Trial · NCT03771898

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898, Phase 2, Completed, Shire; results first posted 2024-06-06

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy. Trials, record NCT03771898. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03771898

On this page

Trial

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

Title: A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
NCT number: NCT03771898
Status: Completed
Phases: Phase 2
Enrollment: 36 participants, actual
Lead sponsor: Shire
Conditions: Metachromatic Leukodystrophy (MLD)
Interventions: SHP611
First posted: 2018-12-11
Last updated: 2026-07-10
Other registries' IDs: eudract:2018-003291-12
Results first posted: 2024-06-06

Source ClinicalTrials.gov

Registered outcomes22

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

List	Outcome	Time frame	Description
Primary	Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control	Baseline up to Week 106	Loss of locomotion was estimated using interval censoring survival analysis. Survival probability free of loss of locomotion based on GMFC-MLD was estimated up to Week 106 (or two years), with associated 2-sided 95 percent (%) confidence interval (CI). GMFC-MLD scale consists of 7 categories, scores ranging from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control). Higher scores mean a worse outcome. The data was reported in terms of Mean as survival function was quantified using a weighted average of percentage of participants not reaching the event of interest, with weights derived from the relative size of treated and control units in the strata used for the stratified log-rank test in the primary analysis.
Secondary	Group A: Number of Participants Who Maintained Their Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106 Compared With Matched External Control Group Data	Baseline up to Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Number of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Group A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Group A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Percent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Group A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score	Baseline up to Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Time to Unreversed Decline From Baseline in GMFM-88 Total Score of Greater Than (>) 20 Points or Unreversed Decline to Less Than (<) 40 Points Whichever Occurs First	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.
Secondary	Change From Baseline in GMFM-88 Total Score at Week 106	Baseline, Week 106	Data collection is ongoing and detailed reporting will be available after the study completion date.

1 to 10 of 22Source ClinicalTrials.gov

See all 22 registered outcomes

Outcomes22

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control	Baseline up to Week 106	The modified full analysis set (mFAS) included all participants from Group A who received at least 1 dose of SHP611 and had at least a screening GMFC-MLD assessment, and the matched external control participants for Group A from GLIA-MLD natural history study. As per planned analysis, this outcome measure was assessed in Group A and GLIA-MLD matched external control for Group A.	percent probability	MEAN
SECONDARY	Group A: Number of Participants Who Maintained Their Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106 Compared With Matched External Control Group Data	Baseline up to Week 106
SECONDARY	Number of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106	Baseline, Week 106
SECONDARY	Group A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories	Baseline, Week 106
SECONDARY	Group A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories	Baseline, Week 106
SECONDARY	Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106	Baseline, Week 106
SECONDARY	Percent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106	Baseline, Week 106
SECONDARY	Group A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score	Baseline up to Week 106
SECONDARY	Time to Unreversed Decline From Baseline in GMFM-88 Total Score of Greater Than (>) 20 Points or Unreversed Decline to Less Than (<) 40 Points Whichever Occurs First	Baseline, Week 106
SECONDARY	Change From Baseline in GMFM-88 Total Score at Week 106	Baseline, Week 106

1 to 10 of 22Source ClinicalTrials.gov

See all 22 outcomes

Results by group2

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

Outcome	Group	Category	Value	Spread	Units
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control	Group A, SHP611		7.1	95% Confidence Interval 0 to 20.2	percent probability
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control	GLIA-MLD Matched External Control for Group A		9.4	95% Confidence Interval 0 to 24.4	percent probability

Source ClinicalTrials.gov

Participant flow15

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Group A, SHP611	15
Overall Study	STARTED	Group B, SHP611	5
Overall Study	STARTED	Group C, SHP611	6
Overall Study	STARTED	Group F, SHP611	9
Overall Study	STARTED	GLIA-MLD Matched External Control for Group A	33
Overall Study	COMPLETED	Group A, SHP611	12
Overall Study	COMPLETED	Group B, SHP611	3
Overall Study	COMPLETED	Group C, SHP611	5
Overall Study	COMPLETED	Group F, SHP611	9
Overall Study	COMPLETED	GLIA-MLD Matched External Control for Group A	0

1 to 10 of 15Source ClinicalTrials.gov

See all 15 participant flow

Adverse events1,740

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
other	Abdominal discomfort	Gastrointestinal disorders	Group A, SHP611	1	15
other	Abdominal discomfort	Gastrointestinal disorders	Group B, SHP611	0	5
other	Abdominal discomfort	Gastrointestinal disorders	Group C, SHP611	0	6
other	Abdominal discomfort	Gastrointestinal disorders	Group F, SHP611	1	9
other	Abdominal pain	Gastrointestinal disorders	Group A, SHP611	2	15
other	Abdominal pain	Gastrointestinal disorders	Group B, SHP611	0	5
other	Abdominal pain	Gastrointestinal disorders	Group C, SHP611	1	6
other	Abdominal pain	Gastrointestinal disorders	Group F, SHP611	1	9
other	Abdominal pain upper	Gastrointestinal disorders	Group A, SHP611	0	15
other	Abdominal pain upper	Gastrointestinal disorders	Group B, SHP611	0	5

1 to 10 of 1,740Source ClinicalTrials.gov

See all 1,740 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Group A, SHP611	0	15
Total, all-cause mortality	Group B, SHP611	1	5
Total, all-cause mortality	Group C, SHP611	0	6
Total, all-cause mortality	Group F, SHP611	0	9
Total, other adverse events	Group A, SHP611	15	15
Total, other adverse events	Group B, SHP611	5	5
Total, other adverse events	Group C, SHP611	6	6
Total, other adverse events	Group F, SHP611	9	9
Total, serious adverse events	Group A, SHP611	14	15
Total, serious adverse events	Group B, SHP611	4	5

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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.

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.

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