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

NCT00681811 | Terminated Phase 2 Results

• 2 other identifiers: HGT-MLD-0492008-000084-41
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures. This group of patients will be offered ongoing treatment with HGT-1111 in this protocol. One infusion will be given every other week until the product is commercially available, the patient discontinues, or the study is terminated by the Sponsor, provided no safety issues have emerged.

Conditions

Late Infantile Metachromatic Leukodystrophy

Keywords

Metazyme; Late Infantile Metachromatic Leukodystrophy

Outcome Measures

Primary Outcomes (1)

• Days of Exposure to HGT-1111

  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.

  Baseline until end of study (Week 139)

Secondary Outcomes (3)

• Level of Cerebrospinal Fluid (CSF) Sulfatide

  Baseline until end of study (Week 139)

• Level of White Matter Metabolites

  Baseline until end of study (Week 139)

• Score of Gross Motor Function Measurement (GMFM)

  Baseline until end of study (Week 139)

Study Arms (2)

HGT-1111 100 U/kg

EXPERIMENTAL

Drug: HGT-1111

HGT-1111 200 U/kg

EXPERIMENTAL

Drug: HGT-1111

Interventions

HGT-1111 DRUG

Patients currently dosed with 100 U/kg or 200 U/kg will continue this treatment. Patients dosed with 50 U/kg will be equally randomized to treatment on 100 U/kg or 200 U/kg. The dose will be adjusted every 6-week to account for changes in body weight.The infusion length will be dependent on the dose. Infusion of 100 U/kg will be diluted in 50 ml isotonic sodium chloride and infused over 30 minutes. Infusion of 200 U/kg will be administered in the same manner except for an infusion time of 60 minutes.

Also known as: rhASA, Metazym

HGT-1111 100 U/kg; HGT-1111 200 U/kg

Eligibility Criteria

• Age: 3 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subject's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related activities (Trial-related activities are any procedures that would not have been performed during normal management of the subject)
• Completion of study HGT-MLD-048 (NCT00633139)
• The subject and his/her guardian(s) must have the ability to comply with the protocol

You may not qualify if:

• Spasticity so severe to inhibit transportation
• Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition that, in the opinion of the Investigator, would preclude participation in the trial
• Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial

Sponsors & Collaborators

• Shire: lead

Study Sites (1)

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Related Publications (1)

• I Dali C, Groeschel S, Moldovan M, Farah MH, Krageloh-Mann I, Wasilewski M, Li J, Barton N, Krarup C. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study. Ann Clin Transl Neurol. 2021 Jan;8(1):66-80. doi: 10.1002/acn3.51254. Epub 2020 Dec 17.

  PMID: 33332761 DERIVED

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

Limitations and Caveats

Early termination due to lack of efficacy.

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2008
• First Posted: May 21, 2008
• Study Start: February 20, 2008
• Primary Completion: October 22, 2010
• Study Completion: October 22, 2010
• Last Updated: June 8, 2021
• Results First Posted: November 8, 2013

Record last verified: 2021-05

Locations

DK (1)