Metachromatic Leukodystrophy (MLD)Phase 2EMBOLDENNCT03771898
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
A Phase 2 study testing SHP611 in people with Metachromatic Leukodystrophy (MLD), run by Shire at 30 sites in 14 countries.
Trial summary
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Read more about this study
- Who is it for?
- Ages 6 to 72 months
- Conditions the study is for
- Metachromatic Leukodystrophy (MLD)
- What is being tested?
- SHP611
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 30 sitesin 14 countries
- Study dates
- 13 May 2019to 6 May 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 72 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- The participant must have a documented diagnosis of MLD (Groups A-F):
- Low ASA activity in leukocytes (compared to laboratory normal range).
- Elevated sulfatides in urine.
- The participant must have a gait disorder due to spastic ataxia or weakness attributable to MLD by the investigator and documented by a primary care physician or a specialist physician by 30 months of age (Groups A-C, and F), or be minimally symptomatic and greater than or equal to (> =) 6 to less than (<) 18 months of age (Group D) or be early symptomatic and > =12 to < 18 months of age (Group E). Participants in Group E must have neurological symptoms either documented by either a primary care physician or a specialist physician.
- The participant's age at the time of informed consent, must be: Group A: 18 to 48 months of age; Group B: 18 to 72 months of age; Group C: 18 to 72 months of age; Group D: >= 6 to < 18 months of age; Group E: > = 12 to < 18 months of age; Group F: 18 to 72 months of age.
- The participant's GMFC-MLD category at screening must be: Group A: GMFC-MLD category of 1 or 2; Group B: GMFC-MLD category of 3; Group C: GMFC-MLD category of 4; Group D: minimally symptomatic, >= 6 to < 18 months of age, with the same arylsulfatase (ASA) allelic constitution as an older sibling with confirmed late infantile or juvenile onset MLD; Group E: early symptomatic, >= 12 to < 18 months of age with a GMFC-MLD category of 1 or 2 with a history of achieving stable walking (defined as at least 1 month of independent walking); Group F: GMFC-MLD category of 5 or 6.
- The participant and his/her parent/representative(s) must have the ability to comply with the clinical protocol.
- Participant's parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the participant.
Who cannot join
- Multiple sulfatase disorder as determined by abnormal activity of another lysosomal sulfatase (based upon the reference laboratory's normal range) or a known genetic disorder other than MLD.
- History of bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy; or undergoes BMT, HSCT, or gene therapy: at any point during the study.
- Primary presentation of MLD was behavioral or cognitive symptoms (per investigator's clinical judgment); behavioral symptoms that are secondary to motor deficits (example [eg], tantrums in response to loss of motor skills) are not exclusionary.
- The participant has any known or suspected hypersensitivity to agents used for anesthesia or has history of difficult airway or potential for airway compromise.
- Any other medical condition or serious comorbid illness that in the opinion of the investigator would preclude participation in the study.
- Participants with laboratory, ECG or vital sign abnormalities reflecting intercurrent illness that may compromise their safety during the trial should not be enrolled. Abnormal laboratory, vital sign and ECG results at screening should be reviewed with the Takeda medical monitor.
Show all 25Show fewer
- The participant is enrolled in another clinical study that involves use of any investigational product (drug or device) within 30 days or 5 half-lives (whichever is longer) prior to study enrollment or at any time during the study.
- The participant has had prior exposure to SHP611.
- The participants must weigh > 11 pound (lbs) (5 kilograms [kg]).
- The participant has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU)
- The participant has had, or may have, an allergic reaction to the materials of construction.
- The participant has shown an intolerance to an implanted device.
- The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port.
- The participant's drug therapy requires substances known to be incompatible with the materials of construction.
- The participant has a known or suspected local or general infection.
- The participant is at risk of abnormal bleeding due to a medical condition or therapy.
- The participant has one or more spinal abnormalities that could complicate safe implantation or fixation.
- The participant has a functioning Cerebro spinal fluid(CSF) shunt device .
- Matched External Control Participants for Group A from Global Leukodystrophy Initiative of Metachromatic Leukodystrophy (GLIA-MLD) The matched external control group must have data for at least baseline gross motor function evaluation. Selection of the external control participants from GLIA-MLD will follow a set of criteria as similar as possible to the inclusion criteria for Group A in the SHP611-201 study protocol.
- A filtering process will be applied to select the external control participants from the GLIA-MLD database, by meeting all of the following 3 filtering criteria:
- Filtering criterion 1: requiring documented diagnosis of MLD, based on
- low arylsulfatase A (ASA) activity in leukocytes AND elevated sulfatides in urine. OR
- biallelic variants in arylsulfatase A gene (ARSA) AND (either low ASA activity in leukocytes OR elevated sulfatides in urine).
- Filtering criterion 2: requiring documented gait disorder. Participants will be considered qualifying if they present with a gait disorder before 2.5 years (30 months) of age and have a medical record reporting a gait abnormality including, but not limited to, the following terms: ataxia, spasticity, and hyper/hypotonia.
- Filtering criterion 3: participants will be considered qualifying if they have at least 1 clinical encounter occurring between the age of 18 to 48 months with a GMFC-MLD category either 1 or 2.
Trial locations and contacts
30 sites in 14 countries.
30 sites in 14 countries
United States11 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30303
Childrens Hospital Colorado
Aurora, Colorado, 80045
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
New York University Langone Medical Center
New York, New York, 10016
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Mayo Clinic - PPDS
Rochester, Minnesota, 55905
University of Utah
Salt Lake City, Utah, 84108
Los Angeles Biomedical Research Institute at Harbor-UCLA
Torrance, California, 90502
Canada4 sites
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7
Montreal Children's Hospital
Montreal, Quebec, H3H 1P3
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
France2 sites
Hôpital Bicêtre - Paris Sud
Le Kremlin-Bicêtre, 94275
CHU Lenval
Nice, 06200
Germany2 sites
Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246
Universitätsklinikum Tübingen
Tübingen, 72076
Spain2 sites
Hospital Universitario Cruces
Barakaldo, Vizcaya, 48903
Hospital Vall d'Hebrón
Barcelona, 8035
Argentina1 site
Hospital Universitario Austral - PIN
Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ
United Kingdom1 site
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH
About this study
Official title: A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated.
Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Being tested
- SHP611
- Medical subject headings
- Leukodystrophy, Metachromatic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SHP611Drug
Also called HGT-1110, TAK-611, recombinant human arylsulfatase A [rhASA]
Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks.
| Study group | Role | What they receive |
|---|---|---|
| SHP611 | Experimental | Participants will receive 150 milligrams (mg) of SHP611 intrathecally (IT) via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once weekly for 106 weeks in six groups (Group A, B, C, D, E, and F) based on participant's age and motor dysfunction. Receives: Drug: SHP611 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: Baseline up to Week 106
Secondary outcomes (21)
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
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Group A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Group A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Percent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Group A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
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
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Change From Baseline in GMFM-88 Total Score at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Group A: Number of Participants With GMFM-88 Total Score Decrease of <= 20 Points From Baseline and a Total Score >=40 Points at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Number of Participants With Change From Baseline in Expressive Language Evaluated by Using the Expressive Language Function Classification in Metachromatic Leukodystrophy (ELFC-MLD) Scale Categories at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline, Week 106
Number of Participants With Treatment-emergent Adverse Event (TEAEs)
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: From start of study drug administration up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Values at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Values at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Cerebrospinal Fluid (CSF) Laboratory Parameters at Week 106
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Anti-Drug Antibody (ADA) Response to SHP611
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With IDDD Implantations
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With Any IDDD Related Malfunctions
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
IDDD Longevity as Assessed by Time to IDDD Failure
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Number of Participants With TEAE Related to IDDD and Surgical Procedure
Data collection is ongoing and detailed reporting will be available after the study completion date.
Measured: Baseline up to Week 106
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2018
- Study start13 May 2019
- Primary completion8 Mar 2023
- Study completion6 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Metachromatic Leukodystrophy (MLD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03771898
- Sponsor's protocol ID
- SHP611-201
- Other IDs
- 2018-003291-12, jRCT2041200086, 2018
- Acronym
- EMBOLDEN
- Status last verified
- Jun 2026
- First submitted
- 10 Dec 2018
- Last updated
- 10 Jul 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
- ClinicalTrials.gov ID
- NCT03771898
- Acronym / sponsor ID
- EMBOLDEN / SHP611-201
- Conditions
- Metachromatic Leukodystrophy (MLD)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Takeda Development Center Americas, Inc.
- Study start
- 13 May 2019 (actual)
- Primary completion
- 8 Mar 2023 (actual)
- Study completion
- 6 May 2026 (actual)
- First posted
- 11 Dec 2018 (actual)
- Results
- Posted 6 Jun 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated.
Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SHP611 | Experimental | Drug: SHP611Participants will receive 150 milligrams (mg) of SHP611 intrathecally (IT) via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once weekly for 106 weeks in six groups (Group A, B, C, D, E, and F) based on participant's age and motor dysfunction. |
Who may qualify
- Ages
- 6 to 72 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- The participant must have a documented diagnosis of MLD (Groups A-F):
- The participant must have a gait disorder due to spastic ataxia or weakness attributable to MLD by the investigator and documented by a primary care physician or a specialist physician by 30 months of age (Groups A-C, and F), or be minimally symptomatic and greater than or equal to (> =) 6 to less than (<) 18 months of age (Group D) or be early symptomatic and > =12 to < 18 months of age (Group E). Participants in Group E must have neurological symptoms either documented by either a primary care physician or a specialist physician.
- The participant's age at the time of informed consent, must be: Group A: 18 to 48 months of age; Group B: 18 to 72 months of age; Group C: 18 to 72 months of age; Group D: >= 6 to < 18 months of age; Group E: > = 12 to < 18 months of age; Group F: 18 to 72 months of age.
- The participant's GMFC-MLD category at screening must be: Group A: GMFC-MLD category of 1 or 2; Group B: GMFC-MLD category of 3; Group C: GMFC-MLD category of 4; Group D: minimally symptomatic, >= 6 to < 18 months of age, with the same arylsulfatase (ASA) allelic constitution as an older sibling with confirmed late infantile or juvenile onset MLD; Group E: early symptomatic, >= 12 to < 18 months of age with a GMFC-MLD category of 1 or 2 with a history of achieving stable walking (defined as at least 1 month of independent walking); Group F: GMFC-MLD category of 5 or 6.
- The participant and his/her parent/representative(s) must have the ability to comply with the clinical protocol.
Key exclusion criteria (25 listed)
- Multiple sulfatase disorder as determined by abnormal activity of another lysosomal sulfatase (based upon the reference laboratory's normal range) or a known genetic disorder other than MLD.
- History of bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy; or undergoes BMT, HSCT, or gene therapy: at any point during the study.
- Primary presentation of MLD was behavioral or cognitive symptoms (per investigator's clinical judgment); behavioral symptoms that are secondary to motor deficits (example [eg], tantrums in response to loss of motor skills) are not exclusionary.
- The participant has any known or suspected hypersensitivity to agents used for anesthesia or has history of difficult airway or potential for airway compromise.
- Any other medical condition or serious comorbid illness that in the opinion of the investigator would preclude participation in the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SHP611Also: HGT-1110, TAK-611, recombinant human arylsulfatase A [rhASA]Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks. | Drug | SHP611 |
Primary outcome
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
Time frame: 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.
21 secondary outcomes
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
Time frame: Baseline up to Week 106
Number of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106
Time frame: Baseline, Week 106
Group A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories
Time frame: Baseline, Week 106
Group A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories
Time frame: Baseline, Week 106
Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106
Time frame: Baseline, Week 106
Percent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106
Time frame: Baseline, Week 106
Group A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score
Time frame: Baseline up to Week 106
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
Time frame: Baseline, Week 106
Change From Baseline in GMFM-88 Total Score at Week 106
Time frame: Baseline, Week 106
Group A: Number of Participants With GMFM-88 Total Score Decrease of <= 20 Points From Baseline and a Total Score >=40 Points at Week 106
Time frame: Baseline, Week 106
Number of Participants With Change From Baseline in Expressive Language Evaluated by Using the Expressive Language Function Classification in Metachromatic Leukodystrophy (ELFC-MLD) Scale Categories at Week 106
Time frame: Baseline, Week 106
Number of Participants With Treatment-emergent Adverse Event (TEAEs)
Time frame: From start of study drug administration up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Values at Week 106
Time frame: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings at Week 106
Time frame: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Values at Week 106
Time frame: Baseline up to Week 106
Number of Participants With Clinically Significant Change From Baseline in Cerebrospinal Fluid (CSF) Laboratory Parameters at Week 106
Time frame: Baseline up to Week 106
Number of Participants With Anti-Drug Antibody (ADA) Response to SHP611
Time frame: Baseline up to Week 106
Number of Participants With IDDD Implantations
Time frame: Baseline up to Week 106
Number of Participants With Any IDDD Related Malfunctions
Time frame: Baseline up to Week 106
IDDD Longevity as Assessed by Time to IDDD Failure
Time frame: Baseline up to Week 106
Number of Participants With TEAE Related to IDDD and Surgical Procedure
Time frame: Baseline up to Week 106
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Shire
30 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SHP611 in people with Metachromatic Leukodystrophy (MLD), run by Shire at 30 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Canada, France, Germany, Spain and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: Baseline up to Week 106). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2026.