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Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02371226
Recruitment Status : Unknown
Verified March 2017 by ArmaGen, Inc.
Recruitment status was:  Active, not recruiting
First Posted : February 25, 2015
Last Update Posted : March 6, 2017
Sponsor:
Information provided by (Responsible Party):
ArmaGen, Inc

Tracking Information
First Submitted Date  ICMJE February 19, 2015
First Posted Date  ICMJE February 25, 2015
Last Update Posted Date March 6, 2017
Actual Study Start Date  ICMJE July 2015
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 19, 2015)
Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: eight weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 19, 2015)
  • plasma pharmacokinetic parameters (maximal concentration, half-life, area under the curve, mean residence time, volume of distribution and clearance of AGT-181) [ Time Frame: 8 weeks ]
  • change in urinary or plasma glycosaminoglycans (GAGs) [ Time Frame: 8 weeks ]
  • change in liver size [ Time Frame: 8 weeks ]
  • change in spleen size [ Time Frame: 8 weeks ]
  • change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid [ Time Frame: 8 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
Official Title  ICMJE A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Alpha-L-iduronidase (HIRMAb-IDUA) Fusion Protein, AGT-181 in Adult Patients With Mucopolysaccharidosis I (MPS I, Hurler Syndrome)
Brief Summary AGT-181 is a fusion protein containing alpha-L-Iduronidase 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description:
Open Label
Primary Purpose: Treatment
Condition  ICMJE Mucopolysaccharidosis I
Intervention  ICMJE Drug: AGT-181 (HIRMAb-IDUA)
intravenous infusion over 3-4 hours
Other Name: fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase
Study Arms  ICMJE
  • Experimental: Cohort 1
    1 mg/kg AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase; HIRMAb-IDUA) administered once weekly x 8 weeks
    Intervention: Drug: AGT-181 (HIRMAb-IDUA)
  • Experimental: Cohort 2
    3 mg/kg AGT-181 (HIRMAb-IDUA) administered once weekly x 8 weeks
    Intervention: Drug: AGT-181 (HIRMAb-IDUA)
  • Experimental: Cohort 3
    6-9 mg/kg AGT-181 (HIRMAb-IDUA) administered once weekly x 8 weeks
    Intervention: Drug: AGT-181 (HIRMAb-IDUA)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Actual Enrollment  ICMJE
 (submitted: January 23, 2017)
3
Original Estimated Enrollment  ICMJE
 (submitted: February 19, 2015)
9
Estimated Study Completion Date  ICMJE June 2017
Actual Primary Completion Date February 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male age 18 years or older
  • Diagnosis of MPS I (documented fibroblast or leukocyte IDUA enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory)
  • Voluntary written consent by patient or legally responsible representative
  • All women of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
  • Negative pregnancy test (females)
  • Must not have received ERT for at least 6 weeks prior to AGT-181 treatment
  • Must have elevated urinary GAGs if no ERT has been received in the prior 3 months

Exclusion Criteria:

  • Refusal to complete baseline evaluations.
  • Any medical condition or other circumstances that may significantly interfere with study compliance
  • Receipt of an investigational drug within the prior 90 days
  • History of diabetes mellitus or hypoglycemia
  • Clinically significant spinal cord compression, evidence of cervical instability.
  • Known hypersensitivity to alpha-L-iduronidase or any of the components of AGT-181.
  • Known to be nonresponsive to standard ERT treatment.
  • Previously successful (engrafted) hematopoietic stem cell transplantation that resulted in normalization of urinary GAGs.
  • Contraindication for lumbar puncture
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02371226
Other Study ID Numbers  ICMJE AGT-181-102
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party ArmaGen, Inc
Study Sponsor  ICMJE ArmaGen, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Patrice Rioux, MD PhD ArmaGen, Inc
PRS Account ArmaGen, Inc
Verification Date March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP