An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

NCT01887938 | Completed Phase 1 DMC

• 3 other identifiers: HGT-MLD-0712012-003775-202024-514403-34-00
• Study Type: interventional
• Target: 24
• Locations: 7 countries
• Sites: 15

Brief Summary

The purpose of this study is to collect long-term safety data in participants with metachromatic leukodystrophy (MLD) who are receiving HGT-1110 and have participated in Study HGT-MLD-070 (NCT01510028) through Week 40.

Conditions

Metachromatic Leukodystrophy (MLD)

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

  An AE is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational drug product-related. TEAEs are defined as all AEs occurring or worsening at or after the first dose of investigational drug product in HGT-MLD-071 or ongoing from HGT-MLD-070 at the time of enrollment into HGT-MLD-071.

  Baseline to Follow-up (Week 628)

• Presence of Anti-HGT-1110 Antibodies in Cerebrospinal Fluid (CSF) and Serum

  The presence of anti-HGT-1110 antibodies in CSF and serum will be assessed.

  Baseline until end of the study (Week 624)

Secondary Outcomes (12)

• Change From Baseline in Motor Function as Assessed by Gross Motor Function Measure (GMFM-88) Total Score at Week 624

  Baseline, Week 624

• Change From Baseline in the Adaptive Behavior Composite Standard Score Measured by the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)

  Baseline, Week 624

• Change From Baseline in the Domain-Specific Caregiver Observed Metachromatic Leukodystrophy Functioning and Outcomes Reporting Tool (COMFORT) Scores

  Baseline, Week 624

• Maximum Observed Serum Concentration (Cmax) of HGT-1110

  Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, and 48 hours post-dose on Weeks 104, 258, 364 and 622 in Cohort 4 and Week 8 in Cohorts 1-3

• Time of Maximum Observed Serum Concentration (Tmax) of HGT-1110

  Pre-dose, 0.5, 1, 2, 4, 8, 12, 24, and 48 hours post-dose on Weeks 104, 258, 364 and 622 in Cohort 4 and Week 8 in Cohorts 1-3

Study Arms (4)

Cohort 1

EXPERIMENTAL

Participants will receive 10 milligram (mg) of HGT-1110 (Recombinant human arylsulfatase A) intrathecal (IT) injection every-other-week (EOW).

Biological: HGT-1110

Cohort 2

EXPERIMENTAL

Participants will receive 30 mg of HGT-1110 IT injection EOW.

Biological: HGT-1110

Cohort 3

EXPERIMENTAL

Participants will receive 100 mg of HGT-1110 IT injection EOW.

Biological: HGT-1110

Cohort 4

EXPERIMENTAL

Participants will receive 100 mg of HGT-1110 IT injection once weekly for 12 weeks followed by 150 mg EOW.

Biological: HGT-1110

Interventions

HGT-1110 BIOLOGICAL

Participants will receive IT injection of HGT-1110.

Also known as: Recombinant human arylsulfatase A

Cohort 1; Cohort 2; Cohort 3; Cohort 4

Eligibility Criteria

• Age: 0 Years - 13 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant has participated in Study HGT-MLD-070 (NCT01510028) through Week 40.
• Participant must have no safety or medical issues that contraindicate participation.
• The participant, participant's parent(s), or legally authorized representative(s) must provide written informed consent and/or assent (if applicable) prior to performing any study-related activities.

You may not qualify if:

• The participant is unable to comply with the protocol (example, is unable to return for safety evaluations, or is otherwise unlikely to complete the study) as determined by the investigator.
• Undergoes bone marrow transplant (BMT), hematopoietic stem cell transplantation (HSCT), or gene therapy at any point during the study.
• The participant has any known or suspected hypersensitivity to agents used for anesthesia or is thought to be at an unacceptably high risk for associated potential complications of airway compromise or other conditions.
• The participant is pregnant or breastfeeding.
• The participant is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or drug delivery device) other than those used in HGT-MLD-070 (NCT01510028) within 6 months 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 SIDDD Instructions for Use (IFU), including:
• The participant has had, or may have, an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device.
• The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the investigator.
• 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 CSF shunt device.
• The participant has shown an intolerance to an implanted device.

Sponsors & Collaborators

• Shire: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (15)

The Children's Hospital at Westmead

Westmead, 2145, Australia

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, 90035-003, Brazil

Location

Detska Interni Klinika, Lf Mu A Fn Brno

Brno, 61300, Czechia

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Hopital Femme Mere Enfant

Bron, 69 677, France

Location

Hopital Gui de Chauliac - CHRU de Montpellier

Montpellier, 34000, France

Location

CHU de Nantes

Nantes, 44093, France

Location

CHR Orleans - Hopital La Source

Orléans, 45067, France

Location

Hôpital de Bicêtre

Le Kremlin-Bicêtre, Île-de-France Region, 94275, France

Location

Center for Pediatric Clinical Studies (CPCS)

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

Klinikum Oldenburg

Oldenburg, 26133, Germany

Location

Marien-Hospital Wesel gGmbh

Wesel, 46483, Germany

Location

Kitakyushu Municipal Yahata Hospital

Fukuoka, 805-8534, Japan

Location

Kurashiki Central Hospital

Okayama Prefecture, 710-8602, Japan

Location

Osaka University Hospital

Osaka, 565-0871, Japan

Location

Related Links

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MeSH Terms

Conditions

Leukodystrophy, Metachromatic

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Sulfatidosis; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Leukoencephalopathies; Demyelinating Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2013
• First Posted: June 27, 2013
• Study Start: May 23, 2013
• Primary Completion: February 13, 2026
• Study Completion: February 13, 2026
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1); AU (1); JP (3); CZ (1); BR (1); DE (3); FR (5)