A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy



Status:Not yet recruiting
Healthy:No
Age Range:Any
Updated:12/13/2018
Start Date:April 30, 2019
End Date:August 15, 2022
Contact:Shire Contact
Email:ClinicalTransparency@shire.com
Phone:+1 866 842 5335

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A Global, Multicenter, Open-label, Matched Historical Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy

The primary purpose of this study is to evaluate the effects of intrathecal (IT)
administration of SHP611 on gross motor function, using the Gross Motor Function
Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical
control data in children with metachromatic leukodystrophy (MLD).


Inclusion Criteria:

- The participant must have a documented diagnosis of metachromatic leukodystrophy (MLD)
(Groups A-D): a. low arylsulfatase A (ASA) activity in leukocytes; b. elevated
sulfatides in urine.

- The participant must have a gait disorder due to spastic ataxia or weakness attributed
to MLD by the investigator and documented by a pediatric neurologist or medical
geneticist by 30 months of age (Groups A-C) or presymptomatic (Group D).

- 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: less than (<) 18 months of age.

- The participant's Gross Motor Function Classification in Metachromatic Leukodystrophy
(GMFC-MLD) level at screening must be: Group A: GMFC-MLD level of 1 or 2; Group B:
GMFC-MLD level of 3; Group C: GMFC-MLD level of 4; Group D: presymptomatic, are
younger siblings of enrolled participants, and have the same ASA allelic constitution.

- 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.

Inclusion criteria for matched historical controls:

- The participant must have a documented diagnosis of MLD: a. low ASA activity in
leukocytes; b. elevated sulfatides in urine.

- Participant must have a gait disorder due to spastic ataxia or weakness attributed to
MLD by the investigator and documented at baseline.

- Participant must have at least 2 motor assessments by GMFC-MLD with the second
assessment occurring at approximately 106 (+/-6) weeks after the first assessment or
else a second assessment measured before week 100 with a GMFC-MLD level 5 or 6.
Participant with GMFC-MLD data (pro or retrospectively determined) must have the
earliest observation of level 1 or 2 (walking with support) in the data
source-verified medical record.

- Participant must be 18 to 48 months of age at the earliest assessment.

Exclusion Criteria:

- Multiple sulfatase disorder as determined by abnormal activity of another lysosomal
sulfatase (based upon the reference laboratory's normal range).

- History of hematopoietic stem cell transplantation (HSCT) or gene therapy or undergoes
HSCT or gene therapy at any point during the study.

- Initial presentation of behavioral or cognitive symptoms of MLD (per investigator's
clinical judgment).

- 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.

- The participant is enrolled in another clinical study that involves use of any
investigational product (drug or device) within 30 days prior to study enrollment or
at any time during the study.

- The participant has a condition that is contraindicated as described in the
SOPH-A-PORT Mini S intrathecal drug delivery device (IDDD) Instructions for Use (IFU).

Exclusion criteria for matched historical controls:

- History of hematopoietic stem cell transplantation (HSCT) or gene therapy or undergoes
HSCT or gene therapy at any point during the study.

- Initial presentation of behavioral or cognitive symptoms of MLD (per investigator's
clinical judgment).

- The participant is enrolled in another clinical study that involves use of any
investigational product (drug or device).
We found this trial at
11
sites
1601 Northwest 12th Avenue
Miami, Florida 33136
(305) 243-6545
Principal Investigator: Mustafa Tekin, MD
Phone: 305-243-2381
University of Miami Miller School of Medicine The University of Miami Leonard M. Miller School...
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Adeline Vanderver, MD
Phone: 267-514-5753
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
Principal Investigator: Josh Bonkowsky, MD
Phone: 801-581-6756
University of Utah Research is a major component in the life of the U benefiting...
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Barbara Burton, MD
Phone: 312-227-6120
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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Cincinnati, Ohio 45229
Principal Investigator: Loren Pena, MD
Phone: 919-684-2036
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Edegem, 2650
Principal Investigator: Francois Eyskens, MD
Phone: 3238213810
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Iowa City, Iowa 52242
Principal Investigator: John Bernat, MD
Phone: 319-356-2675
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550 1st Ave
New York, New York 10016
(212) 263-7300
Principal Investigator: Heather Lau, MD
Phone: 212-263-8344
New York University Langone Medical Center NYU NYU Langone Medical Center, a world-class, patient-centered, integrated,...
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3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
Principal Investigator: Maria Escolar, MD
Phone: 412-692-9955
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200 1st Street Southwest
Rochester, Minnesota 55905
Principal Investigator: Marc Patterson, MD
Phone: 507-284-3351
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Torrance, California 90502
Principal Investigator: Agnes Chen, MD
Phone: 310-222-4160
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Torrance, CA
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