NCT02663024

Brief Summary

This study evaluates the efficacy and safety of three doses of GC1111 in patients with Hunter Syndrome. Participants will be randomized to one of three doses of GC1111 or comparator.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2016

Typical duration for phase_2

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 17, 2016

Completed
9 days until next milestone

First Posted

Study publicly available on registry

January 26, 2016

Completed
10 months until next milestone

Study Start

First participant enrolled

December 1, 2016

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2019

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2020

Completed
Last Updated

January 26, 2016

Status Verified

January 1, 2016

Enrollment Period

3 years

First QC Date

January 17, 2016

Last Update Submit

January 21, 2016

Conditions

Mucopolysaccharidosis II

Outcome Measures

Primary Outcomes (1)

  • Percent change from baseline in urinary GAG(Glycosaminoglycans) at Week 25

    Baseline to Week 25

Secondary Outcomes (13)

  • Change from baseline in urinary GAG at Week 25

    Baseline to Week 25

  • Change from baseline in Six Minute Walk Test at Week 25

    Baseline to Week 25

  • Percent change from baseline in Six Minute Walk Test at Week 25

    Baseline to Week 25

  • Change from baseline in Liver volume at Week 25

    Baseline to Week 25

  • Percent change from baseline in Liver volume at Week 25

    Baseline to Week 25

  • +8 more secondary outcomes

Study Arms (4)

Arm 1

EXPERIMENTAL

0.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Biological: idursulfase beta

Arm 2

EXPERIMENTAL

1.0 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Biological: idursulfase beta

Arm 3

EXPERIMENTAL

1.5 mg/kg, iv, weekly infusion of idursulfase beta for 24 weeks

Biological: idursulfase beta

Arm 4

ACTIVE COMPARATOR

0.5mg/kg, iv, weekly infusion of idursulfase for 24 weeks

Biological: idursulfase

Interventions

idursulfase betaBIOLOGICAL

IV, weekly infusion for 24 weeks

Also known as: GC1111
Arm 1Arm 2Arm 3
idursulfaseBIOLOGICAL

0.5 mg/kg, iv, weekly infusion for 24 weeks

Also known as: Elaprase
Arm 4

Eligibility Criteria

Age5 Years - 35 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Male patients between 5 and 35 years of age
  • Informed consent form signed
  • Patients diagnosed with hunter syndrome
  • Previously untreated with an enzyme replacement therapy

You may not qualify if:

  • History of tracheostomy, bone marrow transplant, or cord blood transplant
  • Treatment with another investigational product within 30 days prior to the start of study drug
  • Known hypersensitivity of any of the ingredients of study drug
  • Patient with severe hunter syndrome who cannot perform 6MWT
  • Female patients

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Mucopolysaccharidosis II

Interventions

idursulfase beta, mouseidursulfase

Condition Hierarchy (Ancestors)

X-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHeredodegenerative Disorders, Nervous SystemMucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Sangmi Kang

CONTACT

82-31-260-1957sm6220@greencross.com

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 17, 2016

First Posted

January 26, 2016

Study Start

December 1, 2016

Primary Completion

December 1, 2019

Study Completion

June 1, 2020

Last Updated

January 26, 2016

Record last verified: 2016-01

Data Sharing

IPD Sharing
Will not share