0
other	Adverse drug reaction	General disorders	200 U/kg HGT1111	1	5	1
other	Generalised oedema	General disorders	100 U/kg HGT1111	0	6	0
other	Generalised oedema	General disorders	200 U/kg HGT1111	1	5	1

1 to 10 of 166Source ClinicalTrials.gov

See all 166 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	100 U/kg HGT1111
Total, all-cause mortality	200 U/kg HGT1111
Total, other adverse events	100 U/kg HGT1111	6	6
Total, other adverse events	200 U/kg HGT1111	5	5
Total, serious adverse events	100 U/kg HGT1111	2	6
Total, serious adverse events	200 U/kg HGT1111	4	5

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
33332761	DERIVED

Source PubMed

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 comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
100 U/kg HGT1111 vs 200 U/kg HGT1111: serious adverse events

Source Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT00681811

GET https://conductscience.org/api/trials/v1/NCT00681811/results?page=2

API reference For AI assistants