NCT02262338

Brief Summary

AGT-182 is a fusion protein containing idursulfase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This study is a safety and dose ranging study to obtain safety and exposure data, as well as information on the biological activity of the investigational drug.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2015

Typical duration for phase_1

Geographic Reach
3 countries

5 active sites

Status
completed

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

October 2, 2014

Completed
11 days until next milestone

First Posted

Study publicly available on registry

October 13, 2014

Completed
6 months until next milestone

Study Start

First participant enrolled

April 1, 2015

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 27, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 27, 2017

Completed
Last Updated

September 18, 2018

Status Verified

September 1, 2018

Enrollment Period

2 years

First QC Date

October 2, 2014

Last Update Submit

September 16, 2018

Conditions

Mucopolysaccharidosis II

Keywords

MPS IIHunter Syndrome

Outcome Measures

Primary Outcomes (1)

  • number of participants with adverse events as a measure of safety and tolerability

    8 weeks (ERT-naive) or 13 weeks (ERT)

Secondary Outcomes (4)

  • plasma pharmacokinetic parameters (maximal concentration, half-life, area under the curve, mean residence time, volume of distribution and clearance of AGT-182)

    8 weeks (ERT-naive) or 13 weeks (ERT)

  • change in urinary or plasma glycosaminoglycans (GAGs)

    8 weeks (ERT-naive) or 13 weeks (ERT)

  • change in liver or spleen size

    8 weeks (ERT-naive) or 13 weeks (ERT)

  • change in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs)

    8 weeks (ERT-naive) or 13 weeks (ERT)

Study Arms (1)

Treated subjects

EXPERIMENTAL

AGT-182 solution for infusion will be administered intravenously at doses of 1.0 mg/kg or 3.0 mg/kg weekly for 8-13 weeks.

Drug: AGT-182

Interventions

AGT-182DRUG

Recombinant HIRMAb-IDS

Treated subjects

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male age 18 years or older
  • Diagnosis of Hunter Syndrome (documented fibroblast or leukocyte IDS enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory - or any level of enzyme deficiency together with the presence of a pathogenic mutation in the IDS gene - and documentation of normal enzymatic activity of at least 1 other sulfatase.)
  • Must fall into one of the following groups:
  • currently receiving standard enzyme replacement therapy (ERT) and be willing to discontinue it for the study duration, taking AGT-182 instead
  • have not received standard ERT for at least 3 months and have elevated uGAGs of at least 3.5 fold above age-related normals at study screening
  • have never received ERT
  • Voluntary written consent
  • Sexually mature males must be advised to use a medically accepted method of contraception throughout the study.

You may not qualify if:

  • Refusal to complete screening/baseline evaluations
  • Receipt of an investigational drug within the prior 90 days
  • Any medical condition or other circumstances that may significantly interfere with study compliance
  • Clinically significant spinal cord compression, evidence of cervical instability
  • Known hypersensitivity to idursulfase or any of the components of AGT-182
  • Known to be nonresponsive to standard ERT treatment (i.e., high uGAG values despite taking full dose standard ERT)
  • History of diabetes mellitus or hypoglycemia
  • Contraindication to lumbar puncture, if the patient agrees to this optional assessment

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Emory University

Decatur, Georgia, 30033, United States

Location

ZKJM MC University of Mainz

Mainz, Germany

Location

Institute of Human Genetics, National Inst of Health, University of the Philippines

Manila, Philippines

Location

MeSH Terms

Conditions

Mucopolysaccharidosis IISudden Infant Death

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 DiseasesDeath, SuddenDeathPathologic ProcessesPathological Conditions, Signs and SymptomsInfant Death

Study Officials

  • Patrice Rioux, MD PhD

    ArmaGen, Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Masking Details
Open Label
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 2, 2014

First Posted

October 13, 2014

Study Start

April 1, 2015

Primary Completion

March 27, 2017

Study Completion

March 27, 2017

Last Updated

September 18, 2018

Record last verified: 2018-09

Locations

DE(1)PH(1)US(3)