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Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

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: NCT02597114
Recruitment Status : Completed
First Posted : November 5, 2015
Last Update Posted : September 25, 2018
Sponsor:
Information provided by (Responsible Party):
ArmaGen, Inc

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 an extension of a safety and dose ranging study to obtain long term safety and exposure data, as well as information on the biological activity of the investigational drug

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis I Drug: AGT-181 Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Masking Description: Open Label
Primary Purpose: Treatment
Official Title: An Extension Study of AGT-181-102 Evaluating Safety and Glycosaminoglycans (GAGs) in Adult Patients With Hurler-Scheie or Scheie Syndrome Who Have Completed 8-Weeks of Dosing With AGT-181 in Study AGT-181-102
Actual Study Start Date : November 2015
Actual Primary Completion Date : March 19, 2018
Actual Study Completion Date : August 2, 2018


Arm Intervention/treatment
Experimental: AGT-181
AGT-181 (fusion protein of anti-human insulin receptor monoclonal antibody and alpha-L-iduronidase) administered once weekly x 26 weeks at same dose level as subject received in core study
Drug: AGT-181
intravenous infusion over 3-4 hours
Other Names:
  • HIRMAb-IDUA
  • fusion protein of monoclonal antibody to human insulin receptor fused to alpha-L-iduronidase




Primary Outcome Measures :
  1. Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: 104 weeks (2 years) ]

Secondary Outcome Measures :
  1. changes in urinary or plasma glycosaminoglycans (GAGs) [ Time Frame: 104 weeks (2 years) ]
  2. change in liver size [ Time Frame: 104 weeks (2 years) ]
  3. change in spleen size [ Time Frame: 104 weeks (2 years) ]
  4. change in levels of heparan sulfate and dermatan sulfate in the cerebrospinal fluid [ Time Frame: 104 weeks (2 years) ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have completed clinical trial AGT-181-102
  • 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)

Exclusion Criteria:

  • Refusal to complete screening evaluations.
  • Any medical condition or other circumstances that may significantly interfere with study compliance
  • Patient is pregnant or lactating
  • Clinically significant spinal cord compression, evidence of cervical instability.
  • Subject developed clinically relevant hypersensitivity to AGT-181

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02597114


Sponsors and Collaborators
ArmaGen, Inc
Investigators
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Study Director: Patrice Rioux, MD PhD ArmaGen, Inc

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Responsible Party: ArmaGen, Inc
ClinicalTrials.gov Identifier: NCT02597114     History of Changes
Other Study ID Numbers: AGT-181-103
First Posted: November 5, 2015    Key Record Dates
Last Update Posted: September 25, 2018
Last Verified: September 2018

Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis I
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Insulin
Insulin, Globin Zinc
Antibodies
Antibodies, Monoclonal
Hypoglycemic Agents
Physiological Effects of Drugs
Immunologic Factors